Suspended

Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency

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What is being tested

recombinant adenovirus containing the ornithine transcarbamylase gene

Genetic
Who is being recruted

Amino Acid Metabolism, Inborn Errors+15

+ Brain Diseases

+ Brain Diseases, Metabolic

From 18 to 65 Years
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 1
Interventional
Study Start: October 1995
See protocol details

Summary

Principal SponsorEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Last updated: June 24, 2005
Sourced from a government-validated database.Claim as a partner

Study start date: October 1, 1995

Actual date on which the first participant was enrolled.

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.

NCT00004386
Principal SponsorEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Last updated: June 24, 2005
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 65 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Amino Acid Metabolism, Inborn ErrorsBrain DiseasesBrain Diseases, MetabolicCentral Nervous System DiseasesMetabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNutritional and Metabolic DiseasesPathologic ProcessesPathological Conditions, Signs and SymptomsBrain Diseases, Metabolic, InbornDisease AttributesGenetic Diseases, InbornGenetic Diseases, X-LinkedMetabolism, Inborn ErrorsOrnithine Carbamoyltransferase Deficiency DiseaseRare DiseasesUrea Cycle Disorders, Inborn

Criteria

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

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
This study has no location dataSave this study to your profile to know when the location data is available.
SuspendedNo study centers