Telomere Biology Disorders With Bone Marrow FailurePhase 1NCT05868499
Rollover Study From EXG-US-01
A Phase 1 study testing EXG34217 in people with Telomere Biology Disorders With Bone Marrow Failure, run by Elixirgen Therapeutics, Inc. at 1 site in United States.
Trial summary
This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Telomere Biology Disorders With Bone Marrow Failure
- What is being tested?
- EXG34217
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jan 2024to 30 Jan 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- -
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
About this study
Official title: Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01
This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
Read the 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.
- Being tested
- EXG34217
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EXG34217Biological
Single infusion
| Study group | Role | What they receive |
|---|---|---|
| EXG34217 | Experimental | single autologous CD34+ cells contacted ex vivo with EXG-001 Receives: Biological: EXG34217 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
Measured: 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)
ECG (standard digital 12-lead in singlicate)
Measured: 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
Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Measured: 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
Change in Antibody against virus vector and transgene
Measured: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Secondary outcomes (2)
Number of participants with a change in telomere length
Change in telomere length in any peripheral blood cells
Measured: 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.
Blood counts: neutrophils,platelets, or hemoglobin
Measured: Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2023
- Study start1 Jan 2024
- Primary completion30 Jan 2027 (estimated)
- Study completion30 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Telomere Biology Disorders With Bone Marrow FailureStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Sequential Assignment
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Elixirgen Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kasiani Myers, MD, Principal investigator, Cincinnati Children Hospital Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05868499
- Sponsor's protocol ID
- EXG-US-02
- Status last verified
- Jul 2024
- First submitted
- 8 May 2023
- Last updated
- 12 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01
- ClinicalTrials.gov ID
- NCT05868499
- Acronym / sponsor ID
- EXG-US-02
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Elixirgen Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Jan 2024 (actual)
- Primary completion
- 30 Jan 2027 (estimated)
- Study completion
- 30 Jan 2027 (estimated)
- First posted
- 22 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Sequential Assignment
| Arm | Type | Receives |
|---|---|---|
| EXG34217 | Experimental | Biological: EXG34217single autologous CD34+ cells contacted ex vivo with EXG-001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EXG34217Single infusion | Biological | EXG34217 |
Primary outcome
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.
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)
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)
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)
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
2 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Kasiani Myers, MD principal investigator
Cincinnati Children Hospital Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EXG34217 in people with Telomere Biology Disorders With Bone Marrow Failure, run by Elixirgen Therapeutics, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 12 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jul 2024.