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"
4 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
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-IT
(EUCTR)
08/10/202021/10/2020Study to Evaluate Efficacy, Safety, and Tolerability of MT 7117 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]
Other descriptive name: A novel synthetic, orally-administered, non-peptide small molecule, which acts as
Mitsubishi Tanabe Development America 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
3EUCTR2019-004226-16-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
4EUCTR2019-004226-16-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