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Trial record 1 of 1 for:    NCT00775931
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Allogeneic Transplantation For Severe Osteopetrosis

This study is currently recruiting participants.
Verified October 2012 by Masonic Cancer Center, University of Minnesota
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00775931
First received: October 16, 2008
Last updated: October 30, 2012
Last verified: October 2012
History of Changes
  Purpose

The purpose of this research is to explore what we believe may be a safer and more effective means of performing stem cell transplantation in patients with Osteopetrosis, using chemotherapy and radiation designed to bring about engraftment and lessen transplant mortality. Prior multi-institutional data in past studies found that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we utilize a reduced intensity design of pre-transplant drugs to try to make transplants safer for this disease, as well as to provide a second infusion of stem cells in patients with matched related or unrelated donors.


Condition Intervention Phase
Severe Osteopetrosis
 Procedure: Stem Cell Transplantation
Drug: Conditioning for transplantation-donor graft
Radiation: Total Lymphoid Irradiation
Drug: Conditioning for cord blood transplant
 Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Masonic Cancer Center, University of Minnesota:

Primary Outcome Measures:
  • Estimate the rate of donor engraftment for patients treated by hematopoietic stem cell transplantation [ Time Frame: Day 100 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Estimate Peri-transplant mortality (deaths) [ Time Frame: day 100 ] [ Designated as safety issue: Yes ]
  • Transplant related toxicity [ Time Frame: Day 100 post transplant ] [ Designated as safety issue: Yes ]
  • Graft-versus-host disease (incidence and severity) [ Time Frame: after transplantation ] [ Designated as safety issue: Yes ]
  • Tolerance of Campath-1H administration [ Time Frame: During study ] [ Designated as safety issue: Yes ]
  • Clinical Disease Monitoring [ Time Frame: post transplant ] [ Designated as safety issue: No ]

Estimated Enrollment: 23
Study Start Date: October 2008
Estimated Study Completion Date: October 2015
Estimated Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Patients with Donor Grafts
This group includes patients that are recipients of unrelated or matched related donor grafts (both peripheral blood and marrow) with a second CD34 cell infusion administered on Day 42.
 Procedure: Stem Cell Transplantation
Undergo allogeneic hematopoietic stem cell transplantation using marrow, peripheral blood or cord blood grafts
Other Names:
  • BMT, HSCT, Bone Marrow Transplant
  • UCBT
Drug: Conditioning for transplantation-donor graft
The pre-transplant conditioning will include: Campath-1H, Busulfan, Fludarabine (marrow and peripheral blood)
Other Name: Busulfex, Fludara, Campath
Radiation: Total Lymphoid Irradiation
Dose 500 cGy via anteroposterior (AP) and posteroanterior(PA) fields (250 cGy AP and 250 cGy PA).
Other Name: TLI
Active Comparator: Patients With Cord Blood Transplant
This group includes patients who received cord blood transplants with more intensive chemotherapy regimen.
 Procedure: Stem Cell Transplantation
Undergo allogeneic hematopoietic stem cell transplantation using marrow, peripheral blood or cord blood grafts
Other Names:
  • BMT, HSCT, Bone Marrow Transplant
  • UCBT
Radiation: Total Lymphoid Irradiation
Dose 500 cGy via anteroposterior (AP) and posteroanterior(PA) fields (250 cGy AP and 250 cGy PA).
Other Name: TLI
Drug: Conditioning for cord blood transplant
The pre-transplant conditioning will include: Campath-1H, Busulfan, and Cyclophosphamide
Other Name: Busulfex, Campath, Cytoxan

Detailed Description:

This revised transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol and a second infusion of stem cells on day 42, 2) the mortality associated with transplant by day 100, 3) patient outcomes, based on differential imaging and biologic evaluations prior to transplantation and at designated points after transplantation (day 100, 6 months, 1, 2 and 5 years). Additional biologic studies will include microarray analysis, and evaluation of blood parameters and genes that may be important in the disease process. In older patients, studies to evaluation osteoclast differentiation and function will also be offered.

  Eligibility

Ages Eligible for Study:   up to 45 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

    1. Bones that are uniformly markedly dense based on skeletal survey
    2. No history that would suggest autosomal dominant inheritance
    3. 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 significantly 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00775931

Contacts
Contact: Paul Orchard, MD 612-626-1926 orcha001@umn.edu
Contact: Teresa Kivisto, RN 612-273-2800 tkivist1@fairview.org

Locations
United States, Minnesota
University of MInnesota, Fairview Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Patricia Kleinke, RN     612-273-0857     pkleink1@fairview.org    
Contact: Cyndie Hibbs     612-625-8542        
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Paul Orchard, MD Masonic Cancer Center, University of Minnesota
  More Information

No publications provided

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00775931     History of Changes
Other Study ID Numbers: MT2008-20, 0808M42261
Study First Received: October 16, 2008
Last Updated: October 30, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by Masonic Cancer Center, University of Minnesota:
osteopetrosis

Additional relevant MeSH terms:
Osteopetrosis
Osteosclerosis
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Busulfan
Alemtuzumab
Immunosuppressive Agents
 Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Therapeutic Uses
Myeloablative Agonists

ClinicalTrials.gov processed this record on May 16, 2013