Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

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

PHASE2/PHASE3

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-09-30

Study Completion Date

2012-12-31

Brief Summary

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The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Malignant infantile osteopetrosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.

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Detailed Description

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Conditions

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Osteopetrosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Intervention

Group Type EXPERIMENTAL

Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)

Intervention Type DRUG

For sibling full match:

* Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po
* Cyclophosphamide 200 mg/kg iv

For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood:

* Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po
* Cyclophosphamide 200 mg/kg iv
* ATG rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg

For haploidentical:

* Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po
* Cyclophosphamide 200 mg/kg iv
* Fludarabine 160 mg/m\^2

Stem Cell Transplantation

Intervention Type PROCEDURE

Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:

1. Sibling full match
2. Other related full match
3. Sibling or other related with 1 mismatch antigen
4. Cord Blood
5. Haploidentical

Cyclosporin, Methotrexate (GVHD prophylaxis)

Intervention Type DRUG

* Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in PBSCT and +11 in BMT or UCBT) then 9 mg/kg/day po
* 10 mg/m\^2 iv day +1 then 6 mg/m\^ iv day +3 and +6 (Not for UCBT)

Interventions

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Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)

For sibling full match:

* Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po
* Cyclophosphamide 200 mg/kg iv

For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood:

* Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po
* Cyclophosphamide 200 mg/kg iv
* ATG rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg

For haploidentical:

* Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po
* Cyclophosphamide 200 mg/kg iv
* Fludarabine 160 mg/m\^2

Intervention Type DRUG

Stem Cell Transplantation

Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:

1. Sibling full match
2. Other related full match
3. Sibling or other related with 1 mismatch antigen
4. Cord Blood
5. Haploidentical

Intervention Type PROCEDURE

Cyclosporin, Methotrexate (GVHD prophylaxis)

* Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in PBSCT and +11 in BMT or UCBT) then 9 mg/kg/day po
* 10 mg/m\^2 iv day +1 then 6 mg/m\^ iv day +3 and +6 (Not for UCBT)

Intervention Type DRUG

Other Intervention Names

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Systemic chemotherapy HSCT Graft-versus-host disease (GVHD) prophylaxis

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of Osteopetrosis confirm by bone biopsy and radiographic imaging
* Age up to 5 year old

Exclusion Criteria

* Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant
* Creatinine clearance ≤ 40ml/min/1.73m\^2 or RTA
* Bilirubin ≥ 3mg/dL
* SGPT ≥ 500 U/L
* Current severe infection
* Evidence of CNS involvement
* Morbidity such as blindness or deafness

Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No