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Gene Therapy

Gene Therapy for Urea Cycle Disorders

Phase 1

Waitlist Available

Research Sponsored by National Center for Research Resources (NCRR)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients at least 6 months old with ornithine transcarbamylase deficiency (OTC) including: Hemizygous OTC or homozygous autosomal recessive disorder with evidence of complete enzyme deficiency

Metabolically stable heterozygous OTC females aged 18 to under 65

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study Summary

This trial will study the effects of gene therapy on patients with urea cycle disorders.

Who is the study for?

This trial is for people with urea cycle disorders, specifically those who lack an enzyme to remove ammonia from the blood. It includes patients over 6 months old with ornithine transcarbamylase deficiency and their heterozygote relatives. Participants must be metabolically stable, not hospitalized for hyperammonemia before, and not pregnant.Check my eligibility

What is being tested?

The study focuses on gene therapy as a potential treatment for urea cycle disorders. By altering genes to function normally, researchers hope to improve nitrogen removal from the blood in affected individuals. The trial has two parts: treatment evaluation and metabolism studies.See study design

What are the potential side effects?

While specific side effects are not listed here, gene therapy can sometimes cause immune reactions, flu-like symptoms, swelling at the injection site, headache or nausea. Each person's reaction may vary.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am over 6 months old with a confirmed diagnosis of OTC deficiency.

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I am a woman aged 18-64 with stable OTC deficiency.

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My orotic acid levels are 5 times higher than normal despite taking allopurinol.

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I have never been hospitalized for high ammonia levels.

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I carry one gene for an inherited disorder, confirmed by family testing.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

0 / 5Eligibility criteria met

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

National Center for Research Resources (NCRR)Lead Sponsor

537 Previous Clinical Trials

316,950 Total Patients Enrolled

Baylor College of MedicineOTHER

1,001 Previous Clinical Trials

6,002,114 Total Patients Enrolled

Brendan LeeStudy ChairBaylor College of Medicine

Media Library

Treatment for Urea Cycle Disorders (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT00004307 — Phase 1

Inborn Errors of Amino Acid Metabolism Research Study Groups:

Inborn Errors of Amino Acid Metabolism Clinical Trial 2023: Treatment for Urea Cycle Disorders Highlights & Side Effects. Trial Name: NCT00004307 — Phase 1

Treatment for Urea Cycle Disorders (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT00004307 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are older adults being included in this investigation?

"This study is open to those aged 6 months old up to 64 years. Additionally, there are 13 separate trials available for minors and 9 studies orientated towards individuals above the pensionable age."

Answered by AI

Has this remedy earned authorization from the FDA?

"Our team has assessed the safety of this medical intervention as a 1 because it is in its earliest clinical trial stage, meaning that there are only limited data points confirming efficacy and safety."

Answered by AI

Is this research endeavor currently accepting new participants?

"According to clinicaltrials.gov, this medical trial is no longer seeking patients; the initial posting date was December 1st 1999 and the last updated date being June 23rd 2005. However, there are 18 other trials that actively seek patient participation at this time."

Answered by AI

Am I a suitable candidate to participate in this experiment?

"This clinical trial necessitates that applicants suffer from a congenital metabolic disorder and be between 6 months to 64 years of age. The total number of participants is slated at 66 individuals."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

1999. "Study of Treatment and Metabolism in Patients With Urea Cycle Disorders". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT00004307.

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