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Gene Therapy Trial Report

Summary

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


NCTID NCT04211714 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Telomere Shortening, Bone Marrow Failure
Disease Ontology Term DOID:0061213
Compound Name EXG34217
Sponsor Elixirgen Therapeutics, Inc.
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant ZSCAN4
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type SeV18/TS15ΔF
Editor Type none
Dose 1 Transduced CD34+ cells

Study Record Dates


Current Stage Phase1
Submit Date 2019-12-06
Completion Date 2027-10-08
Last Update 2026-02-06

Participation Criteria


Eligible Age >=12 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates

Resources/Links