WithdrawnEarly Phase 1

Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

Stopped: Competing protocol opened in Adult SCT that will include pediatric patients and is now multi-center. No patients enrolled on study.

0Started 2020-12-29

Plain-language summary

This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies.

Who can participate

Age range

2 Years – 30 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: * Patients 2-30 years-of-age with confirmed sickle cell disease (SCD) (SS \& sickle beta \[SB\]-thalassemia, both sickle beta 0 \[SB0\] and sickle beta plus \[SB+\]) or severe B-thalassemia major are potentially eligible * Patients with SCD should also meet the following eligibility criteria as outlined by the Center for Medicaid and Medicare Services: sickle cell disease and at least one of the following: * Stroke or neurological deficit lasting \> 24 hours * Recurrent acute chest syndrome (ACS): 2 or more episodes of ACS in 2-year period preceding enrollment * Recurrent vaso-occlusive pain crises: 3 or more episodes per year in 2-year period preceding enrollment or recurrent priapism (3 or more episodes in the 2 years preceding enrollment) * Chronic transfusion program defined as 8 or more packed red blood cells (PRBC) transfusions per year to prevent central nervous system and/or vaso-occlusive complications in 1-year period preceding enrollment * Impaired neuropsychological function and abnormal cerebral magnetic resonance imaging (MRI) scan (silent strokes) * Stage I or II sickle lung disease * Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate 30-50% of predicted normal value) * Bilateral proliferative retinopathy and major visual impairment in at least one eye * Osteonecrosis of multiple joints * Echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) \>= 2.7 m/sec * Patients…

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

Event-free survival (EFS)

Timeframe: At 2 years post-hematopoietic cell transplantation (HCT)

Trial details

NCT IDNCT04776850

SponsorM.D. Anderson Cancer Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2022-12-05

View on ClinicalTrials.gov More Beta Thalassemia Major trials