Recruiting

Observational Study

Sponsor:

Serhat Gumrukcu, MD PhD

Code:

NCT04476550

Conditions

Pompe Disease

Eligibility Criteria

Sex: All

Age: 3 - 30

Healthy Volunteers: Not accepted

Interventions

Filgrastim

Study Details

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 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

Study ID

NCT04476550

Start date

Sep 1, 2022

Status verified date

May, 2022

Completion date

May 31, 2023

Anticipated

Primary completion date

Mar 1, 2023

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 3 - 30

Healthy Volunteers: Not accepted

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 Pompe Disease

Exclusion Criteria:

  • 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

Study Design

Enrollment

12 participants

Anticipated

Interventions and Outcome Measures

Interventions

Filgrastim

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

Primary outcome measure

  • Collection of blood [ Time Frame: Up to 20 days ]

Central Contacts and Locations

Central contacts

Gregory T Howell, BA Psy

424-274-3211greg@seraphmed.com

Locations

Seraph Research Institute

Recruiting

Toluca Lake, California, United States, 91602

Contacts

Anna Hurtado

424-274-3211anna@seraphmd.com

More Information

Sponsor

Serhat Gumrukcu, MD PhD

Last update posted

May 19, 2022

Last verified

May, 2022

Keywords

  • Pompe
  • Pompe Disease
  • HSPC
  • modified bone marrow stem cell

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Serhat Gumrukcu, MD PhD on 2022-05-19.