Clinical Trial of Growth Hormone in MPS I, II, and VI

Stopped early · Phase 2/Phase 3

Conditions studied: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI

In brief

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.

Key facts

Study ID

NCT00748969

Run by

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

People needed

2

Starts

2008-11-01

Expected to finish

2013-09-01

Last updated by the study team

2018-09-05

Who can join

Age: 5 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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 area
• Left ventricular wall thickness greater than or less than 2 standard deviations of normal for body surface area
• More than mild to moderate aortic insufficiency with abdominal aortic run-off
• More than mild to moderate mitral insufficiency with pulmonary hypertension
• Abnormal pulmonary function based on pulmonary function tests within 6 months prior to enrollment:
• abnormal FVC < 80% of predicted for age, gender, and height
• abnormal FEV1 < 80% predicted for age, gender, and height
• abnormal FEV1/FVC
• abnormal oxygen saturation

Where it is running

• University of Minnesota — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.