Myeloid Chimerism study: CliniMACS CD34 Reagent
Official title Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
ClinicalTrials.gov ID: NCT04008368
What this study is testing
- What it's testing
- The study wants to see if a repeat transplant can increase donor cells in the bone marrow and stop the disease from returning.
- Who it's for
- People age 4 or older with sickle cell disease who had a prior transplant that failed, and their willing donor relatives.
- What's involved
- Participants will undergo many screening tests, receive a donor stem cell infusion through a long IV line, take medicines to help accept the cells, stay in the hospital about a month, and have follow‑up visits and tests for several years. Donors will have several visits, take a short drug regimen, and give stem cells from a blood draw.
- Time commitment
- The recipient’s involvement lasts about 5 years with an initial 30‑day hospital stay and frequent visits for the first 3 months, then visits every 6‑12 months; donors have 7‑8 visits over a short period.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 80. Healthy volunteers may be eligible.
You may be able to join if
- - recipient
- Patient with history of SCD or beta thalassemia who previously underwent allogeneic hematopoietic stem cell transplantation (HSCT)
- Patient with recurrent SCD defined as HbS greater than or equal to 50% for donors with sickle cell trait and greater than or equal to 10% for donors...
- Persistent donor chimerism levels
- Age greater than or equal to 4 years
You likely can't join if
- Exclusion criteria- recipient
- ECOG performance status of 3 or more or if \<16 years of age Lansky score of 50 or lower.
- Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month...
- Patients with fever or suspected minor infection should await resolution of symptoms before signing the consent
- Major anticipated illness or organ failure incompatible with survival from PBSC transplant
- Pregnant or breastfeeding
See the full eligibility criteria
- - recipient
- Patient with history of SCD or beta thalassemia who previously underwent allogeneic hematopoietic stem cell transplantation (HSCT)
- Patient with recurrent SCD defined as HbS greater than or equal to 50% for donors with sickle cell trait and greater than or equal to 10% for donors with HbAA with recurrent clinical manifestations (for example but not...
- Persistent donor chimerism levels
- Age greater than or equal to 4 years
- Negative beta-HCG
- Ejection fraction greater than or equal to 35%
- DLCO greater than or equal to 35% (Note: This criterion may be omitted in young children (e.g. near the age of 5 years old) or other individuals who may have difficulty understanding or complying with the instructions...
- Exclusion criteria- recipient
- ECOG performance status of 3 or more or if \<16 years of age Lansky score of 50 or lower.
- Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to signing the consent
- Patients with fever or suspected minor infection should await resolution of symptoms before signing the consent
- Major anticipated illness or organ failure incompatible with survival from PBSC transplant
- Pregnant or breastfeeding
- History of secondary malignancies (other than localized skin cancer)
- Donor-specific HLA antibodies with mean fluorescent intensity \>= 2,000 Exclusion- donor None
The study team makes the final eligibility decision.
Where it is enrolling
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 80 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Bethesda, Maryland, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.