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Open Blood and Marrow Transplant Studies

If you have any questions about the studies below, please email or call 202-476-5456.

Trial #
Title
Description
Sponsor
Status
Investigator

NCT02524847

Therakos: Single-Arm Study to Assess the Efficacy of UVADEX® (Methoxsalen) Sterile Solution in Conjunction with the THERAKOS® CELLEX® Photopheresis System in Pediatric Patients With Steroid-Refractory Acute Graft Versus Host Disease (aGVHD)

The study will evaluate the efficacy of ECP in pediatric patients with steroid refractory a GVHD.

Males or females 1 to 21 years of age at the time of consent are eligible to participate. They must have steroid-refractory grade B-C aGVHD.

Therakos Inc. funded phase III trial multi-center trial

Currently enrolling

David A. Jacobsohn, M.D., Sc.M.

NCT02959944

Ibrutinib: A Randomized, Double-Blind Phase 3 Study of Ibrutinib in Combination With Corticosteroids versus Placebo in Combination With Corticosteroids in Subjects with New Onset Chronic Graft Versus Host Disease (cGVHD)

This is a Phase 3, multicenter, international, randomized, double-blind study of oral ibrutinib to evaluate the efficacy of ibrutinib in combination with prednisone versus placebo in combination with prednisone in subjects with new onset chronic GVHD. Eligible patients will have new onset moderate or severe cGVHD, history of an allogeneic hematopoietic cell transplant (HCT) and need for systemic treatment with corticosteroids for cGVHD.

Pharmacyclics LLC

Currently enrolling

David A. Jacobsohn, M.D., Sc.M.

NCT02867800

Abatacept for Graft Versus Host Disease Prophylaxis after Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease: a Sickle Transplant Alliance for Research Trial

This is a single arm pilot study in pediatric patients receiving allogeneic hematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD). Patients between the ages of 3 and 20.99 years with Hgb SS or SB0 thalassemia receiving HLA matched related or unrelated donor bone marrow transplants who are at increased risk for GVHD will be eligible. All patients getting unrelated donor transplants will be considered to be at increased risk for GVHD; patients receiving related donor transplants meeting at least one of the following two criteria will also be considered to be at increased risk for GVHD: 1) they are at least 10 years old; 2) their donor is at least 10 years old.

Bristol Myer Squibb

Currently enrolling

Allistair Abraham, M.D. 

NCT02766465

A Study to Compare Bone Marrow Transplantation to Standard Care in Adolescents and Young Adults with Severe Sickle Cell Disease

This study is designed as a phase II, multi-center trial of hematopoietic cell transplantation (HCT) versus standard of care in adolescents and young adults with severe sickle cell disease (SCD). Eligible participants are biologically assigned to HCT or standard of care based on the availability of HLA-matched related or unrelated donor after confirmation of clinical eligibility. Insurance coverage and donor availability are not known at referral or consultation. Eligible patients are ≥ 15 and <41years of age with severe sickle cell disease [Hemoglobin SS (Hb SS), Hemoglobin SC (Hb SC) or Hemoglobin S Beta thalassemia (Hb Sβ) genotype] with at least one of the following manifestations: clinically significant neurologic event (stroke) or neurological deficit lasting >24 hours; history of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite adequate supportive care measures (i.e. asthma therapy);three or more pain crises per year in the 2-year period preceding referral (required intravenous pain management in the outpatient or inpatient hospital setting); administration of regular red blood cell (RBC) transfusion therapy; defined as 8 or more transfusion events per year (in the 12 months before enrollment) to prevent vaso-occlusive clinical complications (i.e. pain, stroke, or acute chest syndrome); an echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity ≥ 2.7 m/sec

National Institutes of Health, National Heart, Lung, and Blood Institute, National Cancer Institute

Currently enrolling

Allistair Abraham, M.D. 

HLA Matched Related Hematopoietic Stem Cell Transplantation for Children with Less Severe Sickle Cell Disease: a Sickle Transplant Alliance for Research (STAR) Trial

This is a multicenter, single arm trial aimed to To prospectively assess the safety and efficacy of HLA matched related hematopoietic stem cell transplantation (HSCT) using the reduced intensity conditioning regimen fludarabine, alemtuzumab and melphalan in children with less severe sickle cell disease (SCD). Patients with Hgb SS or SB0 thalassemia who have had less severe courses (less severe disease) will be eligible. To limit the risks of transplantation, only patients who are also at low risk for graft versus host disease (GVHD) will be eligible; to be considered low risk, recipients must be greater than or equal to 2 years and less than 10 years of age at the time of transplant and have a matched sibling donor who is less than 10 years of age.

Currently enrolling

Robert Nickel, M.D., MSc

 

 

NCT03587272

Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children with Sickle Cell Disease (SUN)

This multicenter prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, “SUN”) can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD). Patients with SCD aged 2-21.99 years who have an HLA-identical sibling donor.

Doris Duke Charitable Foundation

Currently enrolling

Allistair Abraham, M.D.

NCT02918292

Optimizing Cord Blood and Haploidentical Aplastic Anemia Transplantation (CHAMP)

Assess overall survival (OS) separately in 2 cohorts (unrelated cord blood [UCB] and haploidentical [haplo] marrow) at 1 year post-hematopoietic stem cell transplantation (HSCT) in patients with severe aplastic anemia (SAA). Diagnosis of SAA, without a fully matched related or unrelated donor available, but with either an UCB or haplo marrow donor available. Exclusions include inherited bone marrow failure syndrome, previous hematopoietic stem cell or solid organ transplant, uncontrolled infection, inadequate organ function, and performance score < 60.

NIH

Currently enrolling

Blachy Davila Saldana, M.D. 

NCT02065869

Bellicum: Phase I/II Study of CaspaCIDe T Cells from an HLA-Partially Matched Family Donor after Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders

The purpose is to evaluate the safety and feasibility of BPX-501 (Genetically Modified T Cells with Suicide Safety Switch)T cells infused after partially mismatched, related, T cell-depleted HSCT in pediatric patients. Male or female 1 to 21 years of age eligible to participate.

Patients with life-threatening hematological malignancies and non-malignant disorders (primary immune deficiencies, severe aplastic anemia not responding to immune suppressive therapy, osteopetrosis, selected cases of hemoglobinopathies and congenital/hereditary cytopenia) are eligible for this study.

Bellicum Pharmaceuticals, Inc. funded phase 1 and 2 of study

Currently enrolling

David A. Jacobsohn, M.D., Sc.M.

NCT02165007

HAPSICKLE: Haploidentical Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia Using CD34+ Positive Selected Grafts

The study is designed as a pilot trial of reduced intensity Haploidentical HSCT in patients with sickle cell disease and thalassemia. The primary objective of the trial is to assess the safety and toxicity of reduced intensity conditioning haploidentical hematopoietic stem cell transplantation in patients with hemoglobinopathy. Individuals up to 22 years at the time of consent are eligible to participate. This trial is a single center study of 6 patients at Children’s National Medical Center.

Catherine Bollard, M.D.

Currently enrolling

Allistair Abraham, M.D.

NCT02504619

CordIn: Allogeneic Stem Cell Transplantation of CordIn, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells in Patients with Hemoglobinopathies

The overall study objectives are to evaluate the safety and efficacy of CordIn: single Ex Vivo expanded cord blood unit transplantation in Patients with Hemoglobinopathies (sickle cell disease (SCD) or thalassemia major)

Participants must be 2- 25 years of age and at least 10 kg and eligible for allogeneic SCT for treatment of SCD or thalassemia. Once the best available cord blood units (CBUs) have been identified, the patient will be screened for the study. The trial consists of three phases: Screening/Preparative phase, Screening/Conditioning phase and Transplantation/Follow-up phase.

Gamida Cell Ltd. funded phase 1 and 2 of the study

Currently enrolling

Allistair Abraham, M.D.

NCT00920972

Wash U (01-0923): Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime

  • Stratum 2: Conditioning includes hydroxyurea and low dose thiotepa in addition to alemtuzumab, fludarabine and melphalan for patients with thalassemia receiving an unrelated donor transplant with bone marrow or umbilical cord blood stem cells.
  • Stratum 3: Conditioning will include hydroxyurea and low dose thiotepa in addition to alemtuzumab, fludarabine and melphalan for patients with hemoglobinopathy, receiving a minimally mismatched unrelated donor transplant with bone marrow or a matched or minimally mismatched single or double umbilical cord blood product.
  • Stratum 4: Conditioning will include hydroxyurea and low dose thiotepa in addition to alemtuzumab, fludarabine and melphalan for patients with non-malignant disorders, (excluding hemoglobinopathy) receiving a minor mismatched unrelated donor transplant with bone marrow or a matched or minimally mismatched single or double umbilical cord blood product.

Washington University School of Medicine

Currently enrolling

Stratum 2, 3 and 4 are open

David A. Jacobsohn, M.D., Sc.M.

NCT02596997

CMX001-351: An Intermediate-Size, Expanded Access Protocol to Provide Brincidofovir for the Treatment of Serious Adenovirus Infection or Disease

The study is only available for emergency use. This is a phase 3 trial. An intermediate-size, expanded access protocol to provide Brincidofovir for the treatment of serious adenovirus infection or disease. This expanded access study consists of a screening evaluation to confirm study eligibility and an initial 12-week treatment phase (Day 1 to Week 12) during which subjects will be administered BCV therapy twice-weekly (BIW) for a nominal 24 doses.

Chimerix

Currently enrolling

David A. Jacobsohn, M.D., Sc.M