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Gene Therapy

Gene Therapy for Ornithine Transcarbamylase Deficiency

Phase 3

Recruiting

Research Sponsored by Ultragenyx Pharmaceutical Inc

Eligibility Criteria Checklist

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

Must have

Confirmed clinical diagnosis of late-onset OTC deficiency with historical documentation via enzymatic (ie, liver biopsy), biochemical (ie, hyperammonemia in the presence of elevated plasma glutamine, low citrulline, and elevated spot urine orotic acid), or molecular testing (ie, OTC analysis)

Free from symptomatic hyperammonemia and has not required emergent active intervention for hyperammonemia within 4 weeks before screening/baseline

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to week 324

Awards & highlights

Study Summary

This trial is testing a new drug to see if it helps people with a certain genetic disorder by allowing them to eat more protein without having high levels of ammonia in their blood.

Who is the study for?

This trial is for people with late-onset OTC deficiency who are on a stable dose of ammonia scavenger therapy and diet, have safe plasma ammonia levels, and agree to use effective contraception. It's not for those in other gene studies, with active hepatitis or significant liver issues, infections, conditions that risk participation or skew results, or detectable antibodies against the AAV8 capsid.Check my eligibility

What is being tested?

The study tests DTX301's ability to improve OTC function by maintaining safe ammonia levels without dietary protein restrictions or alternative meds. Participants will receive either DTX301 with oral corticosteroids or placebos for both and their efficacy will be compared.See study design

What are the potential side effects?

Potential side effects may include reactions related to gene transfer such as immune responses against the vector (AAV8), complications from corticosteroid use like increased infection risk, blood sugar changes, bone weakening among others.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with late-onset OTC deficiency through tests.

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I haven't needed urgent treatment for high ammonia levels in the last 4 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to week 324

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to week 324

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 324 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Percentage of Participants Who Have Achieved Complete Response

Plasma Ammonia as Measured by 24-hour Ammonia (AUC0-24)

Secondary outcome measures

Change from Baseline in Plasma Ammonia (AUC0-24)

Change from Baseline in Plasma Ammonia (AUC0-24) After DTX301 Exposure

Antibodies

+5 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: Placebo, Then DTX301Experimental Treatment5 Interventions

Participants receive single peripheral IV infusion of placebo. At week 64, participants receive single peripheral IV infusion of DTX301 in solution.

Group II: DTX301, Then PlaceboExperimental Treatment5 Interventions

Participants receive single peripheral intravenous (IV) infusion of DTX301 in solution. At week 64, participants receive single peripheral IV infusion of placebo.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Oral Corticosteroids

2015

Completed Phase 4

~1080

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Ultragenyx Pharmaceutical IncLead Sponsor

89 Previous Clinical Trials

179,025 Total Patients Enrolled

Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc

2,777 Previous Clinical Trials

8,063,814 Total Patients Enrolled

Media Library

DTX301 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05345171 — Phase 3

Ovarian Tissue Cryopreservation Research Study Groups: Placebo, Then DTX301, DTX301, Then Placebo

Ovarian Tissue Cryopreservation Clinical Trial 2023: DTX301 Highlights & Side Effects. Trial Name: NCT05345171 — Phase 3

DTX301 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05345171 — Phase 3

Frequently Asked Questions

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

Are there any current openings for participants in this trial?

"That is accurate, the clinicaltrials.gov website shows that this study is open for recruitment. The listing was first created on December 1st, 2022 and updated October 19th, just over 50 patients are needed from 7 different sites."

Answered by AI

In how many different hospitals is this research being conducted?

"There are 7 clinical trial sites available, which are located in University of California in Los Angeles, The Hospital for Sick Children in Toronto and University of Colorado in Aurora among other places."

Answered by AI

Has the FDA looked favorably upon DTX301 in the past?

"There is available clinical data that suggests DTX301's efficacy and safety, so it received a score of 3."

Answered by AI

How many people have signed up to participate in this clinical trial?

"That is accurate. The clinicaltrials.gov website does show that this study is currently looking for 50 participants at 7 sites. This trial was posted on December 1st, 2022 and the most recent update was October 19th, 2022."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency

2022. "Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05345171.

All > Ornithine Transcarbamylase Deficiency