Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 11:07 PM
Ignite Modification Date:
2025-12-24 @ 11:07 PM
NCT ID:
NCT00748969
Eligibility 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
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
5 Years
Maximum Age:
17 Years
Study:
NCT00748969
Study Brief:
Protocol Section:
NCT00748969