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RecruitingPhase 2

Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

A Phase II Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

Who can join

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

Age
16 Years to 60 Years
Sex
All sexes
Healthy volunteers
Not 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 a partly matched stem cell transplant for people ages 16–60 with serious sickle cell disease. It uses medicines and low-dose whole-body radiation, then checks whether the donated cells are making new blood cells 60 days after the transplant.

The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT). The primary objective of the study is to determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irradiation (TBI) for conditioning and post-transplant cyclophosphamide in patients with sickle cell disease.

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

Eligibility

Who can join

  • Be 16–60 years old.
  • Have sickle cell disease with at least one serious problem listed below. These include a stroke or brain or nerve event lasting over 24 hours; at least 3 severe pain episodes per year; at least 2 painful erection episodes per year needing emergency care; or at least 2 hospital stays for acute chest syndrome, a serious lung problem.
  • Other qualifying sickle cell problems include at least 2 antibodies against donated red blood cells during long-term transfusions; damage to both eyes with major vision loss in one; bone death in at least 2 joints; kidney damage; or high blood pressure in the lungs.
  • Have a daily activity score of at least 60, meaning you can meet most personal needs but may need some help.
  • Have a heart pumping score of at least 40% and a lung gas-transfer test result of at least 50% of the expected value.
  • Have a kidney filtering score of at least 50 and an ALT liver blood test no higher than 3 times the normal limit.
  • Be able and willing to sign a form agreeing to join the study.
  • Have a relative who is a partial tissue-type match and could donate stem cells.

Who can't join

  • Have HIV.
  • Be pregnant.
  • Have a brother or sister who is a full tissue-type match for stem cell donation.

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

Official eligibility criteria

Patient Eligibility: 1. Patients with sickle cell disease are eligible if they have any of the following complications: 1.1 Stroke or central nervous system event lasting longer than 24 hours 1.2 Frequent vaso-occlusive pain episodes, defined as ≥ 3 per year requiring emergency room, acute care center, hospital admissions, or home bedrest leading to absence from work or school. 1.3 Recurrent episodes of priapism, defined as ≥ 2 per year requiring emergency room visits 1.4 Acute chest syndrome with recurrent hospitalizations, defined as ≥ 2 lifetime events 1.5 Red-cell alloimmunization (≥ 2 antibodies) during long-term transfusion therapy 1.6 Bilateral proliferative retinopathy with major visual impairment in at least one eye 1.7 Osteonecrosis of 2 or more joints 1.8 Sickle cell nephropathy, defined by a GFR \< 90mL/min/1.73m2 or the presence of macroalbuminuria (urine albumin \> 300 mg/g creatinine) 1.9 Pulmonary hypertension, defined by a mean pulmonary arterypressure \>25mmHg 2. Age 16-60 years 3. Karnofsky performance status of 60 or higher (Appendix A) 4. Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40% 5. Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50% predicted (after adjustment for hemoglobin concentration) 6. Estimated GFR ≥ 50mL/min/1.73m2 as calculated by the modified MDRD equation 7. ALT ≤ 3x upper limit of normal 8. HIV-negative 9. Patient is not pregnant 10. Patient is able and willing to sign informed consent 11. Patient does not have a fully HLA-matched sibling donor 12. Patient has an HLA-haploidentical relative Donor Eligibility Relatives (parents, offspring, siblings, aunts/uncles, cousins) will be tested by molecular typing of HLA class I (A, B, and C) and class II (DRB1) at low resolution. Only those that are an HLA-haploidentical match (≥ 4/8) will be considered as a potential donor. NOTE: If during testing, a fully HLA-matched sibling donor is found and is willing to donate his/her stem cells, the potential subject will not be eligible for this protocol. Donor consent will be obtained as per standard protocol of the bone marrow transplant unit.

Study sites (1)

IL