Rollover Study From EXG-US-01

Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01

This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).

Study Overview

• Status: Not yet recruiting
• Conditions: Telomere Biology Disorders With Bone Marrow Failure
• Intervention / Treatment: Biological: EXG34217

Detailed Description

Patients who received EXG34217 treatment and had at least one follow-up visit (Month 1, 3, 6, or 12) in Study EXG-US-01 will be eligible for this rollover study. Patient will sign a consent form prior to any study related procedure. This study is to add additional follow-up assessments up to 6 years after EXG34217 treatment. This study does not have additional intervention. Additional visits will be every 6 months in the first 3 years and once a year for two years.

• Study Type: Interventional
• Enrollment (Anticipated): 12
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Minako Koga; Phone Number: 2026156004; Email: mkoga@kmphc.com
• Study Contact Backup: Name: Martine A Francis; Phone Number: 13013438894; Email: martine@mafinc.com

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Signed an Institutional Review Board (IRB) approved informed consent document indicating that they understand the purpose of, and procedures required by the study and are willing to participate in the study and comply with all study procedures and restrictions. Informed consent must be obtained from the subject prior to initiating procedures.
2. Have completed the 12-month visit of Study EXG-US-01.

Exclusion Criteria:

-

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent	Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
umber 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)	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Number of participants with a change in Electrocardiography (ECG)	ECG (standard digital 12-lead in singlicate)	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Number of participants with a change in clinical laboratory evaluations	Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Number of participants with a change of Immunogenicity	Change in Antibody against virus vector and transgene	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with a change in telomere length	Change in telomere length in any peripheral blood cells	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Number of participants with improvement of blood counts.	Blood counts: neutrophils,platelets, or hemoglobin	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72

Collaborators and Investigators

• Sponsor: Elixirgen Therapeutics, Inc.
• Investigators: Principal Investigator: Kasiani Myers, MD, Cincinnati Children Hospital Medical Center

Study record dates

Study Major Dates

• Study Start (Anticipated): July 1, 2023
• Primary Completion (Anticipated): January 30, 2027
• Study Completion (Anticipated): January 30, 2027

Study Registration Dates

• First Submitted: May 8, 2023
• First Submitted That Met QC Criteria: May 18, 2023
• First Posted (Actual): May 22, 2023

Study Record Updates

• Last Update Posted (Actual): May 22, 2023
• Last Update Submitted That Met QC Criteria: May 18, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Pancytopenia
• Other Study ID Numbers: EXG-US-02

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No