Reduced Intensity AlloTransplant For Osteopetrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00638820
• Date of registration: 11/03/2008
• Prospective Registration: No
• Primary sponsor: Masonic Cancer Center, University of Minnesota
• Public title: Reduced Intensity AlloTransplant For Osteopetrosis
• Scientific title: Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft
• Date of first enrolment: September 2007
• Target sample size: 3
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00638820
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Paul Orchard, MD
• Affiliation: Masonic Cancer Center, University of Minnesota

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients eligible for transplantation under this protocol will be <45 years of age,
and will be diagnosed with severe osteopetrosis. This will be defined as having the
following manifestations of the disease.

- Bones that are uniformly markedly dense based on skeletal survey

- No history that would suggest autosomal dominant inheritance

- Evidence of hematologic changes that are attributed to the underlying disease,
including the need for ongoing transfusions, OR

- the presence of progressive anemia or thrombocytopenia, OR a white blood cell
differential with a predominance of immature forms and evidence of extramedullary
hematopoiesis, OR

- persistence of serious infectious complications that are thought to be due to the
abnormal architecture of the bone that are resistant to surgical and medical
interventions.

Exclusion Criteria:

- Patients >45 years of age

- Evidence of hepatic failure

- pulmonary dysfunction sufficient to substantially increase the risk of transplant

- Renal dysfunction with glomerular filtration rate (GFR) <30% of predicted.

- Cardiac compromise sufficient to substantially increase the risk of transplantation

- Severe, stable neurologic impairment.

- Human immunodeficiency virus (HIV) positivity.

- Pregnant or lactating females



Age minimum: N/A
Age maximum: 45 Years
Gender: All

Health Condition(s) or Problem(s) studied

Osteopetrosis

Intervention(s)

Drug: Campath, Busulfan, Clofarabine

Procedure: Total Lymphoid Irradiation

Procedure: Stem Cell or Umbilical Cord Blood Transplantation

Primary Outcome(s)

Number of Patients Achieving Donor Cell Engraftment [Time Frame: Day 100]

Secondary Outcome(s)

Number of Patients With Transplant Related Toxicity [Time Frame: Day 100]

Differential Imaging and Biologic Evaluations [Time Frame: Day 100, 6 months, 1, 2 and 5 years]

Number of Patients With Transplant Related Death [Time Frame: Day 100]

Secondary ID(s)

MT2007-06

0704M06581

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 03/02/2010
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00638820