DDrare: Database of Drug Development for Rare Diseases

254. ポルフィリン症
［臨床試験数：59，薬物数：52（DrugBank：17），標的遺伝子数：17，標的パスウェイ数：33］

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	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT00004398 (ClinicalTrials.gov)	January 1998	18/10/1999	Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of Porphyria	Porphyria	Drug: heme arginate;Drug: tin mesoporphyrin	National Center for Research Resources (NCRR)	University of Texas	Completed	18 Years	N/A	Both	24	Phase 1	NULL
2	NCT00004397 (ClinicalTrials.gov)	January 1998	18/10/1999	Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria	Porphyria	Drug: heme arginate;Drug: tin mesoporphyrin	National Center for Research Resources (NCRR)	University of Texas	Completed	18 Years	N/A	Both	20	Phase 1	NULL
3	NCT00004396 (ClinicalTrials.gov)	September 1997	18/10/1999	Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias	Porphyria	Drug: heme arginate;Drug: tin mesoporphyrin	National Center for Research Resources (NCRR)	University of Texas	Completed	18 Years	N/A	Both	32	Phase 2	United States
4	NCT00004789 (ClinicalTrials.gov)	July 1993	24/2/2000	Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria	Porphyria	Drug: heme arginate;Drug: tin mesoporphyrin	National Center for Research Resources (NCRR)	University of Texas	Completed	18 Years	N/A	Both	59	Phase 1;Phase 2	NULL

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

NCT00004398
(ClinicalTrials.gov)

January 1998

18/10/1999

Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of Porphyria

Porphyria

Drug: heme arginate;Drug: tin mesoporphyrin

National Center for Research Resources (NCRR)

University of Texas

Completed

18 Years

N/A

Both

Phase 1

NULL

NCT00004397
(ClinicalTrials.gov)

January 1998

18/10/1999

Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria

Porphyria

Drug: heme arginate;Drug: tin mesoporphyrin

National Center for Research Resources (NCRR)

University of Texas

Completed

18 Years

N/A

Both

Phase 1

NULL

NCT00004396
(ClinicalTrials.gov)

September 1997

18/10/1999

Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias

Porphyria

Drug: heme arginate;Drug: tin mesoporphyrin

National Center for Research Resources (NCRR)

University of Texas

Completed

18 Years

N/A

Both

Phase 2

United States

NCT00004789
(ClinicalTrials.gov)

July 1993

24/2/2000

Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria

Porphyria

Drug: heme arginate;Drug: tin mesoporphyrin

National Center for Research Resources (NCRR)

University of Texas

Completed

18 Years

N/A

Both

Phase 1;Phase 2

NULL