Clinical trial NCT05868499: Rollover Study From EXG-US-01
Official title: Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE1
Conditions: Telomere Biology Disorders With Bone Marrow Failure
Lead sponsor: Elixirgen Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2023-05-22
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Sequential Assignment
Primary purpose: SUPPORTIVE_CARE

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.

Eligibility criteria: 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:

-

Primary outcomes:
measure: Number of participants with adverse events -Safety by Incidence of Treatment-Emergent
timeFrame: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
measure: umber of participants with a change in in physical examination
timeFrame: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
measure: Number of participants with a change in Electrocardiography (ECG)
timeFrame: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
measure: Number of participants with a change in clinical laboratory evaluations
timeFrame: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
measure: Number of participants with a change of Immunogenicity
timeFrame: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Secondary outcomes:
measure: Number of participants with a change in telomere length
timeFrame: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
measure: Number of participants with improvement of blood counts.
timeFrame: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
Study officials:
affiliation: Cincinnati Children Hospital Medical Center
name: Kasiani Myers, MD
role: PRINCIPAL_INVESTIGATOR
References: