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Clinical Specimen Collection From Pompe Disease Patients

NCT04476550 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 12

Last updated 2022-05-19

No results posted yet for this study

Summary

Clinical specimens are required from individuals with Pompe Disease to support process and analytical development for a genetically modified autologous bone marrow cell product currently in preclinical research, FTX-PD01. The intent is for this product to be investigated in a subsequent clinical trial under a future FDA IND to treat Pompe Disease. Enrolled participants provide a venous blood specimen (approximately 20mL) to be used in preclinical studies and research and development of FTX-PD01. Subjects may eventually be asked to undergo mobilized leukapheresis for bone marrow stem cell collection and their specimens will be used to further develop the FTX-PD01 cell product, including a cGMP compliant process to be applied under the future FDA IND.

Conditions

  • Pompe Disease

Interventions

DRUG

Filgrastim

5 days of daily sub-cutaneous administration of 10mcg/kg filgrastim

Sponsors & Collaborators

  • Frida Therapeutics LLC

    collaborator UNKNOWN

  • Serhat Gumrukcu, MD PhD

    lead OTHER

Principal Investigators

  • Serhat Gumrukcu, MD PhD · Seraph Research Institute

Eligibility

Min Age
3 Years
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-09-01
Primary Completion
2023-03-01
Completion
2023-05-31

Countries

  • United States

Study Locations

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Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04476550 on ClinicalTrials.gov