Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
A Pilot Study of Pre-transplant Immunosuppressive Therapy for Haploidentical Transplants in Patients With 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
- 1 Year to 30 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 the safety and harmful effects of a stem cell transplant for people ages 1 to 30 with sickle cell disease. Patients get two rounds of medicine to weaken the immune system, then treatment to destroy bone marrow cells before a transplant from a half-matched family donor. The goal is to make more donors available and help donor cells stay in the body.
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 1 to 30 with sickle cell anemia type Hgb SS or SB° thalassemia, and a starting sickle hemoglobin level above 60%.
- Have at least one of the disease problems listed in the next three bullets.
- A stroke, a nerve or brain problem lasting over 24 hours, or high blood flow speed on a brain ultrasound; or a heart ultrasound showing a tricuspid valve leak speed of at least 2.5 meters per second.
- At least one acute chest syndrome episode (a serious lung problem) in the past two years despite care; or at least one severe sickle cell pain crisis each year for the past two years despite care.
- Repeated unwanted erections needing treatment; bone death in at least two joints despite care; or at least eight red blood cell transfusions yearly for over one year to prevent sickle cell problems.
- Have a half-matched family donor, with no fully matched sibling or 10/10-matched unrelated donor available.
- Men and women who can become pregnant must agree to use birth control or avoid sex before joining and until six months after study participation ends. Report any known or suspected pregnancy right away.
- Be able to understand the study and be willing to sign a written consent form.
Who can't join
- An illness that is not controlled, including an active bacterial, viral, or fungal infection.
- Receiving another experimental drug, biological treatment, chemotherapy, or radiation treatment.
- Past allergic reactions to substances like those used before or after the transplant.
- Pregnancy.
- Active cancer, except non-melanoma skin cancer.
- A medical, brain, nerve, or mental health problem that makes treatment unsafe, slows blood cell recovery, or prevents following treatment; or likely trouble following required safety checks.
- A past stem cell transplant using your own cells or donor cells.
- A fully matched family or unrelated donor is available to donate.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion criteria * Diagnosis: Patients with sickle cell anemia (Hgb SS or SB° Thalassemia) with baseline Hgb S more than 60%. * Disease status: * Significant neurologic event (stroke) or any neurological deficit lasting \> 24 hours; or increased transcranial Doppler velocity (\>200 m/s). * History of one or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea). * History of one or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea). * Recurrent priapism requiring medical therapy. * Osteonecrosis of two or more joints despite the institution of supportive care measures. * Prior treatment with regular RBC transfusion therapy, defined as receiving 8 or more transfusions per year for \> 1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome) * Echocardiograph finding of tricuspid valve regurgitation jet (TRJ) velocity ≥ 2.5 m/sec. * Ages 1 to 30. * Child Bearing Potential- Transplantation could be teratogenic and/or lethal to the developing fetus. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for six months following duration of study participation. Should a woman become pregnant or suspect that she is pregnant while participating on the trial, she should inform her treating physician immediately. * Informed Consent/Assent: All subjects must have the ability to understand and the willingness to sign a written informed consent. * The recipient must have a related donor who is genotypically haploidentical on HLA-A, B, C and DRB1 loci. * No HLA matched sibling or 10/10 matched unrelated donor is available. Exclusion criteria: * Any uncontrolled illness including ongoing or active bacterial, viral or fungal infection. * Patients may not be receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy. * History of allergic reactions attributed to compounds of similar chemical or biologic composition to any in the pre- or post-transplant regimen. * Pregnant women are excluded from this study. * Patients with any active malignancy are ineligible for this study, other than non-melanoma skin cancers. * Medical problem or neurologic/psychiatric dysfunction which would impair patient ability to be compliant with the medical regimen and to tolerate transplantation or would prolong hematologic recovery which in the opinion of the principal investigator would place the recipient at unacceptable risk. * Prior autologous or allogeneic transplant. * Fully HLA-matched related or unrelated donor is available to donate. * Non-Compliance: Subjects, who in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
Study sites (1)
CA
- City of Hope Medical Center · Duarte, CA 91010Mary Suarez, CRC · Study contact · 626-218-5795 · masuarez@coh.orgAnna B. Pawlowska, MD · Principal investigatorMore trials in Duarte