Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00143559
• Date of registration: 01/09/2005
• Prospective Registration: No
• Primary sponsor: St. Jude Children's Research Hospital
• Public title: Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies
• Scientific title: Haploidentical Hematopoietic Stem Cell Transplantation Utilizing Partial T-Cell Depletion as Immunotherapy for Hematologic Malignancies
• Date of first enrolment: August 2005
• Target sample size: 17
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00143559
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Gregory Hale, M.D.
• Affiliation: St. Jude Children's Research Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

Eligible participants were assigned to one of two different strata dependent on diagnosis,
disease status and/or past transplant experience. Both strata received the same
intervention but will be followed and analyzed separately.

- Group A must have one of the following diagnosis

- Acute lymphoid leukemia (ALL) in second or subsequent remission or high risk in
first remission

- Acute myeloid leukemia (AML) in remission or with = 25% blasts in bone marrow

- Chronic myeloid leukemia (CML)

- Juvenile myelomonocytic leukemia (JMML)

- Myelodysplastic syndrome (MDS)

- Paroxysmal nocturnal hemoglobinuria (PNH)

- Hodgkin's (HD) or non-Hodgkin's lymphoma (NHL) in second or subsequent remission
after autologous HSCT, or unable to have hematopoietic stem cells collected for
autologous HSCT

- Group B must have one of the following refractory diagnosis (chemoresistant relapse
or primary induction failure)

- Acute lymphoid leukemia (ALL)

- Acute myeloid leukemia (AML) = 25% blast in bone marrow

- Secondary AML / MDS

- Chronic myeloid leukemia (CML) in accelerated phase or blast crisis

- Juvenile myelomonocytic leukemia (JMML)

- Myelodysplastic syndrome (MDS)

- Hodgkin's (HD) or non-Hodgkin's lymphoma (NHL) with residual disease followed by
autologous HSCT or who have chemo-resistant disease

- Or patients who have undergone prior allogeneic HSCT or who have a co-morbid
condition that in the medical opinion of the Transplant Faculty makes standard
myeloablation prohibited

- At least 2 and less than or equal to 21 years of age

- Lacks suitable HLA-identical sibling or matched available unrelated donor and has a
mismatched family member donor that is available, HIV negative and at least 18 years
old

- Cardiac shortening fraction = 25%

- Creatinine clearance = 40 cc/min/1.73m^2

- FVC = 40% of predicted or pulse oximetry = 92% on room air

- Direct bilirubin = 3 mg/dL or SGPT = 500 U/L

- Karnofsky or Lansky (age dependent) performance score of = 50

Exclusion Criteria:

- Known allergy to murine products

- Lactating (female patient)

- Pregnancy (female patient)

- Active central nervous system (CNS) leukemia



Age minimum: 2 Years
Age maximum: 21 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Chronic Myeloid Leukemia

Hodgkin's Lymphoma

Non-Hodgkin Lymphoma

Acute Lymphoblastic Leukemia

Paroxysmal Nocturnal Hemoglobinuria

Acute Myeloid Leukemia

Myelodysplastic Syndrome

Juvenile Myelomonocytic Leukemia

Leukemia

Intervention(s)

Drug: Systematic chemotherapy and antibodies

Device: Miltenyi CliniMACS

Procedure: Allogeneic stem cell transplantation

Primary Outcome(s)

To measure the rate of disease relapse by six months posttransplant in children and young adults with refractory hematologic malignancies who receive a haploidentical stem cell graft processed using the investigational CliniMACS cell sorting device. [Time Frame: September 2006]

Secondary ID(s)

HAPREF

Source(s) of Monetary Support

Please refer to primary and secondary sponsors