Clinical Trial of Growth Hormone in MPS I, II, and VI (NCT00748969) | Clinical Trial Compass
TerminatedPhase 2/3
Clinical Trial of Growth Hormone in MPS I, II, and VI
Stopped: Insufficient recruitment.
United States2 participantsStarted 2008-11
Plain-language summary
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.
Who can participate
Age range5 Years – 17 Years
SexALL
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Inclusion Criteria:
* A parent or legally authorized representative must provide written informed consent and comply with study assessments for the full duration of the study.
* Chronologic age ≥ 5 years and bone age ≤12 years
* Diagnosis of MPS I, II, or VI
* Height ≤ -2 SDS for age and gender
* Ability to travel to study center for evaluations.
* Ability of the participant to cooperate with study procedures, to notify a guardian of symptoms, and provide assent for participation in the study.
Exclusion Criteria:
* History of treatment with hGH
* Untreated pituitary deficiency
* Pregnancy (positive urine pregnancy test) prior to enrollment in the study
* Participation in another simultaneous medical intervention trial
* Patients with closed epiphysis
* Active neoplasm
* Orthopedic procedure of the femur within the last 6 months.
* Known or suspected allergy to trial product or related products.
* Structural lesion on brain MRI resulting in brain compression
* Any other social or medical condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study.
* Obstructive sleep apnea without BiPAP or tonsillectomy/adenoidectomy treatment.
* CNS shunt.
* Abnormal cardiac function based on echocardiogram within 6 months prior to enrollment :
* Ejection fraction less than 50%
* Left ventricular chamber size greater than or less than 2 standard deviations of normal for body surface are…
What they're measuring
1
Change in Growth Velocity From Baseline to End of Study Year 1.
Timeframe: 12 months
Trial details
NCT IDNCT00748969
SponsorLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center