Clinical trial

A Phase I/II Study to Evaluate the Safety and Tolerability of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

ID

Name

EXG-US-01

Description

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.

Trial arms

Trial start

2021-04-08

Estimated PCD

2027-04-08

Trial end

2027-04-08

Status

Recruiting

Phase

Early phase I

Treatment

EXG34217

Single infusion

Arms:

EXG34217

Size

12

Primary endpoint

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

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

Number of participants with a change in in physical examination

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

Number of participants with a change in Electrocardiography (ECG)

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

Number of participants with a change in clinical laboratory evaluations

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

Number of participants with a change of Immunogenicity

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

Eligibility criteria

Inclusion Criteria: * Age \> 18 years. * 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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ESTIMATED'}}

Updated at

2023-05-10

1 organization

1 product

2 indications

Product

Indication

Telomere Shortening

Indication

Bone Marrow Failure

Organization

Elixirgen Therapeutics