Stem Cell Transplantation for Children Affected With Osteopetrosis

This study has been terminated.
(Due to the principal investigator having left the institution.)
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00145587
First received: September 1, 2005
Last updated: July 26, 2012
Last verified: January 2011
Results First Received: June 22, 2011  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Osteopetrosis
Interventions: Procedure: Stem Cell Transplantation
Device: Miltenyi Biotec CliniMACS
Drug: Systemic chemotherapy and antibodies

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
OPBMT2 was activated, July 2004. From September, 2004 through February, 2008, six transplant participants, five donors, and four genetic testing participants were recruited and enrolled on the study.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Of the fifteen enrollments, the donors and genetic-testing participants did not receive transplants. Of the six transplant participants, five where eligible for and received a haploidentical hematopoietic stem cell transplant (HSCT) and one received a sibling donor HSCT.

Reporting Groups
  Description
Haplo Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT).
Sibling Genetic testing

Participant Flow:   Overall Study
    Haplo     Sibling  
STARTED     5     1  
COMPLETED     5     1  
NOT COMPLETED     0     0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Haplo Patients to receive a haploidentical hematopoietic stem cell transplantation (HSCT).
Sibling Genetic testing
Total Total of all reporting groups

Baseline Measures
    Haplo     Sibling     Total  
Number of Participants  
[units: participants]
  5     1     6  
Age  
[units: participants]
     
<=18 years     5     1     6  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Gender  
[units: participants]
     
Female     1     1     2  
Male     4     0     4  



  Outcome Measures

1.  Primary:   Engraftment   [ Time Frame: 100 days post-transplant ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
This study closed prior to completion of enrollment due to the principal investigator having left the institution.


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Kimberly Kasow, DO
Organization: St. Jude Children's Research Hospital
phone: 901-595-3300
e-mail: kimberly_kasow@med.unc.edu


No publications provided


Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00145587     History of Changes
Other Study ID Numbers: OPBMT2
Study First Received: September 1, 2005
Results First Received: June 22, 2011
Last Updated: July 26, 2012
Health Authority: United States: Food and Drug Administration