SYF505019ed4733496b91b6030a605da1d5Registry ID: 2023-508000-37-01|RecruitingPhase 4
GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE
RigshospitaletCTIS (EMA)
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interventional
N/A
1 country
1
The objective of this trial it to characterize how muscle glycogen is affected by enzyme replacement therapy in treatment naïve patients with Pompe Disease.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
Not reported
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
November 14, 2023
Actual (reported)Change in glycogen concentration of the hamstring muscles between baseline and 12-month follow-up
Change in glycogen concentration of the calf, hamstring, anterior thigh, and lumbar muscles over time – both within the first treatment cycle, and at 3, 6 and 12-months follow-up
Change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 3, 6 and 12-months follow-up.
Correlations between baseline glycogen levels and change in FVC between baseline and 12-months follow-up.
Correlations between baseline glycogen levels and change in 6MWT between baseline and 12-months follow-up.
Correlations between baseline glycogen levels and change in fat-fraction of the calf, hamstring, anterior thigh, and lumbar muscles from baseline to 12-months follow-up.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Rigshospitalet
Copenhagen Oe, 2100, Denmark
Mads Stemmerik
First Submitted
November 14, 2023
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Source: European Medicines Agency (EMA), Clinical Trials Information System (CTIS). Original record and public documents are available on CTIS.
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