Clinical Specimen Collection From Pompe Disease Patients

Key facts

Sponsor

Serhat Gumrukcu, MD PhD

Principal Investigator

Serhat Gumrukcu, MD PhD

Seraph Research Institute

Intervention

Filgrastim(drug)

Enrollment

12 enrolled

Eligibility

3-30 years · All sexes

Timeline

2022 – 2023

Study locations (1)

• Seraph Research Institute, Toluca Lake, California, United States

Collaborators

Frida Therapeutics LLC

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Other trials for Glycogen storage disease due to acid maltase deficiency

Additional recruiting or active studies for the same condition.

• RECRUITINGNANCT07478172
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• RECRUITINGPHASE1, PHASE2NCT07282847
  A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)
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• RECRUITINGPHASE2NCT07123155
  Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
  Shionogi
• RECRUITINGPHASE4NCT06666413
  China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
  Genzyme, a Sanofi Company
• RECRUITINGNCT06795152
  Rare Glycogen Storage Diseases Natural History Study
  Duke University
• RECRUITINGPHASE1, PHASE2NCT06391736
  Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
  GeneCradle Inc
• RECRUITINGNCT06121011
  A Global Prospective Observational Registry of Patients With Pompe Disease
  Amicus Therapeutics
• ACTIVE NOT RECRUITINGEARLY PHASE1NCT06109948
  Study to Assess the Safety, Tolerability, PK and PD of ABX1100
  Aro Biotherapeutics

See all trials for Glycogen storage disease due to acid maltase deficiency →