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NCT00004454 · ClinicalTrials.gov registry record · Phase 1
Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
A Phase 1 study of Mucopolysaccharidosis II, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- Completed
- Registry status
- Phase 1
- Development phase
- 2
- Enrollment target
- 1
- Study location
NCT00004454 is a Phase 1 study of Mucopolysaccharidosis II that has completed, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The registered enrollment target is 2 participants, below the 73-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target (97% lower). The trial reports 1 study location across 1 state.
The verdict
NCT00004454, a Phase 1 study of Mucopolysaccharidosis II, has completed, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 2 participants
- Enrollment target
- 1
- Study location
Study Summary
OBJECTIVES: I. Evaluate the safety and feasibility of treating mucopolysaccharidosis II (mild Hunter syndrome) by lymphocyte gene therapy. II. Determine the levels of iduronate-2-sulfatase enzyme in these patients attained by infusing increasing doses of lymphocytes transduced with a retroviral vector designed for insertion and expression of this iduronate-2-sulfatase gene (L2SN). III. Determine the duration of survival of these transduced cells in these patients. IV. Determine whether monthly infusion of L2SN-transduced lymphocytes accomplishes metabolic correction (as measured by glycosaminoglycan excretion), decrease in liver or spleen volume, any therapeutic effect upon cardiac and pulmonary dysfunction, or any other effects from treatment.
Conditions Studied
Interventions
- GENETIC lymphocyte gene therapy
Study Locations (1)
Minnesota
- University of Minnesota Medical School - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 2 participants |
| Start Date | 1996-10 |
| Phase | Phase 1 |
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)305 total trials
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00004454
The ClinicalTrials.gov registry entry for NCT00004454 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 2 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 73-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target (97% lower). The listed sponsor is Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Mucopolysaccharidosis II appearing as the primary indexed condition, and to 1 intervention - of which lymphocyte gene therapy is the first listed.
NCT00004454 reports 1 study location spanning 1 distinct geographic area - top geographies include Minnesota.
Frequently Asked Questions
What is clinical trial NCT00004454 about?
NCT00004454 is a clinical study titled "Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)". OBJECTIVES: I. Evaluate the safety and feasibility of treating mucopolysaccharidosis II (mild Hunter syndrome) by lymphocyte gene therapy. II. Determine the levels of iduronate-2-sulfatase enzyme in these patients attained by infusing increasing doses of lymphocytes transduced with a retroviral vec...
What is the current status of trial NCT00004454?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 2 participants. The study started on 1996-10.
What conditions does trial NCT00004454 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis II.
What interventions are being tested in trial NCT00004454?
The interventions under investigation include: lymphocyte gene therapy (GENETIC).
Who is sponsoring clinical trial NCT00004454?
This trial is sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00004454 being conducted?
This trial has 1 study location across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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