Stem Cell Transplantation for Children Affected With Osteopetrosis

Status Terminated

Clinical Trial Summary

Malignant infantile osteopetrosis (MIOP) is a rare fatal genetic disorder that is characterized by the bone's inability to regulate remodeling. The only curative therapy is hematopoietic stem cell transplantation. Stem cells provided from an HLA identical matched sibling donor is the standard of care, but not feasible for the majority of patients. In addition, due to the potentially rapid progression of this disease, the time to identify a suitable HLA matched unrelated donor is not optimal. Therefore this study is designed to test the hypothesis that children with osteopetrosis can properly engraft hematopoietic stem cells that are donated from a partially matched parental donor, or "haploidentical" stem cell donor that are processed on the investigational device, CliniMACS selection system.

Clinical Trial Description

The primary objective of this trial will be answered strictly by those patients enrolled who receive a haploidentical stem cell donor graft.

Patients with a matched sibling donor will be offered participation in this clinical trial and will receive a standard myeloablative conditioning regimen followed by the infusion of an unmanipulated bone marrow graft. However, data from these transplant recipients will be reported in a descriptive manner only.

Secondary Objectives in this trial include the following:

- To describe the outcome of children with MIOP who receive hematopoietic stem cells from a matched sibling donor or a haploidentical donor utilizing a uniform approach one year from transplant

- To estimate the fraction of children with MIOP who have a genetic defect correlating to the osteopetrosis phenotype

- To assess carrier-state of the genetic mutation in parents with an affected child

- To assess carrier-state of the genetic mutation in siblings of affected children

- To estimate the effect of age at the time of hematopoietic stem cell transplantation on the overall outcome of children with MIOP

- To describe the kinetics of select cytokine expression before and after transplantation ;

Study Design

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

Related Conditions & MeSH terms

Osteopetrosis

Clinical Trial Details

NCT number	NCT00145587
Study type	Interventional
Source	St. Jude Children's Research Hospital
Contact
Status	Terminated
Phase	N/A
Start date	July 2004
Completion date	February 2009

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT01087398` - Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis	Phase 2/Phase 3
Terminated	`NCT00638820` - Reduced Intensity AlloTransplant For Osteopetrosis	Phase 2
Completed	`NCT00004402` - Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis	Phase 3
Recruiting	`NCT01019876` - Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases	Phase 2/Phase 3
Completed	`NCT01199094` - Clinical Assessment of Patients With High Bone Mass Due to Mutation in Lrp5	N/A
Completed	`NCT00043329` - Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis	Phase 4
Recruiting	`NCT03333200` - Longitudinal Study of Neurodegenerative Disorders
Completed	`NCT02666768` - ACTIMMUNE in Intermediate Osteopetrosis	Phase 2
Terminated	`NCT00145886` - rhPTH Therapy for Low Turnover Bone Fragility	Phase 1
Terminated	`NCT00968864` - T-cell Depleted Alternative Donor Transplantation	Phase 2
Completed	`NCT00730314` - Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells	Phase 1/Phase 2
Active, not recruiting	`NCT03301168` - Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant	Phase 1/Phase 2
Terminated	`NCT02065869` - Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant	Phase 1/Phase 2
No longer available	`NCT01200017` - Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.