254. ポルフィリン症 Porphyria Clinical trials / Disease details
臨床試験数 : 70 / 薬物数 : 54 - (DrugBank : 19) / 標的遺伝子数 : 19 - 標的パスウェイ数 : 35
Showing 1 to 10 of 70 diseases
| 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-ES (EUCTR) | 01/03/2022 | 16/12/2021 | 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 Protoporphyriaor X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classificati ... | 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 | ||
| 2 | NCT05020184 (ClinicalTrials.gov) | December 2021 | 26/7/2021 | Effect of Oral Cimetidine in the Protoporphyrias | Effect of Oral Cimetidine in the Protoporphyrias | Erythropoietic Protoporphyria;X-linked Protoporphyria | Drug: Cimetidine;Drug: Placebo | Amy K. Dickey, M.D. | University of Texas | Not yet recruiting | 15 Years | N/A | All | 20 | Phase 2 | United States |
| 3 | NCT05005975 (ClinicalTrials.gov) | August 10, 2021 | 28/7/2021 | Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With ... | 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 ... | EPP;XLP | Drug: MT-7117 | Mitsubishi Tanabe Pharma Development America, Inc. | NULL | Recruiting | 12 Years | 75 Years | All | 175 | Phase 3 | United States;Australia;Canada;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom |
| 4 | NCT04923516 (ClinicalTrials.gov) | June 2021 | 8/6/2021 | Prevalence of Acute Hepatic Porphyria | Prevalence of Acute Hepatic Porphyria in Population With Suggestive Clinical Picture | Acute Hepatic Porphyria | Other: Dosage of Delta-aminolevulinic acid and Porphobilinogen | Association pour la Recherche en Medecine Interne | NULL | Not yet recruiting | 18 Years | 60 Years | All | 500 | NULL | |
| 5 | EUCTR2019-004226-16-FI (EUCTR) | 29/04/2021 | 05/10/2020 | 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 Protoporphyriaor X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classificati ... | 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 | ||
| 6 | EUCTR2019-004226-16-DE (EUCTR) | 20/04/2021 | 15/09/2020 | 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 Protoporphyriaor X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classificati ... | 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 | ||
| 7 | JPRN-jRCT2071200074 | 02/02/2021 | 24/12/2020 | Expanded Access Protocol of Givosiran for Patients with Acute Hepatic Porphyria | Expanded Access Protocol of Givosiran for Patients with Acute Hepatic Porphyria (AHP) | Acute hepatic porphyria (AHP) | Patients will be administered givosiran 2.5 mg/kg as a subcutaneous (SC) injection once monthly. | Fujii Norihisa | NULL | Not Recruiting | >= 12age old | Not applicable | Both | 10 | N/A | Japan |
| 8 | EUCTR2019-004226-16-GB (EUCTR) | 04/11/2020 | 29/05/2020 | 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 Protoporphyriaor X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classificati ... | 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 | ||
| 9 | EUCTR2019-004226-16-IT (EUCTR) | 08/10/2020 | 21/10/2020 | 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 Protoporphyriaor X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classificati ... | 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 | ||
| 10 | EUCTR2019-004226-16-SE (EUCTR) | 06/07/2020 | 26/05/2020 | 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 Protoporphyriaor X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classificati ... | 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 |