Rollover Study From EXG-US-01

NCT05868499 | Enrolling By Invitation Phase 1 DMC FDA Drug

• 1 other identifier: EXG-US-02
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Telomere Biology Disorders With Bone Marrow Failure

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (2)

• Number of participants with a change in telomere length

  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.

  Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72

Study Arms (1)

EXG34217

EXPERIMENTAL

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

Biological: EXG34217

Interventions

EXG34217 BIOLOGICAL

Single infusion

EXG34217

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Have completed the 12-month visit of Study EXG-US-01.

Sponsors & Collaborators

• Elixirgen Therapeutics, Inc.: lead

Study Sites (1)

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Study Officials

• Kasiani Myers, MD

  Cincinnati Children Hospital Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Sequential Assignment

Study Record Dates

• First Submitted: May 8, 2023
• First Posted: May 22, 2023
• Study Start: January 1, 2024
• Primary Completion (Estimated): January 30, 2027
• Study Completion (Estimated): January 30, 2027
• Last Updated: July 12, 2024

Record last verified: 2024-07

Locations

US (1)