Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT01509768 · ClinicalTrials.gov registry record
Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
A clinical trial of Sanfilippo Syndrome Type B, sponsored by Shire.
- Completed
- Registry status
- 19
- Enrollment target
- 4
- Study locations
NCT01509768: Completed study of Sanfilippo Syndrome Type B, sponsored by Shire.
NCT01509768 is a study of Sanfilippo Syndrome Type B that has completed, run by Shire. The registered enrollment target is 19 participants. The trial reports 4 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01509768, a study of Sanfilippo Syndrome Type B, has completed, sponsored by Shire.
- COMPLETED
- Registry status
- 19 participants
- Enrollment target
- 4
- Study locations
Study Summary
The purpose of this study is to evaluate the natural course of disease progression in Mucopolysaccharidosis Type III (MPS IIIB) patients who are untreated to identify potential surrogate endpoints that may be utilized in future treatment trials of MPS IIIB using predefined assessments including standardized clinical, biochemical, neurocognitive, developmental, and imaging measures.
Conditions Studied
Study Locations (4)
Other
- Johannes Gutenberg University Medical Center - Mainz
- Great Ormond Street Hospital for Children - London
California
- Children's Hospital and Research Center - Oakland
Minnesota
- University of Minnesota - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2012-04-09 |
| Est. Completion | 2013-10-10 |
What the finished NCT01509768 record still lists
NCT01509768 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 19 participants, a relatively small participant target.
The record links to 1 condition, with Sanfilippo Syndrome Type B appearing as the primary indexed condition, and to 0 interventions.
NCT01509768 reports a single indexed study location in Other, California, Minnesota.
Frequently Asked Questions
What is clinical trial NCT01509768 about?
NCT01509768 is a clinical study titled "Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)". The purpose of this study is to evaluate the natural course of disease progression in Mucopolysaccharidosis Type III (MPS IIIB) patients who are untreated to identify potential surrogate endpoints that may be utilized in future treatment trials of MPS IIIB using predefined assessments including stan...
What is the current status of trial NCT01509768?
This trial is currently completed. The enrollment target is 19 participants. The study started on 2012-04-09. Estimated completion is 2013-10-10.
What conditions does trial NCT01509768 study?
This clinical trial studies the following conditions: Sanfilippo Syndrome Type B.
Who is sponsoring clinical trial NCT01509768?
This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01509768 being conducted?
This trial has 4 study locations across California, Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Sanfilippo Syndrome Type B
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT03207464 · 19 participants · Phase 1
Molecular Imaging of NET Using [C-11]MRB-PET in MS
- NCT04318717 · 19 participants · Phase 2
Pembrolizumab and Hypofractionated Radiation Therapy for the Treatment of Mucosal Melanoma
- NCT05103904 · 19 participants · Phase 2
Lenvatinib for the Treatment of Recurrent Hepatocellular Carcinoma After Liver Transplant
- NCT05607407 · 19 participants · Phase 2
Methimazole in Patients With Progressive Glioblastoma
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI