GSDIa Disease Monitoring Program

Conditions

Glycogen storage disease type Ia (GSDIa)

Brief summary

Incidence and severity of SAEs assessed as related to DTX401 by the Investigator, Incidence, relationship, severity, and seriousness of AESIs for AAV therapies (vector-induced hepatic effects [eg, increased aminotransferase levels], malignancy [new or worsening of pre-existing malignancies], thrombotic microangiopathy, dorsal root ganglion/peripheral nerve effects, and any new potential risks of AAV therapies identified over time), Incidence and outcomes of pregnancy in patients treated with DTX401 or patient’s partner, Incidence and severity of SAEs of infusion-related reactions including hypersensitivitya, Incidence and severity of SAEs assessed by the Investigator as related to concomitant immunomodulatory therapies (eg, steroids) including adrenal insufficiency and iatrogenic Cushing’s syndromea

Detailed description

Nutritional Assessment;Clinical/CGM/Laboratory/Imaging; Major Clinical Events (MCEs); Patient Experience Clinical Interview; HEOR:

Related papers

No related papers found yet.

Interventions

DRUGPariglasgene brecaparvovec

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence and severity of SAEs assessed as related to DTX401 by the Investigator, Incidence, relationship, severity, and seriousness of AESIs for AAV therapies (vector-induced hepatic effects [eg, increased aminotransferase levels], malignancy [new or worsening of pre-existing malignancies], thrombotic microangiopathy, dorsal root ganglion/peripheral nerve effects, and any new potential risks of AAV therapies identified over time), Incidence and outcomes of pregnancy in patients treated with DTX401 or patient’s partner, Incidence and severity of SAEs of infusion-related reactions including hypersensitivitya, Incidence and severity of SAEs assessed by the Investigator as related to concomitant immunomodulatory therapies (eg, steroids) including adrenal insufficiency and iatrogenic Cushing’s syndromea	—

Measure

Time frame

Incidence and severity of SAEs assessed as related to DTX401 by the Investigator, Incidence, relationship, severity, and seriousness of AESIs for AAV therapies (vector-induced hepatic effects [eg, increased aminotransferase levels], malignancy [new or worsening of pre-existing malignancies], thrombotic microangiopathy, dorsal root ganglion/peripheral nerve effects, and any new potential risks of AAV therapies identified over time), Incidence and outcomes of pregnancy in patients treated with DTX401 or patient’s partner, Incidence and severity of SAEs of infusion-related reactions including hypersensitivitya, Incidence and severity of SAEs assessed by the Investigator as related to concomitant immunomodulatory therapies (eg, steroids) including adrenal insufficiency and iatrogenic Cushing’s syndromea

—

Secondary

Measure	Time frame
Nutritional Assessment;Clinical/CGM/Laboratory/Imaging; Major Clinical Events (MCEs); Patient Experience Clinical Interview; HEOR:	—

Countries

Denmark, Germany, Italy, Netherlands, Spain

Outcome results

None listed