Mucopolysaccharidosis II Clinical Trial
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
The purpose of this study is to determine whether growth hormone is a safe and effective treatment for short stature in children with Mucopolysaccharidosis type I, II, and VI.
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.
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