Concentration unit: mg milligram(s)
( DrugBank: - / KEGG DRUG: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 254 | ポルフィリン症 | 30 |
254. ポルフィリン症
臨床試験数 : 72 / 薬物数 : 53 - (DrugBank : 16) / 標的遺伝子数 : 19 - 標的パスウェイ数 : 35
Showing 1 to 10 of 30 trials
| 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 | EUCTR2021-001831-17-DE (EUCTR) | 07/04/202220220407 | 22/11/202120211122 | An open label, long-term extension study to determine how safe and tolerable oral Dersimelagon is in subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) An open label, long-term extension study to determine how safe and tolerable oral Dersimelagon is in ... | A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability o ... | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classificat ... | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive na ... | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden | ||
| 2 | EUCTR2021-001831-17-ES (EUCTR) | 01/03/202220220301 | 16/12/202120211216 | An open label, long-term extension study to determine how safe and tolerable oral Dersimelagon is in subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) An open label, long-term extension study to determine how safe and tolerable oral Dersimelagon is in ... | A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability o ... | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classificat ... | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive na ... | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Spain;Germany;United Kingdom;Japan | ||
| 3 | EUCTR2019-004226-16-FI (EUCTR) | 29/04/202120210429 | 05/10/202020201005 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents ... | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Saf ... | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classificat ... | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive na ... | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Not Recruiting | Female: yes Male: yes | 159 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden | ||
| 4 | EUCTR2019-004226-16-DE (EUCTR) | 20/04/202120210420 | 15/09/202020200915 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents ... | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Saf ... | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classificat ... | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive na ... | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Italy;Japan;Sweden | ||
| 5 | EUCTR2019-004226-16-GB (EUCTR) | 04/11/202020201104 | 29/05/202020200529 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents ... | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Saf ... | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classificat ... | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive na ... | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 159 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;Japan;Italy;United Kingdom;Sweden | ||
| 6 | EUCTR2019-004226-16-IT (EUCTR) | 08/10/202020201008 | 21/10/202020201021 | Study to Evaluate Efficacy, Safety, and Tolerability of MT 7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria Study to Evaluate Efficacy, Safety, and Tolerability of MT 7117 in Adults and Adolescents with Eryth ... | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT 7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Saf ... | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classificat ... | Product Name: Dersimelagon Product Code: [MT-7117] Other descriptive name: A novel synthetic, orally-administered, non-peptide small molecule, which acts as Product Name: Dersimelagon Product Code: [MT-7117] Other descriptive name: A novel synthetic, orally-a ... | Mitsubishi Tanabe Development America Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 159 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden | ||
| 7 | EUCTR2019-004226-16-SE (EUCTR) | 06/07/202020200706 | 26/05/202020200526 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents ... | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Saf ... | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classificat ... | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive na ... | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Italy;Japan;Sweden | ||
| 8 | EUCTR2017-002432-17-NL (EUCTR) | 21/06/201820180621 | 19/02/201820180219 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Can ... | ||
| 9 | EUCTR2017-002432-17-FI (EUCTR) | 21/05/201820180521 | 30/11/201720171130 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium; ... | ||
| 10 | EUCTR2017-002432-17-PL (EUCTR) | 12/04/201820180412 | 16/01/201820180116 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Czechia;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Brazil;Belgium;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of United States;Czechia;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Me ... |