DDrare: Database of Drug Development for Rare Diseases

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.

Drug = 1639325-43-1; 5-probe cocktail; A novel synthetic, orally-administered, non-peptide small molecule, which acts as; ALN-60519; ALN-AS1; Afamelanotide; Afamelanotide Implant; CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid); CAS# in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid); COLESTYRAMINE; CUV1647; Chlorpromazine; Chlorpromazine Hydrochloride; Colestipol; Colestyramine; Concentration unit: mg milligram(s); Cysteine; Cysteine hydrochloride; Deferasirox; Dersimelagon; Exjade; Givosiran; Givosiran (ALN-AS1); Glucose; Harvoni; Heme arginate; Hemin; Hemin for injection; Hydroxychloroquine; Iron; Isoniazid; Luteinizing hormone; Luteinizing hormone-releasing factor; MT-7117; MT-7117 High Dose; MT-7117 Low Dose; MT-7117 high dose; MT-7117 low dose; Nle4-D-Phe7-alpha-MSH; Oral Iron; Other: Glucose; Panhematin; Porphobilinogen; Procedure: Phlebotomy; Questran; Recombinant human porphobilinogen deaminase (Porphozym); SCENESSE; Scenesse; Sterile Normal Saline (0.9% NaCl); Tin mesoporphyrin; [MT-7117]; [Nle4,D-Phe7]-a-melanocyte stimulating hormone or NDP-a-MSH

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	NCT04402489 (ClinicalTrials.gov)	June 1, 2020	20/5/2020	Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoetic Protoporphyria or X-Linked Protoporphyria	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	EPP;XLP	Drug: Placebo;Drug: MT-7117 Low Dose;Drug: MT-7117 High Dose	Mitsubishi Tanabe Pharma Development America, Inc.	NULL	Recruiting	12 Years	75 Years	All	159	Phase 3	United States;Australia;Canada;Finland;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom
2	NCT03520036 (ClinicalTrials.gov)	July 5, 2018	23/4/2018	Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria	A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria	Erythropoietic Protoporphyria (EPP)	Drug: MT-7117 low dose;Drug: MT-7117 high dose;Drug: Placebo	Mitsubishi Tanabe Pharma Development America, Inc.	NULL	Completed	18 Years	75 Years	All	102	Phase 2	United States

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

NCT04402489
(ClinicalTrials.gov)

June 1, 2020

20/5/2020

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoetic Protoporphyria or X-Linked Protoporphyria

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

EPP;XLP

Drug: Placebo;Drug: MT-7117 Low Dose;Drug: MT-7117 High Dose

Mitsubishi Tanabe Pharma Development America, Inc.

NULL

Recruiting

12 Years

75 Years

All

159

Phase 3

United States;Australia;Canada;Finland;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom

NCT03520036
(ClinicalTrials.gov)

July 5, 2018

23/4/2018

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

Erythropoietic Protoporphyria (EPP)

Drug: MT-7117 low dose;Drug: MT-7117 high dose;Drug: Placebo

Mitsubishi Tanabe Pharma Development America, Inc.

NULL

Completed

18 Years

75 Years

All

102

Phase 2

United States