Open Blood and Marrow Transplantation Studies

ClinicalTrials.gov Identifier: NCT06585774

Description

• This study will be conducted to compare the efficacy of axatilimab versus placebo in combination with corticosteroids as initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD)

Key Eligibility Criteria

• ≥ 12 years of age with new-onset moderate or severe cGVHD
• History of allo-HCT from any donor HLA type 

Sponsor

• Incyte Corporation
  

Status

• Enrolling

Investigator

• David Jacobsohn, MD, SCM, MBA 

ClinicalTrials.gov Identifier: NCT05600426

Description

• Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia).
  
  This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA.
  
  The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms.
  
  This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.

Key Eligibility Criteria

•  Age ≤25 years old with confirmed diagnosis of idiopathic SAA
• No suitable fully matched related donor available and at least 2 unrelated donors noted on NMDP search who are well matched

Sponsor

• Boston Children's Hospital

Status

• Enrolling

Investigator

• Hyojeong Han, MD

 

ClinicalTrials.gov Identifier: NCT03619551

Description

• The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants.
  
  The study will test if patients receiving transplant using either a low dose busulfan or a medium dose busulfan will have immune recovery of both T and B cells, measured by the ability to respond to immunizations after transplant. The exact regimen depends on the subtype of SCID the patient has. Donors used for transplant must be unrelated or half-matched related (haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T cells removed, using a newer, experimental approach of a well-established technology. Once the stem cell transplant is completed, patients will be followed for 3 years. Approximately 9-18 months after the transplant, vaccinations will be administered, and a blood test measuring whether your child's body has responded to the vaccine will be collected.

Key Eligibility Criteria

• Infants with SCID, either typical or leaky or Omenn syndrome receiving haploidentical related mobilized peripheral blood cells

Status

• Enrolling

Investigator

• Elizabeth Hicks, MD

ClinicalTrials.gov Identifier: NCT05621291

Description

• Participants will visit the clinic every 2 weeks starting 42 days after they receive CART therapy. Each visit will be about the same amount of time as a regular clinic visit, about 8 hours. Participants will have blood drawn for testing on each visit. Bone marrow biopsy/aspirate will be done during 4 of the visits at routine timepoints after CART. A needle will be inserted to draw a sample of tissue from inside the bone in the hip. A small amount of blood and tissue will be tested with ClonoSEQ and to evaluate for normal B-cells side by side with the standard tests. The combined testing may help determine whether participants are eligible for HCT and/or at risk of relapse after CART. Participants will be in the study for 2 years.

Key Eligibility Criteria

• Age >=1 year and <= 25 years old at the time of CD19 CART infusion
• Confirmed diagnosis of CD19+ B-ALL with an informative NGS clonality sample

Sponsor

• National Cancer Institute (NCI)

Status

 

• Enrolling

Investigator

 

• Anant Vatsayan, MD

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). 

Sponsor

• Doris Duke Charitable Foundation

Key Eligibility Criteria

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

Status

• Currently enrolling

Investigator

• Robert Nickel, MD, MSc

ClinicalTrials.gov Identifier: NCT06358638

Description

• This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies. 

 Key Eligibility Criteria:

• General:

• Patients with SCD age 2-24.99 years who have a healthy HLA-identical sibling donor with major ABO incompatibility OR patients with RBC alloantibodies against other donor RBC antigens

• Patients must have an absolute neutrophil count of 1 x 109/L and a platelet count of 100 x 109/L

• Lansky/Karnofsky score of, at least, 70

• Patients with genotypes hemoglobin SS and Sβ0 thalassemia must have at least one of the following:

• History of an abnormal transcranial Doppler measurement defined as TCD velocity ≥200 cm/sec by the non-imaging technique measured at a minimum of two separate occasions

• Progression of CNS vasculopathy on MRA determined to be secondary to SCD

• History of cerebral infarction on brain MRI (overt stroke, or silent stroke if ≥3 mm in one dimension, visible in two planes on fluid-attenuated inversion recovery T2-weighted images)

• History of two or more episodes of Acute Chest Syndrome (ACS) in lifetime

• History of three or more SCD pain events requiring treatment with an opiate or IV pain medication in lifetime

• History of any hospitalization for a complication secondary to SCD (does NOT include empiric hospitalizations for fever only)

• History of two or more episodes of priapism

• Administration of regular RBC transfusions (≥8 transfusions episodes in the previous 12 months).

• At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration

• Patients with all other sickle genotypes (e.g. hemoglobin SC, Sβ+ thalassemia, etc.) must have at least one of the following:

• Clinically significant neurologic event (overt stroke)

• History of two or more episodes of ACS in the 2-year period preceding enrollment

• History of three or more SCD pain events requiring treatment with an opiate or IV pain medication (inpatient or outpatient) in the 1-year period preceding enrollment

• History of any hospitalization for SCD pain or ACS while receiving hydroxyurea treatment

• History of two or more episodes of priapism (erection lasting ≥4 hours or requiring emergent medical care)

• Administration of regular RBC transfusions (≥8 transfusions in the previous 12 months)

• At least two episodes of splenic sequestration requiring red blood cell transfusion or splenectomy after at least one episode of splenic sequestration.

Sponsor

• Children's National Research Institute

Status

• Currently enrolling

Investigator

• Robert Nickel, MD, MSc

ClinicalTrials.gov Identifier: NCT02646839

Description

• This is a phase II, open-label, non-randomized, prospective study of haploidentical transplantation using KIR-favorable donors for children with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). The relationship of KIR2DL1 polymorphisms to survival in children with these diseases undergoing any approach to allogeneic HCT during the study time frame will also be determined.

Key Eligibility Criteria

•  Age < 22 years with ALL high-risk in first remission, ALL in second remission: B-cell; early, Myelodysplastic syndrome (MDS), High-risk AML or AML in second or subsequent morphologic remission receiving a haploidentical transplant

Sponsor

• Michael Pulsipher

Status

• Enrolling

Investigator

Anant Vatsayan, MD

Description

• CD 34 selection for haploidentical transplants

Key Eligibility Criteria

• Patient is deemed by their transplant attending to have a life-threatening disease that is only curable by allogeneic bone marrow transplantation, but does not have a suitable donor
• Patient has undergone an allogeneic bone marrow transplant and has failed to engraft 

Sponsor

• Children's National Hospital

Status

• Enrolling

Investigator

• Evelio Perez- Albuerne, MD, PhD