A Phase 2/3 interventional study of Somatropin (DNA origin) in Mucopolysaccharidosis I, Mucopolysaccharidosis II and Mucopolysaccharidosis VI, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center. Terminated at 1 site in United States. Open to participants aged 5 Years to 17 Years. Per ClinicalTrials.gov, last updated 2018-09-05.
Sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Phase 2/3, Interventional, and Treatment
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.
71 studies on the registry are indexed under Mucopolysaccharidosis II; 8 are open to participants now.
This study's enrollment of 2 is below the median of 20 across 43 interventional studies indexed under Mucopolysaccharidosis II.
Browse Mucopolysaccharidosis II studies →Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center is the lead sponsor of 67 studies on the registry; 1 is open to participants now.
Of its 11 completed or terminated interventional studies of FDA-regulated products, 5 (45%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Growth hormone treatment arm. Somatropin (DNA origin)
Drug: Somatropin (DNA origin)
No growth hormone treatment in year 1; option for treatment in year 2 open-label period.
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.
Also known as: Nutropin AQ
Change in Growth Velocity From Baseline to End of Study Year 1.
Time frame: 12 months
Safety: Number Drug Related SAEs
Time frame: 1 months
| Milestone | Growth Hormone Treatmen | No Growth Hormone Treatment in Year 1 |
|---|---|---|
| Started | 1 | 1 |
| Completed | 0 | 0 |
| Not completed | 1 | 1 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Withdrew: Withdrawal by subject | 0 | 1 |
| cm/yr | Growth Hormone Treatment | No Growth Hormone Treatment |
|---|---|---|
| Change in Growth Velocity From Baseline to End of Study Year 1. | NA | — |
| Participants | Growth Hormone Treatment | No Growth Hormone Treatment |
|---|---|---|
| Safety: Number Drug Related SAEs | 0 | 0 |
Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GH Treatment | — | 0/1 (0%) | 1/1 (100%) |
| No GH Treatment | — | — | — |
| Event | GH Treatment | No GH Treatment |
|---|---|---|
| HeadacheNervous system disorders | 1/1 | — |
| worsening OSARespiratory, thoracic and mediastinal disorders | 1/1 | — |
| Age, Continuous(years) | Growth Hormone Treatmen | No Growth Hormone Treatment in Year 1 | Total |
|---|---|---|---|
| Mean | 10.9 (10.9 to 10.9) | 13.5 (13.5 to 13.5) | 12.2 (10.9 to 13.5) |
| Age, Categorical(Participants) | Growth Hormone Treatmen | No Growth Hormone Treatment in Year 1 | Total |
|---|---|---|---|
| <=18 years | 1 | 1 | 2 |
| Between 18 and 65 years | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Growth Hormone Treatmen | No Growth Hormone Treatment in Year 1 | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 1 | 1 | 2 |
| Region of Enrollment(participants) | Growth Hormone Treatmen | No Growth Hormone Treatment in Year 1 | Total |
|---|---|---|---|
| United States | 1 | 1 | 2 |
This study is terminated, as verified in Nov 2014. You cannot join it, but the record below documents what was studied.
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Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center