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Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis

T

Tehran University of Medical Sciences

Status and phase

Unknown
Phase 3
Phase 2

Conditions

Osteopetrosis

Treatments

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

Study type

Interventional

Funder types

Other

Identifiers

NCT01087398
HORCSCT-0905

Details and patient eligibility

About

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.

Enrollment

10 estimated patients

Sex

All

Ages

Under 5 years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Single Group Assignment

Masking

None (Open label)

10 participants in 1 patient group

Intervention
Experimental group
Treatment:
Procedure: Stem Cell Transplantation
Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)
Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)

Trial contacts and locations

1

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Central trial contact

Amir Ali Hamidieh, MD

Data sourced from clinicaltrials.gov

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