Clinical Specimen Collection From Pompe Disease Patients

Last updated: May 17, 2022
Sponsor: Serhat Gumrukcu, MD PhD
Overall Status: Active - Recruiting

Phase

N/A

Condition

Pompe Disease

Treatment

N/A

Clinical Study ID

NCT04476550
SRPH-LP-02/PoD
  • Ages 3-30
  • All Genders

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male of female aged 3-30
  • Documented diagnosis of Pompe Disease
  • Participants who has not participated in a cell or gene therapy trial for PompeDisease

Exclusion

Exclusion Criteria:

  • Active acute infection at screening
  • Uncontrolled diabetes
  • Uncontrolled hypertension
  • Active DIC, bleeding or coagulopathy which cannot be corrected with minimalintervention
  • Symptomatic, uncontrolled or severe intercurrent illness that would compromise theability to tolerate blood collection or mobilized leukapheresis procedure
  • Systemic chemotherapy less than or equal to 2 weeks (6 weeks for clofarabine ornitrosoureas) or radiation therapy less than or equal to 3 weeks prior toleukapheresis
  • Pregnant or nursing (lactating) women, where pregnancy is defined as the state of afemale after conception and until the termination of gestation, confirmed by apositive hCG laboratory test at screening
  • Any patient that in the opinion of the investigator is not medically stable to undergothe leukapheresis procedure or will not comply with the visit schedules or procedures

Study Design

Total Participants: 12
Study Start date:
September 01, 2022
Estimated Completion Date:
May 31, 2023

Study 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 via mobilized leukapheresis at the second visit. The mobilized leukapheresis procedures will follow the facility's standard operating procedures and protocol requirements for mobilized leukapheresis.

Donors will be males or females between and including the ages of 3 years and 30 years. Volunteers will provide written informed consent and meet all inclusion and exclusion criteria. Each participant can be in the study for up to 120 days (3 months).

The study will be conducted in accordance with human research for the purposes of obtaining clinical specimens for research. There is no endpoint for this study, however, data collected from this study will include, but not be limited to, gender, demographics, medical history, clinical laboratory values, and volume of the blood collected. The data will be summarized in future studies reporting results from a future clinical trial under FDA IND.

Connect with a study center

  • Seraph Research Institute

    Los Angeles, California 90067
    United States

    Site Not Available

  • Seraph Research Institute

    Toluca Lake, California 91602
    United States

    Active - Recruiting

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