Experimental infusion aims to fix bone marrow failure in rare telomere disease
NCT ID NCT04211714
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a single infusion of EXG34217 in 12 people aged 12 and older who have bone marrow failure due to telomere biology disorders. The main goal is to check safety and tolerability, while also looking at changes in telomere length and blood cell counts. Because it is a small Phase I/II study, it is too soon to know if the treatment works.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
EXG34217 (a biological therapy given as a single infusion)
What this could lead to
If it works, this could point toward a treatment that improves blood counts and lengthens telomeres in patients with telomere biology disorders.
What could go wrong
This is a very early, small Phase I/II trial with only 12 participants, focused on safety. It may not show clear benefit, and risks like immune reactions or lack of effectiveness are possible.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for TELOMERE BIOLOGY DISORDERS are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States