2017 Open Blood and Marrow Transplant Studies

If you have any patients who may be eligible for one of the studies below, please contact Divyesh Kukadiya at 202-476-5456 or email DKukadiy@childrensnational.org.

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.

NCT02338479

13 TLEC: Natural History and Biology of Long-Term Late Effects Following Hematopoietic Cell Transplant for Childhood Hematologic Malignancies

This is a prospective non-therapeutic study, assessing the long-term toxicity of pediatric hematopoietic cell transplant (HCT) for hematologic malignancies. The objective is to report the incidence of chronic kidney disease (CKD), metabolic syndrome and osteopenia at one and two-years following allogeneic HCT for hematologic malignancy.

Any male or female younger than 22 years old at admission for HCT is eligible to participate. There is no lower limit on age.

Pediatric Blood and Marrow Transplant Consortium (PBMTC) in collaboration with CIBMTR

Currently enrolling

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

 

 

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 emoglobinopathies 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.

NCT01743131

Abatacept: Abatacept Combined with a Calcineurin Inhibitor and Methotrexate for Graft Versus Host Disease Prophylaxis: A Randomized Controlled Trial

This is a phase II randomized, double blind, placebo-controlled trial. The purpose of this study is to determine the impact that abatacept will make on the incidence of early, severe acute GVHD (aGvHD), when it is added to a standard GvHD prophylaxis regimen during unrelated-donor hematopoietic stem cell transplantation (HSCT) for patients with hematologic malignancies.

Eligible patients will be at least 6 years old, have high-risk leukemia, adequate organ function, lack an HLA-matched related donor and have an unrelated bone marrow or peripheral blood stem cell donor who is HLA-matched at no less than seven of eight loci (A, B, C, DRB1). This may be an allele or antigen mismatch.

Seattle Children's Hospital

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 CordInTM, 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 CordInTM: 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.

NCT00745420

BMT CTN 0601: Unrelated Donor Reduced Intensity Bone Marrow Transplant for Children with Severe Sickle Cell Disease

The primary objectiveis to determine event-free survival (EFS) at 1 year after unrelated donor (URD) hematopoietic stem cell transplantation (HCT) using bone marrow (BM) in patients with sickle cell disease (SCD). ; Death, disease recurrence or graft rejection by 1 year will be considered events for this endpoint. The primary goal of this phase II study is to determine whether a reduced intensity conditioning regimen in the setting of unrelated donor (URD) HCT is successful in achieving donor engraftment.

Patient is 3-19 years of age at time of enrollment. Patients must have symptomatic SCD.

Phase II, single arm, multi-center trial

Currently enrolling

Allistair Abraham, M.D.

NCT02194439

 aGVHD: Bridging Pediatric and Adult Biomarkers in Graft-Versus-Host Disease

This is a multi-center phase 3 clinical trial.

This study is designed to collect longitudinal biological samples from patients after hematopoietic cell transplantation (HCT) cared for at multiple bone marrow transplant centers to validate biomarkers of both acute and chronic GVHD as well as for use in future unspecified research.

The primary objective is to confirm that ST2 alone or the seven-biomarker panel measured at initiation of GVHD therapy predict a) D180 post-therapy non-relapse mortality; b) D28 post-therapy non-response, and c) GVHD grade 1-4 onset D180 post-therapy non-relapse mortality. All patients receiving an allogeneic hematopoietic stem cell transplant, cord blood transplant, bone marrow transplant, T cell depleted marrow, donor lymphocyte infusion (DLI) or donor cellular infusion (DCI) can be included.

 

Currently enrolling

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

NCT02806947

BMT CTN 1501: A Randomized, Phase II, Multicenter, Open Label, Study Evaluating Sirolimus and Prednisone in Patients with Refined Minnesota Standard Risk, Ann Arbor 1/2 Confirmed Acute Graft-Versus-Host Disease

The study is a phase II randomized, open label, multicenter trial.

Patients of all ages with standard-risk acute GVHD, according to the Refined Minnesota Criteria are eligible. The study will assess sirolimus as an alternate steroid-free treatment for acute GVHD.

 

Currently enrolling

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

NCT01186913

PIDTC 6901: A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders

This protocol is a prospective natural history study in which investigators at 13 centers and additional PBMTC centers caring for patients with SCID in North America will participate. This natural history study will enroll patients with SCID, including ADA-SCID and XSCID (Strata A and C) and also leaky SCID, Omenn syndrome and reticular dysgenesis (Stratum B).

NIH

Currently enrolling

Blachy Davila Saldana, M.D. 

NCT02082353

 

PIDTC 6903: Analysis of Patients Treated for Chronic Granulomatous Disease

The primary objective of this protocol is to estimate the 1-year, 2-year and 3-year (and longer if possible) overall survival probabilities post-HCT of CGD subjects born on or after 1988 who receive HCT on or after 1995.

NIH

Currently enrolling

Blachy Davila Saldana, M.D. 

NCT02064933

PIDTC 6904: Analysis of Patients Treated for Wiskott-Aldrich Syndrome

The primary objectives of this study are to estimate survival at 6 months and 1, 2, 3, 5, 10 and 15 years post-HCT, and to study risk factors for overall survival in this patient population.

NIH

Currently enrolling

Blachy Davila Saldana, M.D.