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

Elixirgen Therapeutics

Status and phase

Active, not recruiting

Phase 1

Conditions

Telomere Shortening

Bone Marrow Failure

Treatments

Biological: EXG34217

Study type

Interventional

Funder types

Industry

Identifiers

NCT04211714

EXG-US-01

Details and patient eligibility

About

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.

Full 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)

Enrollment

12 estimated patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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.