Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

NCT01087398 | Unknown Phase 2 DMC

• 1 other identifier: HORCSCT-0905
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Osteopetrosis

Keywords

Osteopetrosis; HSCT

Outcome Measures

Primary Outcomes (1)

• Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT

  1 year

Secondary Outcomes (4)

• One year overall survival after allogeneic HSCT

  1 year

• One year Progressive Free Survival after allogeneic HSCT

  1 year

• Transplantation Related Mortality (TRM) after allogeneic HSCT

  1 year

• Acute and chronic GVHD rate after allogeneic HSCT

  1 year

Study Arms (1)

Intervention

EXPERIMENTAL

Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen); Procedure: Stem Cell Transplantation; Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)

Interventions

Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen) 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

Also known as: Systemic chemotherapy

Intervention

Stem Cell Transplantation 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

Also known as: HSCT

Intervention

Cyclosporin, Methotrexate (GVHD prophylaxis) 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)

Also known as: Graft-versus-host disease (GVHD) prophylaxis

Intervention

Eligibility Criteria

• Age: Up to 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Tehran University of Medical Sciences: lead

Study Sites (1)

Hematology-Oncology & SCT Research Center

Tehran, Tehran Province, 14114, Iran

RECRUITING

MeSH Terms

Conditions

Osteopetrosis

Interventions

Busulfan; Cyclophosphamide; thymoglobulin; fludarabine; Transplantation Conditioning; Neoadjuvant Therapy; Stem Cell Transplantation; Cyclosporine; Methotrexate

Condition Hierarchy (Ancestors)

Osteosclerosis; Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Immunosuppression Therapy; Immunotherapy; Immunomodulation; Biological Therapy; Therapeutics; Immunologic Techniques; Investigative Techniques; Combined Modality Therapy; Cell Transplantation; Cell- and Tissue-Based Therapy; Transplantation; Surgical Procedures, Operative; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins; Aminopterin; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Amir Ali Hamidieh, MD

  Hematology-Oncology and SCT Research Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Amir Ali Hamidieh, MD

CONTACT

84902645; aahamidieh@sina.tums.ac.ir

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 13, 2010
• First Posted: March 16, 2010
• Study Start: September 1, 2009
• Primary Completion: November 1, 2012
• Study Completion: December 1, 2012
• Last Updated: June 4, 2012

Record last verified: 2012-05

Locations

IR (1)