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Experimental infusion aims to fix bone marrow failure in rare telomere disease

NCT ID NCT04211714

Disease control Sponsor: Elixirgen Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2021

Expected to finish

Oct 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age 12 years and above * Mild or moderate bone marrow failure defined by satisfying specific conditions. * Diagnosis of telomere biology disorders Exclusion Criteria: * Women of child bearing potential or breastfeeding. * Patients with cancer who are on active chemotherapeutic treatment. * Patients with severe bone marrow failure. * Clonal cytogenetic abnormalities associated with MDS or AML on bone marrow examination. * Uncontrolled bacterial, viral or fungal infections. * Prior allogeneic marrow or stem cell transplantation. * Patients who are not eligible for G-CSF and plerixafor dosing. * Patients who are not eligible for the apheresis. * Patients currently taking or have taken danazol and androgens within 60 days prior to Day 1. * Patients with any other clinically relevant acute or chronic diseases which could interfere with the patients' safety during the trial, expose them to undue risk, or which could interfere with study objectives. * Patients who have participated in another clinical trial with an investigational drug within the previous 30 days.

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Conditions

The condition(s) this trial relates to.

Bone Marrow Failure Disorders bone marrow failure syndrome telomere syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Bone marrow failure Telomere shortening

Contacts and locations

Locations

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.