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_ gender	Target_ size	Phase	Countries
1	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, 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.	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
2	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	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	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.	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
3	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, 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.	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
4	EUCTR2019-004226-16-NO (EUCTR)		20/05/2020	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, 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.	NULL	NA	Female: yes Male: yes	159	Phase 3	United States;Canada;Finland;Spain;Australia;Germany;Norway;Japan;Italy;United Kingdom;Sweden

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

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, 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.

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

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

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

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.

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

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

Product Name: Dersimelagon
Product Code: MT-7117
INN or Proposed INN: Dersimelagon
Other descriptive name: MT-7117

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;United Kingdom;Japan;Italy;Sweden

EUCTR2019-004226-16-NO
(EUCTR)

20/05/2020

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

Product Name: Dersimelagon
Product Code: MT-7117
INN or Proposed INN: Dersimelagon
Other descriptive name: MT-7117

Mitsubishi Tanabe Pharma Development America (MTDA), Inc.

NULL

Female: yes
Male: yes

159

Phase 3

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