65. Primary immunodeficiency Clinical trials / Disease details
Clinical trials : 500 / Drugs : 614 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 217
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05396105 (ClinicalTrials.gov) | December 28, 2022 | 28/4/2022 | Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema | A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-Inhibitor Deficiency (Type I or Type II) | Hereditary Angioedema;Hereditary Angioedema Type I;Hereditary Angioedema Type II;Hereditary Angioedema Types I and II;Hereditary Angioedema Attack;Hereditary Angioedema With C1 Esterase Inhibitor Deficiency;Hereditary Angioedema - Type 1;Hereditary Angioedema - Type 2;C1 Esterase Inhibitor [C1-INH] Deficiency;C1 Esterase Inhibitor Deficiency;C1 Esterase Inhibitor, Deficiency of;C1 Inhibitor Deficiency | Drug: PHA-022121 low dose;Drug: PHA-022121 medium dose;Drug: PHA-022121 high dose;Drug: PHA-022121 selected dose | Pharvaris Netherlands B.V. | NULL | Recruiting | 18 Years | N/A | All | 72 | Phase 2/Phase 3 | United States;Bulgaria;Czechia;France;Germany;Hungary;Spain |
| 2 | EUCTR2021-001226-21-DE (EUCTR) | 01/12/2022 | 08/03/2022 | A study to evaluate if different doses of KVD900 are safe and effective in treating attacks in patients with Hereditary Angioedema. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with Hereditary Angioedema Type I or II - KONFIDENT | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 300 mg Film Coated Tablet INN or Proposed INN: sebetralstat Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 114 | Phase 3 | Portugal;United States;Slovakia;Greece;Spain;North Macedonia;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Romania;Australia;Bulgaria;Germany;Netherlands;Japan;New Zealand | ||
| 3 | NCT05505916 (ClinicalTrials.gov) | October 24, 2022 | 11/8/2022 | An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE) | An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema Type I or II | Hereditary Angioedema | Drug: KVD900 600 mg | KalVista Pharmaceuticals, Ltd. | NULL | Recruiting | 12 Years | N/A | All | 150 | Phase 3 | United States;Australia;Bulgaria;France;Greece;Hungary;Netherlands;Spain;United Kingdom |
| 4 | EUCTR2021-006906-58-HU (EUCTR) | 11/07/2022 | 13/05/2022 | An extension phase 2/3 study to test the safety of long term administration of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema | A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II) - RAPIDe-2 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2;Phase 3 | France;United States;Czechia;Hungary;Canada;Spain;Poland;Bulgaria;Israel;Germany;United Kingdom;Italy | ||
| 5 | EUCTR2021-001226-21-ES (EUCTR) | 26/05/2022 | 16/03/2022 | A study to evaluate if different doses of KVD900 are safe and effective in treating attacks in patients with Hereditary Angioedema. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with Hereditary Angioedema Type I or II - KONFIDENT | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 300 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 114 | Phase 3 | United States;Greece;Spain;Israel;North Macedonia;United Kingdom;Italy;France;Hungary;Canada;Poland;Romania;Australia;Bulgaria;Netherlands;Germany;New Zealand | ||
| 6 | EUCTR2021-001226-21-HU (EUCTR) | 17/05/2022 | 11/03/2022 | A study to evaluate if different doses of KVD900 are safe and effective in treating attacks in patients with Hereditary Angioedema. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with Hereditary Angioedema Type I or II - KONFIDENT | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 300 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 114 | Phase 3 | United States;Greece;Spain;Israel;North Macedonia;United Kingdom;Italy;France;Hungary;Canada;Poland;Romania;Australia;Bulgaria;Germany;Netherlands;New Zealand | ||
| 7 | NCT05047185 (ClinicalTrials.gov) | April 19, 2022 | 7/9/2021 | Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-Inhibitor Deficiency (Type I or Type II) | Hereditary Angioedema;Hereditary Angioedema Type I;Hereditary Angioedema Type II;Hereditary Angioedema Types I and II;Hereditary Angioedema Attack;Hereditary Angioedema With C1 Esterase Inhibitor Deficiency;Hereditary Angioedema - Type 1;Hereditary Angioedema - Type 2;C1 Esterase Inhibitor Deficiency;C1 Inhibitor Deficiency | Drug: PHA-022121 low dose;Drug: PHA-022121 high dose;Drug: Placebo | Pharvaris Netherlands B.V. | NULL | Recruiting | 18 Years | 75 Years | All | 30 | Phase 2 | United States;Bulgaria;Canada;Germany;Israel;Italy;Poland;Spain;United Kingdom |
| 8 | EUCTR2021-000227-13-BG (EUCTR) | 07/03/2022 | 02/12/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for prophylaxis of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II) - HAE CHAPTER-1 | Hereditary angioedema due to C1-Inhibitor Deficiency (Type I or Type II) MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Level: LLT;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 INN or Proposed INN: Not established Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | Canada;Austria;United States;Ireland;Poland;Italy;United Kingdom;Israel;Bulgaria;Germany;Spain | ||
| 9 | NCT05259917 (ClinicalTrials.gov) | February 23, 2022 | 4/2/2022 | A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE) | A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema Type I or II | Hereditary Angioedema | Drug: Placebo;Drug: KVD900 600 mg;Drug: KVD900 300 mg | KalVista Pharmaceuticals, Ltd. | NULL | Recruiting | 12 Years | N/A | All | 114 | Phase 3 | United States;Australia;Bulgaria;Canada;France;Germany;Greece;Hungary;Israel;Italy;Netherlands;New Zealand;North Macedonia;Poland;Puerto Rico;Romania;Spain;United Kingdom |
| 10 | EUCTR2021-000227-13-PL (EUCTR) | 02/02/2022 | 18/11/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for prophylaxis of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II) - HAE CHAPTER-1 | Hereditary angioedema due to C1-Inhibitor Deficiency (Type I or Type II) MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Level: LLT;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 INN or Proposed INN: Not established Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | United States;Canada;Poland;Israel;Bulgaria;Germany;United Kingdom | ||
| 11 | EUCTR2021-000136-59-BG (EUCTR) | 12/11/2021 | 05/10/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Modified Release Tablets INN or Proposed INN: To be confirmed Other descriptive name: KVD824 hydrochloride | Kalvista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 12 | EUCTR2021-000136-59-IT (EUCTR) | 21/10/2021 | 27/07/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II - N/A | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Compresse a rilascio modificato Product Code: [N/A] Other descriptive name: KVD824 hydrochloride | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 13 | EUCTR2021-000136-59-CZ (EUCTR) | 13/10/2021 | 25/08/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Modified Release Tablets INN or Proposed INN: To be confirmed Other descriptive name: KVD824 hydrochloride | Kalvista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 14 | NCT05055258 (ClinicalTrials.gov) | September 27, 2021 | 14/9/2021 | A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or II | Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of 3 Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Angioedema, Hereditary, Types I and II | Drug: KVD824;Drug: Placebo to KVD824 | KalVista Pharmaceuticals, Ltd. | NULL | Terminated | 18 Years | N/A | All | 33 | Phase 2 | United States;Australia;Bulgaria;Canada;Czechia;France;Germany;Hungary;Italy;New Zealand;North Macedonia;Puerto Rico;United Kingdom |
| 15 | EUCTR2021-000136-59-HU (EUCTR) | 24/09/2021 | 16/07/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Modified Release Tablets INN or Proposed INN: To be confirmed Other descriptive name: KVD824 hydrochloride | Kalvista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 16 | EUCTR2021-000136-59-DE (EUCTR) | 20/09/2021 | 22/07/2021 | A study to assess whether different doses of KVD824 are effective in preventing attacks of Hereditary Angiodedema Type I or Type II. | A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial to Evaluate the Efficacy and Safety of Three Dose Levels of KVD824, an Oral Plasma Kallikrein Inhibitor, for Long-Term Prophylactic Treatment of Hereditary Angioedema Type I or II | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD824 300 mg Modified Release Tablets INN or Proposed INN: To be confirmed Other descriptive name: KVD824 hydrochloride | Kalvista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 2 | United States;Czechia;North Macedonia;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Romania;Australia;Bulgaria;Germany;New Zealand | ||
| 17 | EUCTR2020-003445-11-BG (EUCTR) | 14/04/2021 | 02/03/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Germany;Netherlands | ||
| 18 | EUCTR2020-003445-11-NL (EUCTR) | 14/04/2021 | 01/12/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Netherlands;Germany | ||
| 19 | EUCTR2020-003445-11-DE (EUCTR) | 26/02/2021 | 11/02/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | Spain;Israel;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Netherlands | ||
| 20 | EUCTR2020-003445-11-PL (EUCTR) | 23/02/2021 | 03/12/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Not Recruiting | Female: yes Male: yes | 72 | Phase 2 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Poland;Bulgaria;Netherlands;Germany | ||
| 21 | EUCTR2020-003445-11-HU (EUCTR) | 27/01/2021 | 30/11/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | France;Hungary;Canada;Spain;Poland;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 22 | EUCTR2020-003445-11-FR (EUCTR) | 18/12/2020 | 22/10/2020 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | France;Czech Republic;Hungary;Canada;Spain;Poland;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 23 | NCT04414046 (ClinicalTrials.gov) | July 22, 2020 | 24/5/2020 | TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children | Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children | Primary Immune Deficiency Disorders;Metabolic Disease | Biological: Haploidentical Hematopoietic Cell Transplantation | Johns Hopkins All Children's Hospital | NULL | Recruiting | N/A | 21 Years | All | 17 | Phase 2 | United States |
| 24 | EUCTR2019-001693-28-DE (EUCTR) | 06/02/2020 | 02/09/2019 | Single-arm pharmacokinetic phase 2a study of a single dose intravenous human plasma-derived C1-INH involving 20 HAE type I or type II patients, aged 18 years or older | Prospective, open-label, single arm, multicenter, pharmacokinetic, and safety study of a single dose intravenous human plasma-derived C1 Esterase Inhibitor (C1-INH) concentrate in patients with congenital C1-INH deficiency and hereditary angioedema - CONE-01 | Hereditary angioedema type I and type II MedDRA version: 20.0;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: C1 Esterase Inhibitor Human Product Code: OCTA-C1-INH Other descriptive name: C1 ESTERASE INHIBITOR (HUMAN) | Octapharma Pharmazeutika Produktionsges.m.b.H. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Czech Republic;Russian Federation;Hungary;Ukraine;Poland;Belarus;Bulgaria;Serbia;Germany | ||
| 25 | EUCTR2018-004489-32-AT (EUCTR) | 02/10/2019 | 07/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 26 | EUCTR2018-004489-32-PL (EUCTR) | 29/08/2019 | 29/05/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 27 | EUCTR2018-004489-32-IT (EUCTR) | 23/07/2019 | 05/01/2021 | Uno studio per a) valutare la tollerabilità e i livelli ematici di KVD900 quando somministrato come dose singola ai pazienti e b) per valutare se KVD900 è efficace nel trattamento di attacchi di gonfiore nei pazienti con la malattia genetica , angioedema ereditario. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II - KVD900-201 | Hereditary Angioedema Type I or II MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet Product Code: [KVD900 100 mg Film Coated Tablet] | KalVista Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | France;United States;Czechia;Hungary;Poland;Austria;Netherlands;Germany;United Kingdom;Italy | ||
| 28 | NCT04208412 (ClinicalTrials.gov) | July 2, 2019 | 20/12/2019 | A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II | A Randomized, Double-blind, Placebo-controlled, Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II | Hereditary Angioedema | Drug: KVD900;Drug: Placebo | KalVista Pharmaceuticals, Ltd. | NULL | Completed | 18 Years | N/A | All | 68 | Phase 2 | United States;Austria;Czechia;Germany;Hungary;Italy;Netherlands;North Macedonia;Poland;United Kingdom |
| 29 | EUCTR2018-004489-32-DE (EUCTR) | 24/06/2019 | 12/02/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Poland;Austria;North Macedonia;Netherlands;Germany;Italy;United Kingdom | ||
| 30 | EUCTR2018-004489-32-NL (EUCTR) | 19/06/2019 | 25/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | United States;Hungary;Poland;Austria;North Macedonia;Germany;Netherlands;Italy;United Kingdom | ||
| 31 | EUCTR2018-004489-32-HU (EUCTR) | 21/05/2019 | 25/03/2019 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 2 | Hungary;Macedonia, the former Yugoslav Republic of;Poland;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 32 | EUCTR2018-004489-32-GB (EUCTR) | 13/03/2019 | 27/12/2018 | A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease, Hereditary Angioedema. | A randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, an oral plasma kallikrein inhibitor, in the on-demand treatment of angioedema attacks in adult subjects with hereditary angioedema type I or II | Hereditary Angioedema Type I or II;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 100 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Hungary;Macedonia, the former Yugoslav Republic of;Austria;Netherlands;Germany;United Kingdom | ||
| 33 | EUCTR2018-000673-68-GB (EUCTR) | 09/10/2018 | 27/07/2018 | A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined Immunodeficiency | Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 | Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening. MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells INN or Proposed INN: Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 34 | EUCTR2018-001489-41-SE (EUCTR) | 02/07/2018 | 08/05/2018 | A clinical trial using cell transplantation as treatment for bone marrow failure due to dyskeratosis congenita / telomere disease, inherited genetic conditions. | Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease | Dyskeratosis congenita / telomere disease MedDRA version: 20.0;Level: PT;Classification code 10001756;Term: Allogenic bone marrow transplantation therapy;System Organ Class: 10042613 - Surgical and medical procedures ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Lemtrada INN or Proposed INN: Alemtuzumab Other descriptive name: ALEMTUZUMAB Trade Name: Fludarabine INN or Proposed INN: FLUDARABINE | Karolinska Universitetssjukhuset | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Sweden | ||
| 35 | NCT03576469 (ClinicalTrials.gov) | June 13, 2018 | 15/6/2018 | A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions | CVI - Common Variable Immunodeficiency | Biological: C1-esterase inhibitor [recombinant] (C1-INH-R) | IMMUNOe Research Centers | NULL | Completed | 18 Years | N/A | All | 20 | Phase 4 | United States |
| 36 | EUCTR2017-001275-23-GB (EUCTR) | 21/09/2017 | 03/07/2017 | A clinical trial to study the effects of genetically modified patients' CD34+ cells | Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase Deficiency | Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.1;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: cryopreserved EFS-ADA LV transduced patient CD34+ cells Product Code: OTL-101 INN or Proposed INN: There is no recommended INN Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 2 | United Kingdom | ||
| 37 | NCT04136028 (ClinicalTrials.gov) | September 25, 2015 | 21/10/2019 | IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous Disease | A Retrospective Analysis of Efficacy and Safety of Interleukin-1 Receptor Inhibitor for the Treatment of Granulomatous Complications in Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Kineret | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Completed | N/A | 18 Years | All | 13 | Early Phase 1 | Russian Federation |
| 38 | EUCTR2010-023483-41-FR (EUCTR) | 24/04/2013 | 04/01/2011 | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) | A MULTICENTER STUDY ON THE EFFICACY, SAFETY AND PHARMACOKINETICS OF I10E IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY (PID) | a primary immunodeficiency as defined by the ESID and validated by a reference centre :• X-linked agammaglobulinemia (XLA)• Autosomal recessive inherited agammaglobulinemia (including autosomal recessive hyper-IgM syndrome)• Common variable immunodeficiency (CVID)• IgG subclass deficiency (at least 2 subclasses) with clinical manifestations of immunodeficiency MedDRA version: 12.1;Level: LLT;Classification code 10010112;Term: Common variable immunodeficiency | Product Name: HUMAN NORMAL IMMUNOGLOBULIN FOR Product Code: I10E INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE | LFB BIOTECHNOLOGIES | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | France;Hungary | ||
| 39 | EUCTR2010-024253-36-GB (EUCTR) | 03/08/2012 | 09/05/2012 | A clinical trial to study the effects of genetically modified patients' CD34+ cells | Phase I/II, historical controlled, open-label, non-randomised, single-centre trial to assess the safety and efficacy of EF1aS-ADA lentiviral vector mediated gene modification of autologus CD34+ cells from ADA-deficient individuals - LV Gene Therapy for ADA Deficiency | Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.0;Level: LLT;Classification code 10066372;Term: ADA deficiency;System Organ Class: 100000012248;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: EF1aS-ADA lentiviral vector transduced patient CD34+ cells Product Code: transduced patient CD34+ cells INN or Proposed INN: EF1aS-ADA lentiviral vector gene modified autologous CD34+ cells Other descriptive name: Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene | Great Ormond Street Hospital for Children NHS Trust | NULL | Not Recruiting | Female: no Male: yes | 10 | Phase 1;Phase 2 | United Kingdom | ||
| 40 | EUCTR2005-003201-81-DE (EUCTR) | 20/12/2006 | 19/01/2007 | GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHE | GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHE | Herpes simplex virus type-1 (HSV-1) is the most frequent cause of fatal sporadic encephalitis in humans. Herpes simplex encephalitis (HSE) was one of the first viral infections to be successfully treated with antiviral drugs. Mortality has been significantly reduced since the introduction of acyclovir, a specific inhibitor of HSV replication. Despite appropriate and promptly initiated antiviral therapy the incidence of persistent neurological deficits remain unacceptably high. | Trade Name: Fortecortin Inject 40 mg Amp. Product Name: Fortecortin INN or Proposed INN: dexamethasone 21-dihydrogen phosphate | Universitätsklinikum Heidelberg | NULL | Not Recruiting | Female: yes Male: yes | 450 | Phase 3 | Germany | ||
| 41 | NCT00358657 (ClinicalTrials.gov) | May 24, 2006 | 28/7/2006 | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders | HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide | Immunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;Donor | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| 42 | NCT00325078 (ClinicalTrials.gov) | May 2006 | 11/5/2006 | Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease | Tumor Necrosis Factor Alpha Inhibitor (Lnfliximab, Adalimumab) Treatment for Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease: A Phase I/II Study Assessing Clinical and Immune Responses to Treatment and Genetic Influences | Chronic Granulomatous Disease;Crohn'S-like IBD;Inflammatory Bowel Disease (IBD) | Drug: Infliximab | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 10 Years | N/A | All | 40 | Phase 1/Phase 2 | United States |
| 43 | NCT00263237 (ClinicalTrials.gov) | December 2, 2005 | 7/12/2005 | STA-5326 Meslylate to Treat Gut Inflammation Associated With Common Variable Immunodeficiency | A Pilot Study of Safety and Efficacy of the Oral IL-12/23 Inhibitor, STA-5326 Mesylate, for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency | Common Variable Immunodeficiency | Drug: STA-5326 | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Completed | 18 Years | 75 Years | All | 10 | Phase 1 | United States |
| 44 | NCT00119431 (ClinicalTrials.gov) | September 2005 | 4/7/2005 | Kinetics, Efficacy and Safety of C1-Esteraseremmer-N | Pharmacokinetics, Clinical Efficacy and Safety of C1 Inhibitor Concentrate (C1-Esteraseremmer-N) for the Treatment of Hereditary (and Acquired) Angioedema | Hereditary Angioedema Type I;Angioneurotic Edema | Drug: C1 inhibitor concentrate | Sanquin | NULL | Completed | 18 Years | N/A | Both | 12 | Phase 2 | Netherlands |
| 45 | NCT00004787 (ClinicalTrials.gov) | December 1994 | 24/2/2000 | Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes | Shwachman Syndrome;Fanconi's Anemia;Dyskeratosis Congenita;Thrombocytopenia | Drug: filgrastim | National Center for Research Resources (NCRR) | James Whitcomb Riley Hospital for Children | Completed | N/A | N/A | Both | 20 | Phase 2 | NULL | |
| 46 | EUCTR2020-003445-11-CZ (EUCTR) | 11/02/2021 | A blinded, placebo-controlled and randomized phase 2 study to test different doses of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema (HAE). | A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1 | Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency MedDRA version: 23.1;Level: PT;Classification code 10019860;Term: Hereditary angioedema;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;Classification code 10080957;Term: Hereditary angioedema C1 inhibitor deficiency;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Immune system processes [G12] | Product Name: PHVS416 Product Code: PHA-022121 Other descriptive name: PHA-022121 | Pharvaris Netherlands BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2 | Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Belgium;Poland;Germany;Netherlands | |||
| 47 | EUCTR2021-001226-21-NL (EUCTR) | 28/03/2022 | A study to evaluate if different doses of KVD900 are safe and effective in treating attacks in patients with Hereditary Angioedema. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with Hereditary Angioedema Type I or II - KONFIDENT | Hereditary Angioedema Type I or II MedDRA version: 21.0;Level: LLT;Classification code 10080956;Term: Hereditary angioedema type I;System Organ Class: 100000004850 MedDRA version: 24.0;Classification code 10080960;Term: Hereditary angioedema type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: KVD900 300 mg Film Coated Tablet INN or Proposed INN: None Other descriptive name: KVD900 | KalVista Pharmaceuticals Ltd | NULL | NA | Female: yes Male: yes | 114 | Phase 3 | United States;Greece;Spain;Israel;North Macedonia;United Kingdom;Italy;France;Hungary;Canada;Poland;Romania;Australia;Bulgaria;Netherlands;Germany;New Zealand |