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

• Status: Terminated
• Trial end date: 2013-09-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Los Angeles Biomedical Research Institute; Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Treatments: Hormones

Criteria

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 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