← Back to Search

Gene Therapy

Gene Therapy for Glycogen Storage Disease Type Ia

Phase 3
Waitlist Available
Research Sponsored by Ultragenyx Pharmaceutical Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status
Females of childbearing potential and fertile males must consent to use highly effective contraception from the period following informed consent through the duration of the 144-week study and in cases of early withdrawal at least 48 weeks after the last dose of investigational product (IP). Female subjects must agree not to become pregnant. Male subjects must agree not to father a child or donate sperm
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 144 weeks
Awards & highlights

Study Summary

This trial is testing a new drug to see if it can help people with diabetes better control their blood sugar levels and reduce their dependence on exogenous (external) sources of glucose.

Who is the study for?
This trial is for individuals with Glycogen Storage Disease Type Ia (GSDIa) who are on a stable regimen of cornstarch therapy. They must be willing to use effective contraception and not plan pregnancy. Excluded are those with liver disease, previous gene therapies, detectable AAV8 antibodies, or certain lab abnormalities.Check my eligibility
What is being tested?
The study tests DTX401's ability to reduce reliance on glucose replacement in GSDIa patients versus placebo. It aims to improve glucose control quality over a period of 144 weeks, comparing the effects of an oral corticosteroid treatment against a placebo.See study design
What are the potential side effects?
Potential side effects may include immune reactions to the viral vector used for gene transfer (AAV8), changes in liver function tests, and typical risks associated with corticosteroids such as increased blood sugar levels and susceptibility to infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am on a stable cornstarch-based treatment plan for my condition.
Select...
I agree to use effective birth control during and after the study for the specified times.
Select...
My GSDIa diagnosis was confirmed by genetic testing or liver biopsy.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 144 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 144 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Percent Change from Baseline to Week 48 in Daily Cornstarch Intake
Secondary outcome measures
Change from Baseline to Week 48 in Number of Total Daily Doses of Cornstarch in DTX401 Group Compared to Placebo Group
Change from Baseline to Week 48 in Percentage of Glucose Values in Hypoglycemic Range (<70mg/dL [3.9 mmol/L])
Change from Baseline to Week 48 in Percentage of Glucose Values in the Range of 70-120 mg/dL (3.9-6.7 mmol/L)
+3 more

Side effects data

From 2021 Phase 1 & 2 trial • 12 Patients • NCT03517085
100%
Headache
67%
Arthralgia
67%
Libido Increased
67%
Liver Function Test Increased
67%
Muscle Spasms
33%
Hepatic Enzyme Increased
33%
Dehydration
33%
Migraine
33%
Gastrointestinal Disorder
33%
Upper Respiratory Tract Congestion
33%
Blood Creatine Phosphokinase Increased
33%
Metabolic Disorder
33%
Arthropod Bite
33%
Cough
33%
Plantar Fasciitis
33%
Abdominal Wall Infection
33%
Diarrhoea
33%
Chills
33%
Pyrexia
33%
Thirst
33%
Nasopharyngitis
33%
Upper Respiratory Tract Infection
33%
Anxiety
33%
Nephrolithiasis
33%
Oropharyngeal Pain
33%
Rash
33%
Gastrointestinal Viral Infection
33%
Infection
33%
Sinobronchitis
33%
Sinusitis
33%
Hypertriglyceridaemia
33%
Hypoglycaemia
33%
Nausea
33%
Vomiting
33%
Gastroenteritis
33%
Hair Growth Abnormal
100%
80%
60%
40%
20%
0%
Study treatment Arm
DTX401 Cohort 1
DTX401 Cohort 2
DTX401 Cohort 3
DTX401 Cohort 4
Total

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: DTX401, Then PlaceboExperimental Treatment4 Interventions
Participants receive single peripheral intravenous (IV) infusion of DTX401 in solution. At week 48 participants receive single peripheral IV infusion of Placebo.
Group II: DTX401 (Japan Only)Experimental Treatment2 Interventions
Participants receive single peripheral intravenous (IV)infusion of DTX401 in solution.
Group III: Placebo, Then DTX401Placebo Group4 Interventions
Participants receive single peripheral IV infusion of Placebo. At week 48 eligible participants receive single peripheral IV infusion of DTX401 solution.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
DTX401
2018
Completed Phase 2
~20
Oral corticosteroids
2005
Completed Phase 3
~2070

Find a Location

Who is running the clinical trial?

Ultragenyx Pharmaceutical IncLead Sponsor
89 Previous Clinical Trials
179,028 Total Patients Enrolled
12 Trials studying Glycogen Storage Disease
200 Patients Enrolled for Glycogen Storage Disease
Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc
2,777 Previous Clinical Trials
8,063,434 Total Patients Enrolled
7 Trials studying Glycogen Storage Disease
131 Patients Enrolled for Glycogen Storage Disease

Media Library

DTX401 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05139316 — Phase 3
Glycogen Storage Disease Research Study Groups: Placebo, Then DTX401, DTX401 (Japan Only), DTX401, Then Placebo
Glycogen Storage Disease Clinical Trial 2023: DTX401 Highlights & Side Effects. Trial Name: NCT05139316 — Phase 3
DTX401 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05139316 — Phase 3

Frequently Asked Questions

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

What indications is DTX401 most frequently prescribed for?

"DTX401 has shown efficacy in the treatment of allergic contact dermatitis, nephrotic syndrome, and vasculitis."

Answered by AI

What is the FDA's stance on DTX401?

"DTX401 clinical trials are currently in Phase 3, which means that there is some data to support its efficacy and multiple rounds of data affirming its safety. Therefore, our team at Power has given it a score of 3 for safety."

Answered by AI

Are investigators searching for more participants at this time?

"That is correct. The clinicaltrials.gov website has information suggesting that the study is currently looking for 50 participants between 15 different sites."

Answered by AI

How many people are participating in this clinical trial?

"In order to run this experiment, a total of 50 individuals that meet the set inclusion criteria are required. The sponsor, Ultragenyx Pharmaceutical Inc, has chosen University of Texas Health Science Center at Houston in Houston, Texas and University of Michigan in Ann Arbor, Michigan as two sites for data collection."

Answered by AI

Would it be possible to get a list of North American clinics running this clinical trial?

"A few of the 15 locations where this study is enrolling patients are University of Texas Health Science Center at Houston in Houston, Texas, University of Michigan in Ann Arbor, Michigan, and Children's Hospital Colorado in Aurora, Colorado."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
University of Connecticut Health Center
Mount Sinai
Duke University
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria

How responsive is this trial?

Typically responds via
Email
Most responsive sites:
  1. University of Connecticut Health Center: < 48 hours
Average response time
  • < 2 Days
Recent research and studies
clinicaltrials.govStudy
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
2021. "A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05139316.
All > Glycogen Storage Disease
~13 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top