Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-01-31

Study Completion Date

2018-12-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Haploidentical Hematopoietic Stem Cell Transplantation Malignant Disease Non-malignant Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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HAPLO

Group Type EXPERIMENTAL

Fludarabine

Intervention Type DRUG

40mg/M2 once daily IV on days -7 to -2

Cyclophosphamide

Intervention Type DRUG

50 mg/kg IV on day -3 and -2

anti-thymocyte globulin

Intervention Type BIOLOGICAL

filgrastim

Intervention Type BIOLOGICAL

Beginning on day 4 and continuing until blood counts recover

Total body irradiation

Intervention Type RADIATION

200 cGy per day on D-6 to -4 (eligible disease except aplastic anemia) 200 cGy per day on D-5 \& -4 (severe aplastic anemia)

TCRαβ-depleted hematopoietic cell transplantation

Intervention Type PROCEDURE

CliniMACS

Intervention Type DEVICE

Immunogenetic depletion of TCRαβ cells

Interventions

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Fludarabine

40mg/M2 once daily IV on days -7 to -2

Intervention Type DRUG

Cyclophosphamide

50 mg/kg IV on day -3 and -2

Intervention Type DRUG

anti-thymocyte globulin

Intervention Type BIOLOGICAL

filgrastim

Beginning on day 4 and continuing until blood counts recover

Intervention Type BIOLOGICAL

Total body irradiation

200 cGy per day on D-6 to -4 (eligible disease except aplastic anemia) 200 cGy per day on D-5 \& -4 (severe aplastic anemia)

Intervention Type RADIATION

TCRαβ-depleted hematopoietic cell transplantation

Intervention Type PROCEDURE

CliniMACS

Immunogenetic depletion of TCRαβ cells

Intervention Type DEVICE

Eligibility Criteria

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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

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No