Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency - Tabular View

Tracking Information

First Received Date ^ICMJE

October 18, 1999

Last Updated Date

June 23, 2005

Start Date ^ICMJE

October 1995

Primary Completion Date

Current Primary Outcome Measures ^ICMJE

Original Primary Outcome Measures ^ICMJE

Change History

Complete list of historical versions of study NCT00004386 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Original Secondary Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency

Official Title ^ICMJE

Brief Summary

OBJECTIVES:

Evaluate the safety and feasibility of administering recombinant adenovirus containing the ornithine transcarbamylase gene to adults with partial ornithine transcarbamylase deficiency.

Detailed Description

PROTOCOL OUTLINE: This a dose escalation study to estimate the maximum tolerated dose of recombinant adenovirus encoded with the ornithine transcarbamylase gene.

Patients receive a single dose of virus infused into the liver under fluoroscopic guidance. Groups of 3 patients receive successively higher doses of virus; each cohort is observed for safety for 3 weeks before entry of the next group.

Patients are followed weekly for 1 month, then every 3 months until stable.

Study Phase

Phase I

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment

Condition ^ICMJE

Ornithine Transcarbamylase Deficiency Disease

Intervention ^ICMJE

Gene Transfer: recombinant adenovirus containing the ornithine transcarbamylase gene

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Terminated

Enrollment ^ICMJE

Completion Date

Primary Completion Date

Eligibility Criteria ^ICMJE

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Confirmed partial ornithine transcarbamylase deficiency Asymptomatic, i.e., at least 1 month since hyperammonemia --Prior/Concurrent Therapy-- Not specified --Patient Characteristics-- No pregnant or nursing women Negative pregnancy test required of fertile women

Gender

Both

Ages

18 Years to 65 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

Administrative Information

NCT ID ^ICMJE

NCT00004386

Responsible Party

Study ID Numbers ^ICMJE

199/12054, CSH-3660

Study Sponsor ^ICMJE

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborators ^ICMJE

Children's Research Institute

Investigators ^ICMJE

Study Chair:

Mark Batshaw

Children's Research Institute

Information Provided By

Office of Rare Diseases (ORD)

Verification Date

January 2000

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP