Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

NCT04211714 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: EXG-US-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Telomere Shortening; Bone Marrow Failure

Outcome Measures

Primary Outcomes (5)

• Number of participants with adverse events -Safety by Incidence of Treatment-Emergent Adverse Events

  Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.

  Multiple times for the duration of the study (baseline through Month 12)

• Number of participants with a change in in physical examination

  Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)

  Multiple times for the duration of the study (baseline through Month 12)

• Number of participants with a change in Electrocardiography (ECG)

  ECG (standard digital 12-lead in singlicate)

  Multiple times for the duration of the study (baseline through Month 12)

• Number of participants with a change in clinical laboratory evaluations

  Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)

  Multiple times for the duration of the study (baseline through Month 12)

• Number of participants with a change of Immunogenicity

  Change in Antibody against virus vector and transgene

  Multiple times for the duration of the study (baseline through Month 12)

Secondary Outcomes (2)

• Number of participants with a change in telomere length

  Screening, Month1,3,6 and 12

• Number of participants with improvement of blood counts.

  Multiple times for the duration of the study (baseline through Month 12)

Study Arms (1)

EXG34217

EXPERIMENTAL

single autologous CD34+ cells contacted ex vivo with EXG-001

Biological: EXG34217

Interventions

EXG34217 BIOLOGICAL

Single infusion

EXG34217

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 12 years and above
• Mild or moderate bone marrow failure defined by satisfying specific conditions.
• Diagnosis of telomere biology disorders

You may not qualify if:

• 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.

Sponsors & Collaborators

• Elixirgen Therapeutics, Inc.: lead

Study Sites (1)

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

• Myers KC, Davies SM, Lutzko C, Wahle R, Grier DD, Aubert G, Norris K, Baird DM, Koga M, Ko AC, Amano T, Amano M, Yu H, Ko MSH. Clinical Use of ZSCAN4 for Telomere Elongation in Hematopoietic Stem Cells. NEJM Evid. 2025 Mar;4(3):EVIDoa2400252. doi: 10.1056/EVIDoa2400252. Epub 2025 Feb 25.

  PMID: 39998303 DERIVED

MeSH Terms

Conditions

Bone Marrow Failure Disorders

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Kasiani Myers, MD

  Cincinnati Children Hospital Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 6, 2019
• First Posted: December 26, 2019
• Study Start: April 8, 2021
• Primary Completion (Estimated): October 8, 2026
• Study Completion (Estimated): October 8, 2027
• Last Updated: February 6, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)