Treatment for Ornithine Carbamoyltransferase Deficiency Disease

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Hopital Femme Mere Enfant, Bron, France
Ornithine Carbamoyltransferase Deficiency Disease+1 More
DTX301 - Genetic
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels with removal of dietary protein restriction and alternative pathway medication.

Eligible Conditions

  • Ornithine Carbamoyltransferase Deficiency Disease
  • OTC Deficiency

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Ornithine Carbamoyltransferase Deficiency Disease

Study Objectives

2 Primary · 9 Secondary · Reporting Duration: Up to Week 324

Baseline, Week 64
Change from Baseline to Week 64 in Cogstate Cognitive Assessment
Change from Baseline to Week 64 in the Hyperammonemia Questionnaire
Change in Plasma Ammonia (AUC0-24) From Baseline to Week 64 For All Participants
Week 324
Number of Participants With Clinically Significant Changes From Baseline in Laboratory Values, Physical Examination Results, and Vital Sign Measurements
Week 64
Change from Baseline in Plasma Ammonia (AUC0-24) After Reduction or Discontinuation of Baseline Disease Management For DTX301 Participants
Pre-enrollment, Baseline, Week 64
Rate of Hyperammonemic Crises (HACs) from Baseline to Week 64 Compared to Pre-enrollment
Up to Week 324
Number of Participants With Anti-OTC Antibodies
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs, Related Serious TEAEs and Adverse Events of Special Interest (AESIs)
Week 64
Percentage of Participants Who Have Achieved Complete Response (DTX301 vs Placebo)
Percentage of Participants Who Have Achieved Complete Response, Response, or No Response
Plasma Ammonia as Measured by 24-hour Ammonia (AUC0-24) at Week 64

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Ornithine Carbamoyltransferase Deficiency Disease

Trial Design

2 Treatment Groups

DTX301, Then Placebo
1 of 2
Placebo, Then DTX301
1 of 2
Experimental Treatment

50 Total Participants · 2 Treatment Groups

Primary Treatment: Treatment · Has Placebo Group · Phase 3

DTX301, Then PlaceboExperimental Group · 5 Interventions: Placebo for oral corticosteroids, Placebo, DTX301, Oral Corticosteroids, Sodium Acetate · Intervention Types: Drug, Other, Genetic, Drug, Drug
Placebo, Then DTX301Experimental Group · 5 Interventions: Placebo for oral corticosteroids, Placebo, DTX301, Oral Corticosteroids, Sodium Acetate · Intervention Types: Drug, Other, Genetic, Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Oral Corticosteroids
2015
Completed Phase 4
~1080

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to week 324
Closest Location: University of California · Los Angeles, CA
Photo of ca university of california irvine  1Photo of ca university of california irvine  2Photo of university of california los angeles  3
1993First Recorded Clinical Trial
1 TrialsResearching Ornithine Carbamoyltransferase Deficiency Disease
155 CompletedClinical Trials

Eligibility Criteria

Age Any Age · All Participants · 7 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
Plasma ammonia level on Day 1 (predose) is ≤ 200 µmol/L.
You are on ongoing daily ammonia scavenger therapy.
You are on a protein-restricted diet.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.