Clinical Trial of Growth Hormone in MPS I, II, and VI
Drug: Somatropin (DNA origin)
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II/III, Randomized, Clinical Trial of the Effects of Nutropin AQ® on Growth and Bone Metabolism in Children With MPS I, II, and VI and Short Stature|
- Change in Growth Velocity From Baseline to End of Study Year 1. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Safety of Nutropin AQ® in Children With MPS I, II, and VI. Safety Endpoints Are: • Physical Examinations • Fundoscopic Examinations • Adverse Events • Radiographic Examinations of Spine and Lower Extremities [ Time Frame: 1 months ] [ Designated as safety issue: Yes ]Three days after starting treatment with Nutropin AQ the subjects mother reported that the study subject developed headache, increased "noisy and more labored" breathing (history of obstructive sleep apnea treated with BIPAP), and fatigue with watery eyes. Symptoms resolved with discontinuation of Nutropin AQ and before evaluation by physician. Subject withdrew from the study due to these adverse effects.
- Change in Bone Mineral Density, Content, and Strength by DXA and pQCT, and Change in Serum Markers of Bone Metabolism. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Change in Mobility Measured by Force and Gait Measurements. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Change in Muscle Strength Measured by Biodex and Hand Grip Dynamometer. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
- Change in Neuropsychological Functioning and Brain Volumetrics. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
|Study Start Date:||November 2008|
|Study Completion Date:||September 2013|
|Primary Completion Date:||September 2013 (Final data collection date for primary outcome measure)|
Experimental: Growth hormone treatmen
Growth hormone treatment arm. Somatropin (DNA origin)
Drug: Somatropin (DNA origin)
The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.
Other Name: Nutropin AQ
No Intervention: No growth hormone treatment in year 1
No growth hormone treatment in year 1; option for treatment in year 2 open-label period.
Although children with MPS I, II, and VI who are treated with Hematopoietic Cell Transplantation (HCT) and/or enzyme replacement therapy (ERT) are living into adulthood with good cognitive development, their quality of life is significantly impacted by their skeletal abnormalities (i.e., kyphosis, scoliosis, and genu valgum), contractures, and severe short stature. Here at the University of Minnesota we have seen some promising clinical outcomes in children with MPS IH whom we have treated with human growth hormone (hGH). There are currently no reports in the literature of the impact of treating children with MPS and short stature, with hGH on their growth velocity or characteristic skeletal abnormalities. This study will advance the care of these children by providing data in this yet unexplored area of pediatric medicine with the goal of improving the quality of life for these children by improving height, mobility, and neuropsychological functioning.
This is a Phase II/III randomized, single-center, 12 month clinical trial of growth hormone in male and female participants with MPS I, II, or VI, followed by 12 months open label. Participants with height ≤ -2 SDS for age and gender will be randomized for the first 12 months 1:1 to treatment or no treatment. At the conclusion of the 12 months, all subjects will be offered an additional 12 months of treatment.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00748969
|United States, Minnesota|
|University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|Principal Investigator:||Lynda E Polgreen, M.D.||University of Minnesota - Clinical and Translational Science Institute|