254. Porphyria
59 clinical trials,   52 drugs   (DrugBank: 17 drugs),   17 drug target genes,   33 drug target pathways

Searched query = "Porphyria", "CEP", "Erythropoietic protoporphyria", "EPP", "X-linked dominant protoporphyria", "XLDP", "PCT", "HEP", "AIP", "ADP", "VP", "Hereditary coproporphyria", "HCP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1EUCTR2019-004226-16-GB
(EUCTR)
04/11/202029/05/2020A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked ProtoporphyriaA 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 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]
Product Name: Dersimelagon
Product Code: MT-7117
INN or Proposed INN: Dersimelagon
Other descriptive name: MT-7117
Mitsubishi Tanabe Pharma Development America (MTDA), Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
159Phase 3United States;Canada;Finland;Spain;Australia;Norway;Germany;Japan;Italy;United Kingdom;Sweden
2EUCTR2019-004226-16-SE
(EUCTR)
06/07/202026/05/2020A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked ProtoporphyriaA 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 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]
Product Name: Dersimelagon
Product Code: MT-7117
INN or Proposed INN: Dersimelagon
Other descriptive name: MT-7117
Mitsubishi Tanabe Pharma Development America (MTDA), Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
159Phase 3United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden
3NCT04402489
(ClinicalTrials.gov)
June 1, 202020/5/2020Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoetic Protoporphyria or X-Linked ProtoporphyriaA 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 ProtoporphyriaEPP;XLPDrug: Placebo;Drug: MT-7117 Low Dose;Drug: MT-7117 High DoseMitsubishi Tanabe Pharma Development America, Inc.NULLRecruiting12 Years75 YearsAll159Phase 3United States;Australia;Canada;Finland;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom
4NCT03520036
(ClinicalTrials.gov)
July 5, 201823/4/2018Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic ProtoporphyriaA Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic ProtoporphyriaErythropoietic Protoporphyria (EPP)Drug: MT-7117 low dose;Drug: MT-7117 high dose;Drug: PlaceboMitsubishi Tanabe Pharma Development America, Inc.NULLCompleted18 Years75 YearsAll102Phase 2United States
5EUCTR2019-004226-16-NO
(EUCTR)
20/05/2020A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked ProtoporphyriaA 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 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]
Product Name: Dersimelagon
Product Code: MT-7117
INN or Proposed INN: Dersimelagon
Other descriptive name: MT-7117
Mitsubishi Tanabe Pharma Development America (MTDA), Inc.NULLNAFemale: yes
Male: yes
159Phase 3United States;Canada;Finland;Spain;Australia;Germany;Norway;Japan;Italy;United Kingdom;Sweden