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NCT04211714 · ClinicalTrials.gov registry record · Phase 1
Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
A Phase 1 study of Bone Marrow Failure and Telomere Shortening, sponsored by Elixirgen Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 1
- Study location
NCT04211714: Active Phase 1 study of Bone Marrow Failure and Telomere Shortening, sponsored by Elixirgen Therapeutics.
NCT04211714 is a Phase 1 study of Bone Marrow Failure and Telomere Shortening that is active but no longer recruiting, run by Elixirgen Therapeutics. The registered enrollment target is 12 participants, below the 28-participant average among 5 other Bone Marrow Failure trials with a reported enrollment target (57% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04211714, a Phase 1 study of Bone Marrow Failure and Telomere Shortening, is active but no longer recruiting, sponsored by Elixirgen Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 1
- Study location
Study 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.
Primary Outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
Conditions Studied
Interventions
- BIOLOGICAL EXG34217
Study Locations (1)
Ohio
- Cincinnati Children's Hospital - Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2021-04-08 |
| Est. Completion | 2027-10-08 |
| Phase | Phase 1 |
What the registry record for NCT04211714 still lists
NCT04211714 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 28-participant average among 5 other Bone Marrow Failure trials with a reported enrollment target (57% lower).
The record links to 2 conditions, with Bone Marrow Failure appearing as the primary indexed condition, and to 1 intervention - of which EXG34217 is the first listed.
NCT04211714 reports a single indexed study location in Ohio.
Frequently Asked Questions
What is clinical trial NCT04211714 about?
NCT04211714 is a clinical study titled "Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure". 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.
What is the current status of trial NCT04211714?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2021-04-08. Estimated completion is 2027-10-08.
What conditions does trial NCT04211714 study?
This clinical trial studies the following conditions: Bone Marrow Failure, Telomere Shortening.
What interventions are being tested in trial NCT04211714?
The interventions under investigation include: EXG34217 (BIOLOGICAL).
Who is sponsoring clinical trial NCT04211714?
This trial is sponsored by Elixirgen Therapeutics, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04211714 being conducted?
This trial has 1 study location across Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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