Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

May 31, 2012 updated by: Tehran University of Medical Sciences
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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

10

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Iran, Islamic Republic of
      • Tehran, Iran, Islamic Republic of, 14114
        • Recruiting
        • Hematology-Oncology & SCT Research Center
        • Sub-Investigator:
          • Mahdi Jalili, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 5 years (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: 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
Other Names:
  • Systemic chemotherapy
Procedure: 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
Other Names:
  • HSCT
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)
Other Names:
  • Graft-versus-host disease (GVHD) prophylaxis

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT
Time Frame: 1 year
1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
One year overall survival after allogeneic HSCT
Time Frame: 1 year
1 year
One year Progressive Free Survival after allogeneic HSCT
Time Frame: 1 year
1 year
Transplantation Related Mortality (TRM) after allogeneic HSCT
Time Frame: 1 year
1 year
Acute and chronic GVHD rate after allogeneic HSCT
Time Frame: 1 year
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Tehran University of Medical Sciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2009

Primary Completion (ANTICIPATED)

November 1, 2012

Study Completion (ANTICIPATED)

December 1, 2012

Study Registration Dates

First Submitted

March 13, 2010

First Submitted That Met QC Criteria

March 15, 2010

First Posted (ESTIMATE)

March 16, 2010

Study Record Updates

Last Update Posted (ESTIMATE)

June 4, 2012

Last Update Submitted That Met QC Criteria

May 31, 2012

Last Verified

May 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HORCSCT-0905

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Osteopetrosis

Clinical Trials on Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Dominican Republic

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe