Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
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- STATUS
- Recruiting
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- End date
- Apr 8, 2024
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- participants needed
- 12
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- sponsor
- Elixirgen Therapeutics, Inc.
Summary
This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.
Description
This is a Phase I/II, open label study in up to 12 subjects with telomere biology disorders with bone marrow failure. The study is open to all participants regardless of gender or ethnicity. Subjects who are enrolled but not evaluable will be replaced.
Subjects will sign a consent form prior to any study related procedure and will complete baseline screening assessments. Subjects for this study will not require any preparative regimen such as chemotherapy or radiation.
The study will be conducted in three parts
- Peripheral blood mononuclear cells (PBMNCs) collection; mobilization and apheresis,
- Ex vivo cell processing
- Processed cell infusion and post-infusion safety monitoring,
- Follow-up (Week 2, 3,4,5, Months 1, 2,3,4,5,6,9 and 12)
Details
| Condition | Telomere Shortening, Bone Marrow Failure |
|---|---|
| Treatment | EXG34217 |
| Clinical Study Identifier | NCT04211714 |
| Sponsor | Elixirgen Therapeutics, Inc. |
| Last Modified on | 12 May 2022 |
How to participate?
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn moreIf you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Learn moreComplete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
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