Rollover Study From EXG-US-01
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: EXG34217.
- Who it may be relevant to
- Registry conditions: Telomere Biology Disorders With Bone Marrow Failure. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01
Overview
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
- Biological EXG34217
Single infusion
Primary outcome measures
- 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]
- 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]
- 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]
- 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]
- 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]
Secondary outcome measures (2)
- 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]
- 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]
Eligibility criteria
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
\-
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Supportive care
Study locations
United States · 1 center
- Cincinnati Children's Hospital — Cincinnati
Identifiers
NCT: NCT05868499 · EXG-US-02