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RecruitingPhase 2

Allo HSCT for High Risk Hemoglobinopathies

Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

Who can join

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

Age
Up to 55 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

Not stated in the study description — ask the study team about visits and procedures.

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 stem cell transplants from a donor to treat serious blood disorders. It is for people with high-risk inherited blood disorders or other red blood cell disorders that need regular blood transfusions.

A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.

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

Eligibility

Who can join

  • Have sickle cell disease, which makes red blood cells an abnormal shape.
  • People with sickle cell disease and a fully matched sibling donor may qualify no matter how often or how severe their symptoms are. The patient or parents must learn the risks and benefits and freely agree.
  • Have alpha- or beta-thalassemia, a blood disorder that requires regular blood transfusions.
  • Have Diamond Blackfan anemia, a disorder that limits red blood cell production.
  • Have another blood disorder that is not cancer.
  • Score at least 60 on the Karnofsky or Lansky scale, which measures daily activity or play. The study team may consider people with lower scores.
  • If sexually active and you or your partner could become pregnant, agree to use highly effective birth control during treatment and for at least 4 months after transplant.

Who can't join

  • Are pregnant, breastfeeding, or planning to become pregnant during the study. Anyone who could become pregnant must have a negative pregnancy test within 30 days before treatment starts.
  • Have HIV at a level that can be detected in the blood. All people with HIV need an infection specialist's review and an HIV care plan before transplant.
  • Have an active infection that is not controlled. An infection may be allowed if stable or improving after 1 week of proper treatment, or 4 weeks for a known or suspected fungal infection.
  • Have a known allergy to any part of the study treatment.
  • Have a mental health condition or social situation that the study doctor thinks would make it hard to follow study requirements.
  • Have another illness or medical issue that the study doctor thinks would prevent you from joining.

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

Official eligibility criteria

Inclusion Criteria: * Sickle Cell Disease (SCD) * SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent * Transfusion Dependent Alpha- or Beta- Thalassemia * Diamond Blackfan Anemia * Other Non-Malignant Hematologic Disorders * Karnofsky ≥ 60%, Lansky play score ≥ 60. Patients with lower performance score can be considered based on study team's evaluation. * Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the transplant. Exclusion Criteria: * Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 30 days of the start of treatment * HIV infection with a detectable viral load. All HIV+ patients must be evaluated by infectious disease (ID) and an HIV management plan established prior to transplantation. * Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted * Known allergy to any of the study components * Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements * Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study

Study sites (1)

MN