Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an allogeneic transplant.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome|
- Toxicities [ Time Frame: Prior to Starting Enzyme Throughout 2 Years on Therapy ] [ Designated as safety issue: Yes ]Adverse events that occur after administration with Laronidase.
- Change in Orthopedic Measures [ Time Frame: From baseline every 6 months for 2 Years ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]including biomarkers, cytokines, markers of inflammation and the development of antibody
- Change in Bone Density [ Time Frame: From baseline to 1 and 2 Years ] [ Designated as safety issue: Yes ]Measurements from dual energy x-ray absorptiometry (DXA)
|Study Start Date:||May 2012|
|Estimated Study Completion Date:||March 2015|
|Estimated Primary Completion Date:||March 2015 (Final data collection date for primary outcome measure)|
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
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.
|Contact: Paul Orchard, MDfirstname.lastname@example.org|
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Paul Orchard, MD 612-626-2313 email@example.com|
|Principal Investigator: Paul Orchard, MD|
|Principal Investigator:||Paul Orchard, MD||Masonic Cancer Center, University of Minnesota|