Back to resultsRollover Study From EXG-US-01
Primary Purpose
Telomere Biology Disorders With Bone Marrow Failure
Status
Not yet recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
EXG34217
Sponsored by
About this trial
This is an interventional supportive care trial for Telomere Biology Disorders With Bone Marrow Failure
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: -
Sites / Locations
- Cincinnati Children's Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
EXG34217
Arm Description
single autologous CD34+ cells contacted ex vivo with EXG-001
Outcomes
Primary Outcome Measures
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.
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)
Number of participants with a change in Electrocardiography (ECG)
ECG (standard digital 12-lead in singlicate)
Number of participants with a change in clinical laboratory evaluations
Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Number of participants with a change of Immunogenicity
Change in Antibody against virus vector and transgene
Secondary Outcome Measures
Number of participants with a change in telomere length
Change in telomere length in any peripheral blood cells
Number of participants with improvement of blood counts.
Blood counts: neutrophils,platelets, or hemoglobin
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05868499
Brief Title
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
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
July 1, 2023 (Anticipated)
Primary Completion Date
January 30, 2027 (Anticipated)
Study Completion Date
January 30, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Elixirgen Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Telomere Biology Disorders With Bone Marrow Failure
7. Study Design
Primary Purpose
Supportive Care
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Model Description
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
EXG34217
Arm Type
Experimental
Arm Description
single autologous CD34+ cells contacted ex vivo with EXG-001
Intervention Type
Biological
Intervention Name(s)
EXG34217
Intervention Description
Single infusion
Primary Outcome Measure Information:
Title
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent
Description
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
Time Frame
Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Title
umber of participants with a change in in physical examination
Description
Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
Time Frame
Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Title
Number of participants with a change in Electrocardiography (ECG)
Description
ECG (standard digital 12-lead in singlicate)
Time Frame
Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Title
Number of participants with a change in clinical laboratory evaluations
Description
Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Time Frame
Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Title
Number of participants with a change of Immunogenicity
Description
Change in Antibody against virus vector and transgene
Time Frame
Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Secondary Outcome Measure Information:
Title
Number of participants with a change in telomere length
Description
Change in telomere length in any peripheral blood cells
Time Frame
Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Title
Number of participants with improvement of blood counts.
Description
Blood counts: neutrophils,platelets, or hemoglobin
Time Frame
Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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:
-
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Martine A Francis
Phone
13013438894
Email
martine@mafinc.com
First Name & Middle Initial & Last Name or Official Title & Degree
Minako Koga
Phone
2026156004
Email
mkoga@kmphc.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kasiani Myers, MD
Organizational Affiliation
Cincinnati Children Hospital Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
12. IPD Sharing Statement
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Rollover Study From EXG-US-01
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