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

Status

Enrolling by invitation

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05868499

Enrollment

Registered

2023-05-22

Start date

2024-01-01

Completion date

2027-01-30

Last updated

2024-07-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Telomere Biology Disorders With Bone Marrow Failure

Brief summary

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

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.

Interventions

BIOLOGICALEXG34217

Single infusion

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

SUPPORTIVE_CARE

Masking

NONE

Intervention model description

Sequential Assignment

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
umber of participants with a change in in physical examination	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	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 clinical laboratory evaluations	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Number of participants with a change of Immunogenicity	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	Change in Antibody against virus vector and transgene
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	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 Electrocardiography (ECG)	Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72	ECG (standard digital 12-lead in singlicate)

Secondary

Measure	Time frame	Description
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	Blood counts: neutrophils,platelets, or hemoglobin
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	Change in telomere length in any peripheral blood cells

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026