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New treatment option for Sickle Cell Disease
Official title Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease
ClinicalTrials.gov ID: NCT03279094
What this study is testing
What is Hematopoietic stem cell transplantation?
Hematopoietic stem cell transplantation is an investigational medicine, given as an once-daily, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 30
You may be able to join if
- 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...
- History of one or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive...
You likely can't join if
- 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...
See the full eligibility 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...
- 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...
- Informed Consent/Assent: All people 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.
- 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...
- Prior autologous or allogeneic transplant.
- Fully HLA-matched related or unrelated donor is available to donate.
- Non-Compliance: people, who in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
The study team makes the final eligibility decision.
Where it's taking place
- Duarte, California, 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 - 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, 1 year to 30 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Duarte, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.