Status:
TERMINATED
Clinical Trial of Growth Hormone in MPS I, II, and VI
Lead Sponsor:
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Conditions:
Mucopolysaccharidosis I
Mucopolysaccharidosis II
Eligibility:
All Genders
5-17 years
Phase:
PHASE2
PHASE3
Brief 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.
Detailed Description
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...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
November 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2013
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT00748969
Start Date
November 1 2008
End Date
September 1 2013
Last Update
September 5 2018
Active Locations (1)
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1
University of Minnesota
Minneapolis, Minnesota, United States, 55455