Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents

• Status: Unknown status
• Trial end date: 2018-12-01
• Target enrollment: 0
• Participant gender: All

Summary

Purpose of study: This phase I/II trial is to evaluate the safety and feasibility of TCRαβ-depleted graft from haploidentical family donors in treating children and adolescents with malignant or non-malignant diseases.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Asan Medical Center
• Treatments: Antilymphocyte Serum; Cyclophosphamide; Fludarabine

Criteria

Inclusion Criteria:

A. Disease inclusions

1. Hematologic malignancy:

• Acute lymphoblastic leukemia including induction failure, CR1 (Ph+, t(4:11), hypodiploid and other very high risk features), ≥ CR2, infant ALL with MLL or other unfavorable features

• Acute myeloid leukemia excluding CR1 with t(8:21), inv(16), t(15:17), and Down syndrome

• Myelodysplastic syndrome: RCC with -7 or RCC in need of transfusion

• Chronic myeloid leukemia in AP

• Juvenile myelomonocytic leukemia

• Malignant lymphoma, NHL or HD, after failed autologous HSCT

• Other

2. Non-hematologic malignancy

• Relapsed or refractory solid tumors including neuroblastoma, rhabdomyosarcoma and so on

3. Non-malignant hematologic disease

• Acquired severe and very severe aplastic anemia

• Fanconi anemia

• Paroxysmal nocturnal hemoglobinuria

• Congenital dyserythropoietic anemia

• Others

4. Inherited or metabolic disease

• Hemophagocytic lymphohistiocytosis

• Malignant osteopetrosis

• Storage diseases

• Others B. Recipient inclusions

1. Age < 21 years 2. No HLA-identical stem cell donor available 3. Lansky-Play performance score >60 4. No active infection at the time of transplantation

Exclusion Criteria:

1. HIV-infection

2. Presence of active and serious infection

3. Cardiac ejection fraction <35% on echocardiography

4. Severe pulmonary dysfunction (DLCO <30%)

5. Liver function abnormalities with bilirubin >4mg/dL and elevation of transaminases > 400U/L

6. Concurrent severe or uncontrolled medical disease

7. Patients who are pregnant

8. Patients unwilling or unable to comply with the protocol or unable to give informed consent