Trial · NCT05139316
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 20
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Main Study: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams) | 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. |
| Primary | Japan Cohort: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams) | Baseline, Weeks 24, 30, 36, 42 and 48 | |
| Secondary | Main Study: Change From Baseline to Week 48 in Number of Total Daily Doses of Cornstarch | Baseline, Weeks 24, 30, 36, 42 and 48 | The LS mean and SE are based on MMRM analysis. |
| Secondary | Japan Cohort: Change From Baseline to Week 48 in Number of Total Daily Doses of Cornstarch | Baseline, Weeks 24, 30, 36, 42 and 48 | |
| Secondary | 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)]) | Baseline, Weeks 24, 30, 36, 42 and 48 | The LS mean and SE are based on MMRM analysis. |
| Secondary | Japan Cohort: Change From Baseline to Week 48 in Percentage of Glucose Values in Hypoglycemic Range (< 70 mg/dL [3.9 mmol/L]) | Baseline, Weeks 24, 30, 36, 42 and 48 | |
| Secondary | Main Study: Patient Global Impression of Change (PGIC) Assessment Score at Week 48 | 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. |
| Secondary | Japan Cohort: PGIC Assessment Score at Week 48 | 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. |
| Secondary | 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) | Baseline, Weeks 36 and 48 | The LS mean and SE are based on MMRM analysis. |
| Secondary | Japan Cohort: Change From Baseline to Week 48 in Time to Hypoglycemia (< 54 mg/dL [3.0 mmol/L]) During the CFC | Baseline, Weeks 36 and 48 | |
| Secondary | 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) | Baseline, Weeks 24, 30, 36, 42 and 48 | The LS mean and SE are based on MMRM analysis. |
| Secondary | 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) | Baseline, Weeks 24, 30, 36, 42 and 48 | |
| Secondary | 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 | 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 | 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. |
| Secondary | Japan Cohort: Number of Participants With TEAEs, AESIs, Serious TEAEs, Related TEAEs, Fatal AEs, and Discontinuation From Study or Investigational Product Due to AEs | Day 1 to Week 48 | 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. |
| Other | Main Study: Percent Change From Baseline to Week 96 in Daily Cornstarch Intake (Grams) | Baseline, Week 96 | |
| Other | Main Study: Change From Baseline to Week 96 in Number of Total Daily Doses of Cornstarch | Baseline, Week 96 | |
| Other | Main Study: Change From Baseline to Week 96 in Percentage of Glucose Values in Hypoglycemic Range (Less Than (<) 70 Milligrams Per Deciliter (mg/dL) [3.9 Millimoles Per Liter (mmol/L)]) | Baseline, Week 96 | |
| Other | Main Study: Patient Global Impression of Change (PGIC) Assessment Score at Week 96 | Week 96 | 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. |
| Other | Main Study: Change From Baseline to Week 96 in Time to Hypoglycemia (< 54 mg/dL [3.0 mmol/L]) During the Controlled Fasting Challenge (CFC) | Baseline, Week 96 | |
| Other | Main Study: Change From Baseline to Week 96 in Percentage of Glucose Values in the Range of 70-120 mg/dL (3.9- 6.7 mmol/L) | Baseline, Week 96 |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
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