Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01173016|
Recruitment Status : Unknown
Verified February 2016 by Masonic Cancer Center, University of Minnesota.
Recruitment status was: Active, not recruiting
First Posted : July 30, 2010
Last Update Posted : February 24, 2016
|Condition or disease||Intervention/treatment||Phase|
|Hurler Syndrome||Drug: Laronidase||Phase 1|
The primary objective of this pilot study is to determine the feasibility of giving weekly Laronidase for 2 years in patients with Hurler syndrome after allogeneic transplantation. Specifically, i) the ability to enroll patients, ii) continued compliance throughout the study with drug administration and testing, as well as iii) the relevance of various endpoint determinations will be assessed. The findings of the pilot study will be used to assess whether a subsequent larger study will be conducted.
Secondary Objectives: The secondary objectives of this study will focus on the toxicity associated with weekly Laronidase in this patient population, and the evaluation of a variety of testing and efficacy parameters that would be utilized to measure outcomes and determine benefit in patients treated on a subsequent larger study.
Eligible patients will receive Laronidase as an infusion over several hours once a week at a local site. The dosing of enzyme will be the standard doses recommended by Genzyme.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome|
|Study Start Date :||May 2012|
|Estimated Primary Completion Date :||March 2016|
|Estimated Study Completion Date :||March 2016|
Experimental: Laronidase After Transplantation
Patients with Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant >2 years previously and treated with Laronidase weekly for 2 years after transplant.
Laronidase 0.58 mg/kg intravenously (IV) once a week for a maximum of 2 years
Other Name: Aldurazyme
- Toxicities [ Time Frame: Prior to Starting Enzyme Throughout 2 Years on Therapy ]Adverse events that occur after administration with Laronidase.
- Change in Orthopedic Measures [ Time Frame: From baseline every 6 months for 2 Years ]Measurements from pQCT, bone mineral density, bone mineral content, bone geometry and strength.
- Change in Markers of Bone Metabolism [ Time Frame: From baseline every 6 months through 2 years ]Measurements of Serum osteocalcin (OCN) and bone-specific alkaline phosphatase (BSAP), carboxyterminal telopeptide of type I collagen (ICTP) and carboxyterminal telopeptide α1 chain of type I collagen (CTX)
- Change in Flexibility and Muscle Strength [ Time Frame: From baseline every 6 months through 2 years ]determined by Biodex and Physical Therapy evaluations, including a 6 minute walk study. The Biodex III isokinetic strength testing system will be used to assess strength at the knee and elbow for each participant.
- Change in 0xygen Utilization to Monitor "Fitness" [ Time Frame: From baseline every 6 months through 2 years ]The measurement of peak oxygen uptake (VO2 peak) during exercise testing is considered the best single physiologic indicator of an individual's cardiorespiratory fitness and be also be used to monitor changes in cardiorespiratory fitness over time .
- Change in Neuropsychological Function [ Time Frame: From baseline every 6 months through 2 years ]Continuous scores gathered regarding tests of intelligence, attention, learning and memory, visual processing, fine motor, and quality of life
- Change in Laboratory Measurements [ Time Frame: From baseline every 3 months through 2 years ]including biomarkers, cytokines, markers of inflammation and the development of antibody
- Change in Bone Density [ Time Frame: From baseline to 1 and 2 Years ]Measurements from dual energy x-ray absorptiometry (DXA)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01173016
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|Principal Investigator:||Paul Orchard, MD||Masonic Cancer Center, University of Minnesota|