Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Osteopetrosis Clinical Trial

Official title:

Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01087398
• Other study ID #: HORCSCT-0905
• Status: Recruiting
• Phase: Phase 2/Phase 3
• First received: March 13, 2010
• Last updated: May 31, 2012
• Start date: September 2009
• Est. completion date: December 2012

Study information

• Verified date: May 2012
• Source: Tehran University of Medical Sciences
• Contact: Amir Ali Hamidieh, MD
• Phone: 84902645
• Email: aahamidieh[@]sina.tums.ac.ir
• Is FDA regulated: No
• Health authority: Iran: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: December 2012
• Est. primary completion date: November 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 5 Years

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Osteopetrosis

Intervention

Drug:
• 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

Procedure:
• Stem Cell Transplantation
Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source: Sibling full match Other related full match Sibling or other related with 1 mismatch antigen Cord Blood Haploidentical

Drug:
• 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)

Locations

Country	Name	City	State
Iran, Islamic Republic of	Hematology-Oncology & SCT Research Center	Tehran

Sponsors (1)

Lead Sponsor	Collaborator
Tehran University of Medical Sciences

Country where clinical trial is conducted

Iran, Islamic Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT		1 year	No
Secondary	One year overall survival after allogeneic HSCT		1 year	No
Secondary	One year Progressive Free Survival after allogeneic HSCT		1 year	No
Secondary	Transplantation Related Mortality (TRM) after allogeneic HSCT		1 year	No
Secondary	Acute and chronic GVHD rate after allogeneic HSCT		1 year	No

External Links

•   Hematology-Oncology and Stem Cell Transplantation Research Center