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

Sponsor

Elixirgen Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04211714

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Telomere Shortening Bone Marrow Failure	Biological: EXG34217	Phase 1

Detailed Description

This is a Phase I/II, open label study in up to 12 subjects with telomere biology disorders with bone marrow failure. The study is open to all participants regardless of gender or ethnicity. Subjects who are enrolled but not evaluable will be replaced.

Subjects will sign a consent form prior to any study related procedure and will complete baseline screening assessments. Subjects for this study will not require any preparative regimen such as chemotherapy or radiation.

The study will be conducted in three parts

Peripheral blood mononuclear cells (PBMNCs) collection; mobilization and apheresis,
Ex vivo cell processing
Processed cell infusion and post-infusion safety monitoring,
Follow-up (Week 2, 3,4,5, Months 1, 2,3,4,5,6,9 and 12)

Study Design

Study Type:

Interventional

Anticipated Enrollment :

12 participants

Allocation:

N/A

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Apr 8, 2021

Anticipated Primary Completion Date :

Apr 8, 2023

Anticipated Study Completion Date :

Apr 8, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: EXG34217 single autologous CD34+ cells contacted ex vivo with EXG-001	Biological: EXG34217 Single infusion

Arm

Intervention/Treatment

Experimental: EXG34217

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

Biological: EXG34217

Single infusion

Outcome Measures

Primary Outcome Measures

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 treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
Number of participants with a change in in physical examination [Multiple times for the duration of the study (baseline through Month 12)]
Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
Number of participants with a change in Electrocardiography (ECG) [Multiple times for the duration of the study (baseline through Month 12)]
ECG (standard digital 12-lead in singlicate)
Number of participants with a change in clinical laboratory evaluations [Multiple times for the duration of the study (baseline through Month 12)]
Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Number of participants with a change of Immunogenicity [Multiple times for the duration of the study (baseline through Month 12)]
Change in Antibody against virus vector and transgene

Secondary Outcome Measures

Number of participants with a change in telomere length [Screening, Month1,3,6 and 12]
Change in telomere length in any peripheral blood cells
Number of participants with improvement of blood counts. [Multiple times for the duration of the study (baseline through Month 12)]
Blood counts: neutrophils,platelets, or hemoglobin

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Cincinnati Children's Hospital	Cincinnati	Ohio	United States	45229

Sponsors and Collaborators

Elixirgen Therapeutics, Inc.

Investigators

Principal Investigator: Kasiani Myers, MD, Cincinnati Children Hospital Medical Center

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Elixirgen Therapeutics, Inc.

ClinicalTrials.gov Identifier:

NCT04211714

Other Study ID Numbers:

EXG-US-01

First Posted:

Dec 26, 2019

Last Update Posted:

Apr 25, 2022

Last Verified:

Apr 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Product Manufactured in and Exported from the U.S.:

Additional relevant MeSH terms:

Bone Marrow Failure Disorders

Pancytopenia

Study Results

No Results Posted as of Apr 25, 2022