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Enrolling by Invitation

NCT Number: NCT05868499

Rollover Study From EXG-US-01

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

Enrolling by Invitation

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Key information

Conditions

Telomere Biology Disorders With Bone Marrow Failure

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

-

Treatment and study plan

EXG34217

Biological

Single infusion

Primary outcomes

  1. Number of participants with adverse events -Safety by Incidence of Treatment-Emergent

    Time frame: 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.

  2. umber of participants with a change in in physical examination

    Time frame: 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)

  3. Number of participants with a change in Electrocardiography (ECG)

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

    ECG (standard digital 12-lead in singlicate)

  4. Number of participants with a change in clinical laboratory evaluations

    Time frame: 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)

  5. Number of participants with a change of Immunogenicity

    Time frame: 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

Secondary outcomes

  1. Number of participants with a change in telomere length

    Time frame: 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

  2. Number of participants with improvement of blood counts.

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

    Blood counts: neutrophils,platelets, or hemoglobin

Sponsors and collaborators

Lead sponsor

Elixirgen Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
May 22, 2023
Registry last updated
Jul 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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