254. ポルフィリン症 Porphyria Clinical trials / Disease details
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 72 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 | NCT05308472 (ClinicalTrials.gov) | October 31, 2022 | 21/3/2022 | Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Parti ... | (AURORA) A Randomized, Double-blind, Placebo-Controlled Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX (PPIX) Concentrations in Participants With Erythropoietic Protoporphyria (EPP) (AURORA) A Randomized, Double-blind, Placebo-Controlled Study of Bitopertin to Evaluate the Safety, ... | Erythropoietic Protoporphyria | Drug: DISC-1459;Drug: Placebo | Disc Medicine, Inc | NULL | Recruiting | 18 Years | N/A | All | 75 | Phase 2 | United States |
| 2 | NCT05020184 (ClinicalTrials.gov) | June 14, 2022 | 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;Wake Forest University Health Sciences | Recruiting | 15 Years | N/A | All | 20 | Phase 2 | United States |
| 3 | EUCTR2018-004164-60-NL (EUCTR) | 19/05/2022 | 21/02/2022 | Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients with Variegate Porphyria (VP)-related skin disease Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients with Variegate Porphyria(VP)- ... | A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients with Variegate Porphyria (VP)-related skin disease. - Phase IIa VP Study A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Afamelanotide ... | Variegate Porphyria (VP)-related skin disease;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Variegate Porphyria(VP)-related skin disease;Therapeutic area: Body processes [G] - Metabolic Phenom ... | Trade Name: SCENESSE | CLINUVEL (UK) LTD | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 6 | Phase 2 | Netherlands;Switzerland | ||
| 4 | EUCTR2021-001831-17-DE (EUCTR) | 07/04/2022 | 22/11/2021 | 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;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden | ||
| 5 | 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 | ||
| 6 | 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 | Active, not recruiting | 12 Years | 75 Years | All | 175 | Phase 3 | United States;Australia;Canada;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom |
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | Complete | >= 12age old | Not applicable | Both | 10 | N/A | Japan |
| 10 | 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 |