Clinical Specimen Collection From Pompe Disease Patients
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|ClinicalTrials.gov Identifier: NCT04476550|
Recruitment Status : Recruiting
First Posted : July 20, 2020
Last Update Posted : May 19, 2022
|Condition or disease||Intervention/treatment|
|Pompe Disease||Drug: Filgrastim|
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.
|Study Type :||Observational|
|Estimated Enrollment :||12 participants|
|Official Title:||Developing a Potential Cure for Pompe Disease: Clinical Specimen Collection From Individuals With Pompe Disease|
|Estimated Study Start Date :||September 1, 2022|
|Estimated Primary Completion Date :||March 1, 2023|
|Estimated Study Completion Date :||May 31, 2023|
- Drug: Filgrastim
5 days of daily sub-cutaneous administration of 10mcg/kg filgrastimOther Name: Granulocyte-Colony Stimulating Factor (G-CSF)
- Collection of blood [ Time Frame: Up to 20 days ]Collection of peripheral blood (up to 20ml)
- Collection of HSPCs [ Time Frame: Up to 100 days ]Collection of HSPCs through G-CSF mobilized leukapheresis
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04476550
|Contact: Gregory T Howell, BA Psyfirstname.lastname@example.org|
|United States, California|
|Seraph Research Institute||Recruiting|
|Toluca Lake, California, United States, 91602|
|Contact: Anna Hurtado 424-274-3211 email@example.com|
|Principal Investigator:||Serhat Gumrukcu, MD PhD||Seraph Research Institute|