By InvitationPhase 1

Rollover Study From EXG-US-01

United States12 participantsStarted 2024-01-01

Plain-language summary

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

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

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

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

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

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

Trial details

NCT IDNCT05868499

SponsorElixirgen Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-01-30

View on ClinicalTrials.gov More Telomere Biology Disorders With Bone Marrow Failure trials

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.