RightFit
RecruitingPhase 1 / Phase 2

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease or Beta-Thalassemia and Falling Donor Myeloid Chimerism Levels

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
2 Years to 80 Years
Sex
All sexes
Healthy volunteers
Accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Visits mentioned: 8 visits
  • Time periods mentioned: 5 years
  • Procedures mentioned: Blood draws, Physical exams, X-rays, Infusions

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Design: Participants will be screened with medical history, physical exam, and blood tests."
  • "Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams."
  • "Donors will have 7-8 visits."
  • "In first 3 months after the infusion, recipients will have many visits."
  • "Then they will have visits every 6 months to 1 year for 5 years."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests a second blood stem cell transplant for people age 4 or older whose sickle cell disease or beta thalassemia has returned after a transplant. Beta thalassemia is an inherited blood disorder. The treatment aims to make more room in the bone marrow for donor cells, using the same related donor as before.

Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body. Objective: To cure people s sickle cell disease by giving a second treatment that makes more room in their bone marrow for donor cells. Eligibility: People ages 4 and older with sickle cell disease who had a transplant but the disease returned, and their donor relatives. Donors can be 2 years of age or older. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams. They must have a caregiver. Donors will have 7-8 visits. They will take a drug for 5-6 days to prepare them for the donation. For the donation, blood is taken from a vein in the arm or groin. The stem cells are collected. The rest of the blood is returned. This may be repeated. Recipients will get a long IV line in their arm or chest for about 1-2 months. They will take drugs to help their body accept the donor cells. They will get the donor cells and red blood cell transfusions through the line. They will stay in the hospital about 30 days after the transfusion of donor cells. In first 3 months after the infusion, recipients will have many visits. Then they will have visits every 6 months to 1 year for 5 years. During those visits they will repeat some of the screening tests....

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Recipients must be age 4 or older and have had a blood stem cell transplant from a donor.
  • Sickle cell disease or beta thalassemia must have returned, with symptoms and blood test results that meet the study rules. Some sickle cell blood test rules may be waived by the study doctor.
  • Recipients must still have some cells from the first donor.
  • Recipients must have a negative pregnancy blood test.
  • The heart must pump out at least 35% of its blood with each beat.
  • A test of how well the lungs move oxygen into the blood must be at least 35%. This test may be skipped for people who cannot follow the testing instructions.
  • Recipients must have a caregiver.
  • Donors must be the relative who donated for the first transplant, be age 2 to under 80, pass donation checks, and agree to donate stem cells and blood for research.

Who can't join

  • Recipients whose daily activity or ability to care for themselves is too limited under the study's scoring rules.
  • Recipients with an uncontrolled bacterial, viral, or fungal infection in the month before signing the consent form.
  • Recipients with a fever or a possible minor infection must wait until symptoms clear before signing the consent form.
  • Recipients with an expected major illness or organ failure that would make surviving the transplant unlikely.
  • Recipients who are pregnant or breastfeeding.
  • Recipients with a history of a second cancer, except skin cancer limited to one area.
  • Recipients with immune proteins that target the donor's cells at or above the study's test cutoff.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

* INCLUSION CRITERIA: Inclusion criteria- recipient 1. Patient with history of SCD or beta thalassemia who previously underwent allogeneic hematopoietic stem cell transplantation (HSCT) 2. 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 limited to recurrent painful crises, acute chest syndrome, priapism, or severe anemia) or patients with recurrent beta thalassemia defined as clinical manifestations such as transfusion-dependence or evidence of extramedullary hematopoiesis. The HbS requirement may be waived by the PI or designee for reasons such as the patient is requiring chronic transfusion therapy or otherwise meets clinical criteria for return of SCD. 3. Persistent donor chimerism levels 4. Age greater than or equal to 4 years 5. Negative beta-HCG 6. Ejection fraction greater than or equal to 35% 7. 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 for accurate testing.) Inclusion- donor Related donor from original transplant, deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Related donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Age \>= 2 years \<80 years old EXCLUSION CRITERIA: Exclusion criteria- recipient 1. ECOG performance status of 3 or more or if \<16 years of age Lansky score of 50 or lower. 2. Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to signing the consent 3. Patients with fever or suspected minor infection should await resolution of symptoms before signing the consent 4. Major anticipated illness or organ failure incompatible with survival from PBSC transplant 5. Pregnant or breastfeeding 6. History of secondary malignancies (other than localized skin cancer) 7. Donor-specific HLA antibodies with mean fluorescent intensity \>= 2,000 Exclusion- donor None

Study sites (1)

MD