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Unknown

EXG34217 for Bone Marrow Failure

Phase 1
Recruiting
Led By Kasiani Myers, MD
Research Sponsored by Elixirgen Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Mild or moderate bone marrow failure defined by satisfying specific conditions
Diagnosis of telomere biology disorders
Timeline
Screening 3 weeks
Treatment Varies
Follow Up screening, month1,3,6 and 12
Awards & highlights

Study Summary

This trial will study a new drug to treat bone marrow failure in patients with telomere biology disorders.

Who is the study for?
This trial is for adults over 18 with mild or moderate bone marrow failure due to telomere biology disorders. It's not for those with severe bone marrow failure, certain genetic abnormalities, uncontrolled infections, previous transplants, or who can't undergo specific treatments. Pregnant or breastfeeding women and patients on recent trials or cancer treatment are also excluded.Check my eligibility
What is being tested?
The study tests the safety and effects of a drug called EXG34217 in patients with bone marrow failure linked to short telomeres. As an early-stage (Phase I/II) trial at a single center, it's designed to see how well participants tolerate this medication.See study design
What are the potential side effects?
Since EXG34217 is being studied primarily for its safety and tolerability, potential side effects aren't listed but may include typical reactions related to new medications such as allergic responses, gastrointestinal issues, fatigue, blood count changes among others.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My bone marrow is not working well, but it's not severe.
Select...
I have been diagnosed with a telomere biology disorder.
Select...
I am older than 18 years.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~screening, month1,3,6 and 12
This trial's timeline: 3 weeks for screening, Varies for treatment, and screening, month1,3,6 and 12 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with a change in Electrocardiography (ECG)
Number of participants with a change in clinical laboratory evaluations
Number of participants with a change in in physical examination
+2 more
Secondary outcome measures
Number of participants with a change in telomere length
Number of participants with improvement of blood counts.

Trial Design

1Treatment groups
Experimental Treatment
Group I: EXG34217Experimental Treatment1 Intervention
single autologous CD34+ cells contacted ex vivo with EXG-001

Find a Location

Who is running the clinical trial?

Elixirgen Therapeutics, Inc.Lead Sponsor
2 Previous Clinical Trials
52 Total Patients Enrolled
Kasiani Myers, MDPrincipal InvestigatorCincinnati Children Hospital Medical Center
2 Previous Clinical Trials
52 Total Patients Enrolled

Media Library

EXG34217 (Unknown) Clinical Trial Eligibility Overview. Trial Name: NCT04211714 — Phase 1
Telomere Shortening Research Study Groups: EXG34217
Telomere Shortening Clinical Trial 2023: EXG34217 Highlights & Side Effects. Trial Name: NCT04211714 — Phase 1
EXG34217 (Unknown) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04211714 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any vacancies available to participants in this clinical trial?

"Affirmative. The data posted on clinicaltrials.gov suggests that recruitment for this medical study is still ongoing, with the initial posting of April 8th 2021 and its most recent update being April 22nd 2022. A total of 12 patients are required from one site to complete the trial."

Answered by AI

What is the total number of participants involved in this clinical experiment?

"Affirmative. Clinicaltrials.gov verifies that this research project, initially posted on April 8th 2021, is still underway and looking to recruit 12 participants from one medical centre."

Answered by AI

Has EXG34217 been greenlighted by the FDA?

"Due to this being a Phase 1 trial with limited data about efficacy and safety, EXG34217 only received a score of 1."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
2021. "Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04211714.
~6 spots leftby Apr 2027
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