Glycogen Storage Disease Type IAPhase 3NCT05139316
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
A Phase 3 study testing DTX401 and Oral prednisolone in people with Glycogen Storage Disease Type IA, run by Ultragenyx Pharmaceutical Inc at 20 sites in 9 countries.
Trial summary
The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Glycogen Storage Disease Type IA
- What is being tested?
- DTX401 and Oral prednisolone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 52 participants
- Where
- 20 sitesin 9 countries
- Study dates
- 8 Nov 2021to 20 Feb 2026
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Documented GSDIa with confirmation by molecular testing or enzymatic activity on liver biopsy
- Currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status.
- Willing and able to complete the informed consent process and to comply with study procedures and visit schedule
- 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
Who cannot join
- Detectable pre-existing antibodies to the AAV8 capsid
- History of liver transplant, including hepatocyte cell therapy/ transplant
- History of liver disease
- Presence of liver adenoma >5 cm in size
- Presence of liver adenoma >3 cm and ≤5 cm in size that has a documented annual growth rate of ≥0.5 cm per year
- Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN), total bilirubin >1.5 × ULN, alkaline phosphatase >2.5 × ULN
Show all 11Show fewer
- Non-fasting triglycerides ≥1000 mg/dL
- Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study.
- Current or previous participation in another gene transfer study
- History of illicit drug use within 60 days prior to screening or positive results from an 8-panel urine drug screen during the Screening Period completed at 2 time points at least 4 weeks apart
- Note: additional inclusion/exclusion criteria may apply, per protocol
Trial locations and contacts
20 sites in 9 countries.
20 sites in 9 countries
United States9 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Cleveland Clinic
Cleveland, Ohio, 44195
Duke University
Durham, North Carolina, 27710
University of Connecticut Health Center
Farmington, Connecticut, 06030
University of Texas
Houston, Texas, 77030
Children's Hospital of Orange County
Orange, California, 92868
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Primary Children's Hospital
Salt Lake City, Utah, 84132
Mount Sinai
The Bronx, New York, 10467
Japan3 sites
Kumamoto University Hospital
Kumamoto
Osaka City General Hospital
Osaka
Fujita Health University Hospital
Toyoake
Italy2 sites
Istituto Giannina Gaslini
Genova, Linguria, 16147
University of Naples
Naples, 80131
Brazil1 site
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903
Spain1 site
Hospital Clinico Universitario de Santiago
Santiago de Compostela, A Coruna, 15706
About this study
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia
The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control.
Read the detailed description
Study DTX401-CL301 is a phase 3 study to determine the efficacy and confirm the safety of DTX401 in patients 8 years and older with glycogen storage disease type Ia (GSDIa).
Participants were randomized 1:1 to DTX401 or placebo group and followed closely for 48 weeks. At week 48 eligible participants crossed over and received DTX401 if they had previously received placebo or placebo if they had previously received DTX401 and were followed closely for an additional 96 weeks. After completion of week 144 or early withdrawal, participants were offered enrollment into a Disease Monitoring Program (DMP) where they will be followed for at least 10 years post DTX401 infusion.
In Japan, there was a single open label study arm and all participants were treated with DTX401. At week 48, Japanese participants were offered enrollment into a Disease Monitoring Program (DMP) where they will be followed for at least 10 years post DTX401 infusion.
- Conditions
- Glycogen Storage Disease Type IA
- Being tested
- DTX401, Placebo, Oral prednisolone, Placebo for oral prednisolone
- Keywords
- glycogen storage disorder Ia, AAV, gene therapy, von Gierke disease, glucose metabolism disorder, GSDIa, GSD1
- Medical subject headings
- Hepatorenal form of glycogen storage disease, Glycogen Storage Disease Type I, Glucose Metabolism Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DTX401Genetic
Also called pariglasgene brecaparvovec
nonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8)
PlaceboOther
Normal Saline infusion
Oral prednisoloneDrug
Participants who receive DTX401 solution will receive oral prednisolone
Placebo for oral prednisoloneDrug
Participants who receive placebo will receive placebo oral prednisolone to maintain the study blind
| Study group | Role | What they receive |
|---|---|---|
| Placebo Followed by DTX401 | Experimental | Participants received a single peripheral intravenous (IV) infusion of placebo on Day 1 along with a placebo-to-match (PTM) of the prophylactic steroid regimen of prednisolone. At Week 48, participants received a single peripheral IV infusion of 1.0 × 10^13 genome copies per kilogram [GC/kg]) of DTX401 along with a prophylactic steroid regimen of prednisolone. Receives: Genetic: DTX401; Other: Placebo; Drug: Oral prednisolone; Drug: Placebo for oral prednisolone |
| DTX401 Followed by Placebo | Placebo comparator | Participants received a single peripheral IV infusion of 1.0 × 10^13 GC/kg of DTX401 on Day 1, along with a prophylactic steroid regimen of prednisolone. At Week 48, participants received a single peripheral IV infusion of placebo with a PTM of the prophylactic steroid regimen of prednisolone. Receives: Genetic: DTX401; Other: Placebo; Drug: Oral prednisolone; Drug: Placebo for oral prednisolone |
| DTX401 (Japan Only) | Experimental | Participants received a single peripheral IV infusion of 1.0 × 10^13 GC/kg of DTX401 on Day 1, along with a prophylactic steroid regimen of prednisolone. Receives: Genetic: DTX401; Drug: Oral prednisolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Main Study: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams)
The least square (LS) mean and standard error (SE) are based on mixed-model for repeated measures (MMRM) analysis.
Measured: Baseline, Weeks 24, 30, 36, 42 and 48
Japan Cohort: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams)
Measured: Baseline, Weeks 24, 30, 36, 42 and 48
Secondary outcomes (12)
Main Study: Change From Baseline to Week 48 in Number of Total Daily Doses of Cornstarch
The LS mean and SE are based on MMRM analysis.
Measured: Baseline, Weeks 24, 30, 36, 42 and 48
Japan Cohort: Change From Baseline to Week 48 in Number of Total Daily Doses of Cornstarch
Measured: Baseline, Weeks 24, 30, 36, 42 and 48
Main Study: Change From Baseline to Week 48 in Percentage of Glucose Values in Hypoglycemic Range (Less Than (<) 70 Milligrams Per Deciliter (mg/dL) [3.9 Millimoles Per Liter (mmol/L)])
The LS mean and SE are based on MMRM analysis.
Measured: Baseline, Weeks 24, 30, 36, 42 and 48
Japan Cohort: Change From Baseline to Week 48 in Percentage of Glucose Values in Hypoglycemic Range (< 70 mg/dL [3.9 mmol/L])
Measured: Baseline, Weeks 24, 30, 36, 42 and 48
Main Study: Patient Global Impression of Change (PGIC) Assessment Score at Week 48
The PGIC captures the participants' perspective on changes in their glycogen storage disease type Ia (GSDIa) since the start of the study using a 7-point response scale ranging from much improved (+ 3) to much worse (- 3). The total score ranges from + 3 to - 3, with a higher score indicating best outcome.
Measured: Week 48
Japan Cohort: PGIC Assessment Score at Week 48
The PGIC captures the participants' perspective on changes in their GSDIa since the start of the study using a 7-point response scale ranging from much improved (+ 3) to much worse (- 3). The total score ranges from + 3 to - 3, with a higher score indicating best outcome.
Measured: Week 48
Main Study: Change From Baseline to Week 48 in Time to Hypoglycemia (< 54 mg/dL [3.0 mmol/L]) During the Controlled Fasting Challenge (CFC)
The LS mean and SE are based on MMRM analysis.
Measured: Baseline, Weeks 36 and 48
Japan Cohort: Change From Baseline to Week 48 in Time to Hypoglycemia (< 54 mg/dL [3.0 mmol/L]) During the CFC
Measured: Baseline, Weeks 36 and 48
Main Study: 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)
The LS mean and SE are based on MMRM analysis.
Measured: Baseline, Weeks 24, 30, 36, 42 and 48
Japan Cohort: 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)
Measured: Baseline, Weeks 24, 30, 36, 42 and 48
Main Study: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs), Serious TEAEs, Related TEAEs, Fatal AEs, and Discontinuation From Study or Investigational Product Due to AEs
Adverse event (AE) is defined as any untoward medical occurrence associated with use of a drug in humans, whether or not considered drug related. TEAE is defined as any AE not present prior to initiation of drug treatment or any AE already present that worsens in either intensity or frequency following exposure to drug treatment. SAE is an AE that meets any of following criteria in view of either Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; disability/incapacity; congenital anomaly/birth defect not present at screening; other important medical events. AESIs are events of scientific or medical interest specific to drug treatment requiring further investigation and close monitoring to further characterize them. Hepatic reactions (vector-induced hepatic effects) were AESIs for this study and were defined as clinically significant increases in aminotransferase levels (AST/ALT) in the judgment of Investigator.
Measured: PEAP Arms - TEAEs with onset from Day 1 to Week 48; Crossover and Follow-up - Placebo to DTX401 Arm: TEAEs with onset after Week 48 dosing with DTX401 to Week 144; Crossover and Follow-up DTX401 Arm: TEAEs from Week 48 to Week 144
Japan Cohort: Number of Participants With TEAEs, AESIs, Serious TEAEs, Related TEAEs, Fatal AEs, and Discontinuation From Study or Investigational Product Due to AEs
AE is defined as any untoward medical occurrence associated with use of a drug in humans, whether or not considered drug related. TEAE is defined as any AE not present prior to initiation of drug treatment or any AE already present that worsens in either intensity or frequency following exposure to drug treatment. SAE is an AE that meets any of following criteria in view of either Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; disability/incapacity; congenital anomaly/birth defect not present at screening; other important medical events. AESIs are events of scientific or medical interest specific to drug treatment requiring further investigation and close monitoring to further characterize them. Hepatic reactions were considered to be important identified risk for this study and were defined as clinically significant increases in aminotransferase levels (AST/ALT) in the judgment of Investigator.
Measured: Day 1 to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2021
- Study start8 Nov 2021
- Primary completion20 Feb 2024
- Study completion20 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Glycogen Storage Disease Type IAStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05139316
- Sponsor's protocol ID
- DTX401-CL301
- Other IDs
- 2020-004184-12, 2023-508750-25-00, 2023
- Status last verified
- Sep 2026
- First submitted
- 14 Jul 2021
- Last updated
- 14 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia
- ClinicalTrials.gov ID
- NCT05139316
- Acronym / sponsor ID
- DTX401-CL301
- Conditions
- Glycogen Storage Disease Type IA
- Keywords
- glycogen storage disorder Ia, AAV, gene therapy, von Gierke disease, glucose metabolism disorder, GSDIa, GSD1
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 8 Nov 2021 (actual)
- Primary completion
- 20 Feb 2024 (actual)
- Study completion
- 20 Feb 2026 (actual)
- First posted
- 1 Dec 2021 (actual)
- Results
- Posted 14 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control.
Detailed description
Study DTX401-CL301 is a phase 3 study to determine the efficacy and confirm the safety of DTX401 in patients 8 years and older with glycogen storage disease type Ia (GSDIa).
Participants were randomized 1:1 to DTX401 or placebo group and followed closely for 48 weeks. At week 48 eligible participants crossed over and received DTX401 if they had previously received placebo or placebo if they had previously received DTX401 and were followed closely for an additional 96 weeks. After completion of week 144 or early withdrawal, participants were offered enrollment into a Disease Monitoring Program (DMP) where they will be followed for at least 10 years post DTX401 infusion.
In Japan, there was a single open label study arm and all participants were treated with DTX401. At week 48, Japanese participants were offered enrollment into a Disease Monitoring Program (DMP) where they will be followed for at least 10 years post DTX401 infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo Followed by DTX401 | Experimental | Genetic: DTX401, Other: Placebo, Drug: Oral prednisolone, Drug: Placebo for oral prednisoloneParticipants received a single peripheral intravenous (IV) infusion of placebo on Day 1 along with a placebo-to-match (PTM) of the prophylactic steroid regimen of prednisolone. At Week 48, participants received a single peripheral IV infusion of 1.0 × 10^13 genome copies per kilogram [GC/kg]) of DTX401 along with a prophylactic steroid regimen of prednisolone. |
| DTX401 Followed by Placebo | Placebo comparator | Genetic: DTX401, Other: Placebo, Drug: Oral prednisolone, Drug: Placebo for oral prednisoloneParticipants received a single peripheral IV infusion of 1.0 × 10^13 GC/kg of DTX401 on Day 1, along with a prophylactic steroid regimen of prednisolone. At Week 48, participants received a single peripheral IV infusion of placebo with a PTM of the prophylactic steroid regimen of prednisolone. |
| DTX401 (Japan Only) | Experimental | Genetic: DTX401, Drug: Oral prednisoloneParticipants received a single peripheral IV infusion of 1.0 × 10^13 GC/kg of DTX401 on Day 1, along with a prophylactic steroid regimen of prednisolone. |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Documented GSDIa with confirmation by molecular testing or enzymatic activity on liver biopsy
- Currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status.
- Willing and able to complete the informed consent process and to comply with study procedures and visit schedule
- 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
Key exclusion criteria (11 listed)
- Detectable pre-existing antibodies to the AAV8 capsid
- History of liver transplant, including hepatocyte cell therapy/ transplant
- History of liver disease
- Presence of liver adenoma >5 cm in size
- Presence of liver adenoma >3 cm and ≤5 cm in size that has a documented annual growth rate of ≥0.5 cm per year
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DTX401Also: pariglasgene brecaparvovecnonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8) | Genetic | DTX401 (Japan Only), DTX401 Followed by Placebo, Placebo Followed by DTX401 |
| PlaceboNormal Saline infusion | Other | DTX401 Followed by Placebo, Placebo Followed by DTX401 |
| Oral prednisoloneParticipants who receive DTX401 solution will receive oral prednisolone | Drug | DTX401 (Japan Only), DTX401 Followed by Placebo, Placebo Followed by DTX401 |
| Placebo for oral prednisoloneParticipants who receive placebo will receive placebo oral prednisolone to maintain the study blind | Drug | DTX401 Followed by Placebo, Placebo Followed by DTX401 |
Primary outcome
Main Study: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams)
Time frame: Baseline, Weeks 24, 30, 36, 42 and 48
The least square (LS) mean and standard error (SE) are based on mixed-model for repeated measures (MMRM) analysis.
Japan Cohort: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams)
Time frame: Baseline, Weeks 24, 30, 36, 42 and 48
12 secondary outcomes
Main Study: Change From Baseline to Week 48 in Number of Total Daily Doses of Cornstarch
Time frame: Baseline, Weeks 24, 30, 36, 42 and 48
Japan Cohort: Change From Baseline to Week 48 in Number of Total Daily Doses of Cornstarch
Time frame: Baseline, Weeks 24, 30, 36, 42 and 48
Main Study: Change From Baseline to Week 48 in Percentage of Glucose Values in Hypoglycemic Range (Less Than (<) 70 Milligrams Per Deciliter (mg/dL) [3.9 Millimoles Per Liter (mmol/L)])
Time frame: Baseline, Weeks 24, 30, 36, 42 and 48
Japan Cohort: Change From Baseline to Week 48 in Percentage of Glucose Values in Hypoglycemic Range (< 70 mg/dL [3.9 mmol/L])
Time frame: Baseline, Weeks 24, 30, 36, 42 and 48
Main Study: Patient Global Impression of Change (PGIC) Assessment Score at Week 48
Time frame: Week 48
Japan Cohort: PGIC Assessment Score at Week 48
Time frame: Week 48
Main Study: Change From Baseline to Week 48 in Time to Hypoglycemia (< 54 mg/dL [3.0 mmol/L]) During the Controlled Fasting Challenge (CFC)
Time frame: Baseline, Weeks 36 and 48
Japan Cohort: Change From Baseline to Week 48 in Time to Hypoglycemia (< 54 mg/dL [3.0 mmol/L]) During the CFC
Time frame: Baseline, Weeks 36 and 48
Main Study: 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)
Time frame: Baseline, Weeks 24, 30, 36, 42 and 48
Japan Cohort: 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)
Time frame: Baseline, Weeks 24, 30, 36, 42 and 48
Main Study: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs), Serious TEAEs, Related TEAEs, Fatal AEs, and Discontinuation From Study or Investigational Product Due to AEs
Time frame: PEAP Arms - TEAEs with onset from Day 1 to Week 48; Crossover and Follow-up - Placebo to DTX401 Arm: TEAEs with onset after Week 48 dosing with DTX401 to Week 144; Crossover and Follow-up DTX401 Arm: TEAEs from Week 48 to Week 144
Japan Cohort: Number of Participants With TEAEs, AESIs, Serious TEAEs, Related TEAEs, Fatal AEs, and Discontinuation From Study or Investigational Product Due to AEs
Time frame: Day 1 to Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Ultragenyx Patient Advocacy/GSDIA Disease InformationLink provided by the sponsor.
- Ultragenyx Transparency CommitmentLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Ultragenyx Pharmaceutical Inc
20 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing DTX401 and Oral prednisolone in people with Glycogen Storage Disease Type IA, run by Ultragenyx Pharmaceutical Inc at 20 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, Japan, Italy, Brazil, Canada and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Main Study: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams) (time frame: Baseline, Weeks 24, 30, 36, 42 and 48). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.