Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00920647 · ClinicalTrials.gov registry record · Phase 1
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
A Phase 1 study of Hunter Syndrome, sponsored by Shire.
- Completed
- Registry status
- Phase 1
- Development phase
- 16
- Enrollment target
- 3
- Study locations
NCT00920647: Completed Phase 1 study of Hunter Syndrome, sponsored by Shire.
NCT00920647 is a Phase 1 study of Hunter Syndrome that has completed, run by Shire. The registered enrollment target is 16 participants, below the 353-participant average among 5 other Hunter Syndrome trials with a reported enrollment target (95% lower). The trial reports 3 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00920647, a Phase 1 study of Hunter Syndrome, has completed, sponsored by Shire.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 16 participants
- Enrollment target
- 3
- Study locations
Study Summary
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously. A new formulation of idursulfase, idursulfase-IT, that differs from that of the intravenous (IV) formulation, Elaprase, has been developed to be suitable for delivery into the cerebrospinal fluid (CSF) via intrathecal administration. This Phase I/II study is designed to obtain necessary safety and exposure data, as well as secondary and exploratory outcome measures, to be interpreted and used in the design of subsequent clinical trials.
Conditions Studied
Interventions
- OTHER Control
- DRUG Idursulfase IT (1 mg)
- DRUG Idursulfase IT (10 mg)
- DRUG Idursulfase IT (30 mg)
Study Locations (3)
Other
- Birmingham Children's Hospital - Birmingham
- Birmingham Children's Hospital NHS Foundation Trust - Birmingham
North Carolina
- University of North Carolina at Chapel Hill - Chapel Hill
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2009-11-18 |
| Est. Completion | 2012-10-29 |
| Phase | Phase 1 |
What the finished NCT00920647 record still lists
NCT00920647 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 353-participant average among 5 other Hunter Syndrome trials with a reported enrollment target (95% lower).
The record links to 1 condition, with Hunter Syndrome appearing as the primary indexed condition, and to 4 interventions - of which Control is the first listed.
NCT00920647 reports a single indexed study location in Other, North Carolina.
Frequently Asked Questions
What is clinical trial NCT00920647 about?
NCT00920647 is a clinical study titled "A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®". Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously. A new formulation of idursulfase, idursulfase-IT, that differs from that of the intravenous (IV) formulation, Elaprase, has been developed to be ...
What is the current status of trial NCT00920647?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 16 participants. The study started on 2009-11-18. Estimated completion is 2012-10-29.
What conditions does trial NCT00920647 study?
This clinical trial studies the following conditions: Hunter Syndrome.
What interventions are being tested in trial NCT00920647?
The interventions under investigation include: Control (OTHER), Idursulfase IT (1 mg) (DRUG), Idursulfase IT (10 mg) (DRUG), Idursulfase IT (30 mg) (DRUG).
Who is sponsoring clinical trial NCT00920647?
This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00920647 being conducted?
This trial has 3 study locations across North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Hunter Syndrome
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.
-
An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
COMPLETED · Phase 1
-
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
ACTIVE NOT RECRUITING · Phase 2
-
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
COMPLETED · Phase 2
-
Hunter Outcome Survey (HOS)
COMPLETED
-
Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
COMPLETED
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
- NCT02422641 · 16 participants · Phase 2
Prospective Evaluation Of High-Dose Systemic Methotrexate In Patients With Breast Cancer And Leptomeningeal Metastasis
- NCT02851758 · 16 participants · NA
Transplantation of Autologously Derived Mitochondria Following Ischemia
- NCT03291028 · 16 participants
Analysis of Primary and Metastatic Tumors in Patients With Renal Cell Carcinoma and Urothelial Carcinoma
- NCT03425526 · 16 participants · Phase 1
Donor T Cell Therapy in Treating Immunocompromised Patients With Adenovirus-Related Disease
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