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

Bone Marrow Failure Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04211714
• Other study ID #: EXG-US-01
• Status: Recruiting
• Phase: Phase 1
• Start date: April 8, 2021
• Est. completion date: April 8, 2027

Study information

• Verified date: May 2023
• Source: Elixirgen Therapeutics, Inc.
• Contact: Martine Francis
• Phone: 301-343-8894
• Email: martine[@]mafinc.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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)

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: April 8, 2027
• Est. primary completion date: April 8, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Bone Marrow Failure
• Bone Marrow Failure Disorders
• Pancytopenia
• Telomere Shortening

Intervention

Biological:
• EXG34217
Single infusion

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital	Cincinnati	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Elixirgen Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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	Number of participants with a change in telomere length	Change in telomere length in any peripheral blood cells	Screening, Month1,3,6 and 12
Secondary	Number of participants with improvement of blood counts.	Blood counts: neutrophils,platelets, or hemoglobin	Multiple times for the duration of the study (baseline through Month 12)