A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adenoassociated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia.

Conditions

Glycogen Storage Disease Type Ia (GSDIa)

Brief summary

"Percent change from Baseline to Week 48 in daily cornstarch intake for the DTX401 Group compared with the Placebo Group."

Detailed description

"1. Change from Baseline to Week 48 in number of total daily doses of cornstarch 2. Change from Baseline to Week 48 in the percentage of glucose values in hypoglycemic range (<70 mg/dL [3.9 mmol/L]), assessed for noninferiority; if non-inferiority is established, the endpoint will be tested for superiority. 3. PGIC assessment score at Week 48. ", "4. Change from Baseline to Week 48 in time to hypoglycemia (<54 mg/dL [3.0 mmol/L]) during the CFC. 5. 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], assessed for noninferiority; if non-inferiority is established, the endpoint will be tested for superiority.", 6. Incidence, severity, and relationship to investigational product of TEAEs, TEAEs of special interest, serious TEAEs, related TEAEs, discontinuations from study or investigational product due to AEs, and fatal AEs.

Related papers

No related papers found yet.

Papers published before 2008-02-04 may not appear yet. Historical indexing is in progress.

Interventions

DRUG10 mg

DRUGPrednisolon STADA® 5 mg Tabletten

DRUGPrednisolone placebo hard capsule

DRUGPrednisolone Placebo hard capsule

DRUG5mg

DRUGPrednisolon STADA® 10 mg Tabletten

DRUGPariglasgene brecaparvovec

DRUGNormal Saline

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
"Percent change from Baseline to Week 48 in daily cornstarch intake for the DTX401 Group compared with the Placebo Group."	—

Secondary

Measure	Time frame
"1. Change from Baseline to Week 48 in number of total daily doses of cornstarch 2. Change from Baseline to Week 48 in the percentage of glucose values in hypoglycemic range (<70 mg/dL [3.9 mmol/L]), assessed for noninferiority; if non-inferiority is established, the endpoint will be tested for superiority. 3. PGIC assessment score at Week 48. ", "4. Change from Baseline to Week 48 in time to hypoglycemia (<54 mg/dL [3.0 mmol/L]) during the CFC. 5. 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], assessed for noninferiority; if non-inferiority is established, the endpoint will be tested for superiority.", 6. Incidence, severity, and relationship to investigational product of TEAEs, TEAEs of special interest, serious TEAEs, related TEAEs, discontinuations from study or investigational product due to AEs, and fatal AEs.	—

Measure

Time frame

"1. Change from Baseline to Week 48 in number of total daily doses of cornstarch 2. Change from Baseline to Week 48 in the percentage of glucose values in hypoglycemic range (<70 mg/dL [3.9 mmol/L]), assessed for noninferiority; if non-inferiority is established, the endpoint will be tested for superiority. 3. PGIC assessment score at Week 48. ", "4. Change from Baseline to Week 48 in time to hypoglycemia (<54 mg/dL [3.0 mmol/L]) during the CFC. 5. 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], assessed for noninferiority; if non-inferiority is established, the endpoint will be tested for superiority.", 6. Incidence, severity, and relationship to investigational product of TEAEs, TEAEs of special interest, serious TEAEs, related TEAEs, discontinuations from study or investigational product due to AEs, and fatal AEs.

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Countries

Denmark, Germany, Italy, Netherlands, Spain

Outcome results

None listed