Clinical Trials Logo
  1. Home
  2. Mucopolysaccharidosis II
  3. NCT00748969

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

Mucopolysaccharidosis II Clinical Trial

Official title:
Phase II/III, Randomized, Clinical Trial of the Effects of Nutropin AQ® on Growth and Bone Metabolism in Children With MPS I, II, and VI and Short Stature

Clinical Trial 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.

Clinical Trial 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, their quality of life is significantly impacted by their skeletal abnormalities (i.e., kyphosis, scoliosis, and genu valgum), contractures, and severe short stature. Here at the University of Minnesota we have seen some promising clinical outcomes in children with MPS IH whom we have treated with human growth hormone (hGH). There are currently no reports in the literature of the impact of treating children with MPS and short stature, with hGH on their growth velocity or characteristic skeletal abnormalities. This study will advance the care of these children by providing data in this yet unexplored area of pediatric medicine with the goal of improving the quality of life for these children by improving height, mobility, and neuropsychological functioning.

This is a Phase II/III randomized, single-center, 12 month clinical trial of growth hormone in male and female participants with MPS I, II, or VI, followed by 12 months open label. Participants with height ≤ -2 SDS for age and gender will be randomized for the first 12 months 1:1 to treatment or no treatment. At the conclusion of the 12 months, all subjects will be offered an additional 12 months of treatment. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00748969
Study type Interventional
Source Los Angeles Biomedical Research Institute
Contact
Status Terminated
Phase Phase 2/Phase 3
Start date November 2008
Completion date September 2013
View Details
See also
  Status Clinical Trial Phase
Withdrawn NCT05238324 - Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II Phase 1
Completed NCT03529786 - Mucopolysaccharidosis Type II Natural History
Recruiting NCT02254863 - UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Phase 1
Terminated NCT01675674 - Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics N/A
Enrolling by invitation NCT06075537 - An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 Phase 2/Phase 3
Recruiting NCT05422482 - A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ Phase 1
Completed NCT00069641 - Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) Phase 2/Phase 3
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Active, not recruiting NCT04348136 - An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II Phase 2/Phase 3
Completed NCT04007536 - A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
Completed NCT00004454 - Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) Phase 1/Phase 2
Active, not recruiting NCT04628871 - Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Recruiting NCT05619900 - Registry of Patients Diagnosed With Lysosomal Storage Diseases
Completed NCT01301898 - To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients Phase 1/Phase 2
Terminated NCT03041324 - Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II Phase 1/Phase 2
Enrolling by invitation NCT05368038 - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Completed NCT03128593 - A Study of JR-141 in Patients With Mucopolysaccharidosis Type II Phase 1/Phase 2
Withdrawn NCT04591834 - Mucopolysaccharidosis Type II Observational
Enrolling by invitation NCT04597385 - Long-term Follow-Up for RGX-121
Active, not recruiting NCT03153319 - Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI Phase 1/Phase 2