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

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

Baseline Characteristics

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 ]

Show Outcome Measure 1

Serious Adverse Events

Other Adverse Events

More Information

Certain Agreements:

All Principal Investigators ARE employed by the organization sponsoring the study.

Limitations and Caveats

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.

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