ClinicalTrials.gov

History of Changes for Study: NCT01521429
Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI
Latest version (submitted November 20, 2019) on ClinicalTrials.gov
  • A study version is represented by a row in the table.
  • Select two study versions to compare. One each from columns A and B.
  • Choose either the "Merged" or "Side-by-Side" comparison format to specify how the two study versions are to be displayed. The Side-by-Side format only applies to the Protocol section of the study.
  • Click "Compare" to do the comparison and show the differences.
  • Select a version's Submitted Date link to see a rendering of the study for that version.
  • The yellow A/B choices in the table indicate the study versions currently compared below. A yellow table row indicates the study version currently being viewed.
  • Hover over the "Recruitment Status" to see how the study's recruitment status changed.
  • Study edits or deletions are displayed in red.
  • Study additions are displayed in green.
Study Record Versions
Version A B Submitted Date Changes
1 January 25, 2012 None (earliest Version on record)
2 September 18, 2012 Sponsor/Collaborators and Study Status
3 June 3, 2013 Study Status, Eligibility, Study Identification, Study Design and Sponsor/Collaborators
4 October 30, 2014 Contacts/Locations, Sponsor/Collaborators, Study Status and Study Identification
5 November 6, 2014 Groups and Interventions, Study Description and Study Status
6 June 23, 2015 Study Status
7 March 9, 2016 Study Status
8 May 3, 2017 Recruitment Status, Study Status, Contacts/Locations and Study Design
9 August 6, 2018 Study Status
10 November 20, 2019 Recruitment Status, Study Status and Study Identification
Comparison Format:

Scroll up to access the controls

Study NCT01521429
Submitted Date:  January 25, 2012 (v1)

Open or close this module Study Identification
Unique Protocol ID: 0906M68810
Brief Title: Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI
Official Title: Longitudinal Study of Bone and Endocrine Disease in Children With MPS I, II, and VI: A Multicenter Study of the Lysosomal Disease Network. AKA Natural History of Bone Disease and the Impact of Growth Hormone Treatment in MPS.
Secondary IDs:
Open or close this module Study Status
Record Verification: November 2011
Overall Status: Recruiting
Study Start: August 2009
Primary Completion:
Study Completion:
First Submitted: November 11, 2011
First Submitted that
Met QC Criteria:
January 25, 2012
First Posted: January 30, 2012 [Estimate]
Last Update Submitted that
Met QC Criteria:
January 25, 2012
Last Update Posted: January 30, 2012 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Lynda Polgreen
Responsible Party: Sponsor-Investigator
Investigator: Lynda Polgreen
Official Title: Assistant Professor
Affiliation: University of Minnesota
Collaborators: UCSF Benioff Children's Hospital Oakland
University of Utah
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: No
Open or close this module Study Description
Brief Summary:

Mucopolysaccharidoses (MPS) I, II, and VI are a group of rare, lysosomal storage disorders in which certain enzymes are missing that are normally required for breaking down specific complex carbohydrates.

Deficiencies in these enzymes result in the build-up of toxic substances in various tissues and cause damage to the body's organs. In the past, children diagnosed with MPS I, II, or VI frequently died in early to late childhood. Recent advances in treatments including hematopoietic cell transplantation (HCT) and enzyme replacement therapy (ERT) have been helping children with these disorders live into adulthood.

Although it appears that poor growth and abnormal bone development persist, this has not yet been well studied. Researchers would like to know more about the long-term course of MPS I, II, and VI. Therefore, the purpose of this study is to learn more about growth, bone disease and endocrine dysfunction in children with MPS I, II and VI over time. This information is needed to:

  1. determine long-term benefits and limitations of current treatments including HCT or ERT
  2. direct development of further treatment options
  3. improve clinical care for children affected by the disorders
Detailed Description:
Open or close this module Conditions
Conditions: Mucopolysaccharidoses
Keywords:
Open or close this module Study Design
Study Type: Observational
Observational Study Model: Cohort
Time Perspective: Prospective
Biospecimen Retention: Samples With DNA
Biospecimen Description: Serum, plasma, white cells for DNA extraction, and urine will be retained.
Enrollment: 50 [Anticipated]
Number of Groups/Cohorts 1
Open or close this module Groups and Interventions
Groups/Cohorts Interventions
MPS I, II, or VI
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Annual change in dual energy x-ray absorptiometry (DXA)
[ Time Frame: baseline, year 1, year 2, year 3 ]

Measurement of bone density and body composition
Secondary Outcome Measures:
1. Annual change in Peripheral quantitative computer tomography (pQCT)
[ Time Frame: baseline, year 1, year 2, year 3 ]

Measurement of volumetric bone density, bone geometry, bone strength, and muscle fat
2. Annual change in Biomarkers of bone remodeling
[ Time Frame: baseline, year 1, year 2, year 3 ]

Measurements of bone turnover
3. Annual change in Biodex
[ Time Frame: baseline, year 1, year 2, year 3 ]

Measurement of muscle strength
4. Annual change in Endocrine function tests
[ Time Frame: baseline, year 1, year 2, year 3 ]

Thyroid function, growth factor levels, pubertal hormones, vitamin D
5. Annual change in growth measurements
[ Time Frame: baseline, year 1, year 2, year 3 ]

sitting and standing heights, arm and tibial length
Open or close this module Eligibility
Study Population: community sample
Sampling Method: Non-Probability Sample
Minimum Age: 5 Years
Maximum Age: 16 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  • Diagnosis of MPS I, II, or VI
  • Ability to travel to study center for evaluations.
  • Age ≥ 5 years and < 18 years: age at entry into study must be ≥5 years and ≤16 years to ensure a minimum of 2 study visits.

Exclusion Criteria:

  • Pregnancy (will be determined at each study visit)
  • Participation in any other study within the past 12 months which would result in increasing the child's radiation exposure above 500 mrem for the calendar year.
  • Participants who cannot comply with study procedures or have other factors that would inhibit their participation as determined by the PI's discretion.
Open or close this module Contacts/Locations
Central Contact Person: Jane Kennedy, RN
Telephone: 612-626-5609
Email: kenne634@umn.edu
Central Contact Backup: Lynda Polgreen, MD, MS
Telephone: 612-624-4459
Email: polgr001@umn.edu
Study Officials: Lynda E Polgreen, MD, MS
Principal Investigator
University of Minnesota
Locations: United States, California
Children's Hospital & Research Center Oakland
[Recruiting]
Oakland, California, United States, 94609
Contact:Contact: Ellen Fung, PhD 510-428-3885 Ext. 4939 EFung@mail.cho.org
Contact:Principal Investigator: Ellen Fung, PhD, RD, CCD
United States, Minnesota
University of Minnesota
[Recruiting]
Minneapolis, Minnesota, United States, 55454
Contact:Contact: Jane Kennedy, RN 612-626-5609 kenne634@umn.edu
Contact:Principal Investigator: Lynda Polgreen, MD, MS
United States, Utah
University of Utah
[Recruiting]
Salt Lake City, Utah, United States, 84108
Contact:Contact: Lisa Smith 801-581-2764 lisa.smith@hsc.utah.edu
Contact:Principal Investigator: David Viskochil, MD
Contact:Sub-Investigator: David Stevenson, MD, PhD
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Citations:
Links:
Available IPD/Information:

Scroll up to access the controls Scroll to the Study top

U.S. National Library of Medicine | U.S. National Institutes of Health | U.S. Department of Health & Human Services