Status:
UNKNOWN
Clinical Specimen Collection From Pompe Disease Patients
Lead Sponsor:
Serhat Gumrukcu, MD PhD
Collaborating Sponsors:
Frida Therapeutics LLC
Conditions:
Pompe Disease
Eligibility:
All Genders
3-30 years
Brief 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 preclinica...
Detailed Description
This protocol is to collect blood and HSPC specimens from individuals with Pompe Disease. The first blood draw will be done at the first study visit and if eligible, the second collection will be done...
Eligibility Criteria
Inclusion
- Male of female aged 3-30
- Documented diagnosis of Pompe Disease
- Participants who has not participated in a cell or gene therapy trial for Pompe Disease
Exclusion
- Active acute infection at screening
- Uncontrolled diabetes
- Uncontrolled hypertension
- Active DIC, bleeding or coagulopathy which cannot be corrected with minimal intervention
- Symptomatic, uncontrolled or severe intercurrent illness that would compromise the ability to tolerate blood collection or mobilized leukapheresis procedure
- Systemic chemotherapy less than or equal to 2 weeks (6 weeks for clofarabine or nitrosoureas) or radiation therapy less than or equal to 3 weeks prior to leukapheresis
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test at screening
- Any patient that in the opinion of the investigator is not medically stable to undergo the leukapheresis procedure or will not comply with the visit schedules or procedures
Key Trial Info
Start Date :
September 1 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 31 2023
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT04476550
Start Date
September 1 2022
End Date
May 31 2023
Last Update
May 19 2022
Active Locations (1)
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1
Seraph Research Institute
Toluca Lake, California, United States, 91602