DDrare: Database of Drug Development for Rare Diseases

Searched query = "Urea cycle disorder", "N-acetylglutamate synthase deficiency", "NAGS deficiency", "Carbamoyl phosphate synthetase I deficiency", "CPS1 deficiency", "Ornithine transcarbamylase deficiency", "OTC deficiency", "Classic citrullinemia", "Citrullinemia type I", "Argininosuccinic aciduria", "Argininemia", "Hiperornitinemia-hiperamonemia-homocitrulinuria syndrome", "HHH syndrome"
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 = 4-phenylbutyrate; ALLOGENIC HUMAN HETEROLOGOUS LIVER CELLS; ARCT-810; ASA; Acetohydroxamic Acid; Acetohydroxamic Acid Oral Tablet [Lithostat]; Acetohydroxamic acid; Arginine; Behavioral: Protein and calorie controlled diet; Carglumic acid; Celulas hepatocitos troncales adultas alogenicas de higado expandidas; DTX301; GT4P; Genetic: Ornithine transcarbamylase vector; Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene; Genetic: scAAV8OTC; Glyceryl tri-(4-phenylbutyrate); Glyceryl tri-(4-phenylbutyrate)(GT4P); HHALPC; HHLivC; HPN-100; HepaStem; HepaStem Infusion; Heparesc; Heterologous Human Adult Liver-derived Progenitor Cells; Heterologous Human Adult Liver-derived Progenitor Cells (HHALPC); Human Heterologous Liver Cells; Human heterologous liver cells (for infusion); Isotopic Intravenous [13C]-Urea; KB195; MRT5201; NaPBA; Neo-ASA; Nitric Oxide; Nitric oxide supplement; Oral prednisone; Ornithine; Phenylbutyrate; Prednisone; Promethera HepaStem; RAVICTI; Ravicti; SYNB1020; Sodium Phenylbutyrate; Urea

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	EUCTR2018-000156-18-FR (EUCTR)	02/11/2020	31/01/2020	A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term	A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency	Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301	Ultragenyx Pharmaceutical, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	18	Phase 1;Phase 2	United States;France;Canada;Spain;United Kingdom
2	EUCTR2018-000156-18-GB (EUCTR)	15/08/2018	02/05/2018	A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term	A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency	Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301	Ultragenyx Pharmaceutical, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	18	Phase 1;Phase 2	United States;Canada;Spain;United Kingdom
3	EUCTR2018-000156-18-ES (EUCTR)	18/06/2018	14/05/2018	A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term	A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency	Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301	Ultragenyx Pharmaceutical, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	9	Phase 1;Phase 2	United States;Canada;Spain;United Kingdom
4	EUCTR2016-001057-40-ES (EUCTR)	08/03/2017	27/12/2016	An early-phase clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency.	A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency.	Ornithine transcarbamylase deficiency MedDRA version: 19.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301	Dimension Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	12	Phase 1;Phase 2	France;United States;Canada;Spain;United Kingdom
5	NCT02991144 (ClinicalTrials.gov)	January 2017	9/12/2016	Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency	A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency	Ornithine Transcarbamylase (OTC) Deficiency	Genetic: scAAV8OTC;Drug: Oral prednisone	Ultragenyx Pharmaceutical Inc	NULL	Recruiting	18 Years	N/A	All	18	Phase 1;Phase 2	United States;Canada;Spain;United Kingdom

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

EUCTR2018-000156-18-FR
(EUCTR)

02/11/2020

31/01/2020

A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term

A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency

Ornithine transcarbamylase deficiency
MedDRA version: 20.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: DTX301
INN or Proposed INN: Not yet assigned
Other descriptive name: DTX301

Ultragenyx Pharmaceutical, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

United States;France;Canada;Spain;United Kingdom

EUCTR2018-000156-18-GB
(EUCTR)

15/08/2018

02/05/2018

A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term

Product Name: DTX301
INN or Proposed INN: Not yet assigned
Other descriptive name: DTX301

Ultragenyx Pharmaceutical, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

United States;Canada;Spain;United Kingdom

EUCTR2018-000156-18-ES
(EUCTR)

18/06/2018

14/05/2018

A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term

Product Name: DTX301
INN or Proposed INN: Not yet assigned
Other descriptive name: DTX301

Ultragenyx Pharmaceutical, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

United States;Canada;Spain;United Kingdom

EUCTR2016-001057-40-ES
(EUCTR)

08/03/2017

27/12/2016

An early-phase clinical study of a virus transferring the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency.

A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency.

Ornithine transcarbamylase deficiency
MedDRA version: 19.0;Level: LLT;Classification code 10071107;Term: Ornithine transcarbamylase deficiency;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: DTX301
INN or Proposed INN: Not yet assigned
Other descriptive name: DTX301

Dimension Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

France;United States;Canada;Spain;United Kingdom

NCT02991144
(ClinicalTrials.gov)

January 2017

9/12/2016

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency

Ornithine Transcarbamylase (OTC) Deficiency

Genetic: scAAV8OTC;Drug: Oral prednisone

Ultragenyx Pharmaceutical Inc

NULL

Recruiting

18 Years

N/A

All

Phase 1;Phase 2

United States;Canada;Spain;United Kingdom