Open Blood and Marrow Transplantation Studies

ClinicalTrials.gov Identifier: NCT02867800

Description

• This is a single arm pilot study in pediatric patients receiving allogeneic hematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD).

Key Eligibility Criteria

• 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.

Sponsor

• Bristol Myer Squibb

Status

• Currently enrolling

Investigator

• Robert Nickel, MD, MSc

ClinicalTrials.gov Identifier: NCT02766465

Description

• 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).

Key Eligibility Criteria

• 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.

Sponsor

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

Status

• Currently enrolling

Investigator

• Robert Nickel, MD, MSc

Description

• 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).

Key Eligibility Criteria

• 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.

Status

• Currently enrolling

Investigator

• Robert Nickel, MD, MSc

ClinicalTrials.gov Identifier: NCT03587272

Description

• 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).

Key Eligibility Criteria

• Patients with SCD aged 2-21.99 years who have an HLA-identical sibling donor.

Sponsor

• Doris Duke Charitable Foundation

Status

• Currently enrolling

Investigator

• Robert Nickel, MD, MSc

ClinicalTrials.gov Identifier: NCT02065869

Description

• 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.

Key Eligibility Criteria

• 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.

Sponsor

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

Status

• Currently enrolling

Investigator

• David A. Jacobsohn, MD, ScM

ClinicalTrials.gov Identifier: NCT00920972

Description

• 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.

Sponsor

• Washington University School of Medicine

Status

• Currently enrolling: Stratum 2, 3 and 4 are open

Investigator

• David A. Jacobsohn, MD, ScM