Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03153319 · ClinicalTrials.gov registry record · Phase 1
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
A Phase 1 study of Mucopolysaccharidosis II and Mucopolysaccharidosis VI, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
- Active
- Registry status
- Phase 1
- Development phase
- 14
- Enrollment target
- 1
- Study location
NCT03153319: Active Phase 1 study of Mucopolysaccharidosis II and Mucopolysaccharidosis VI, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
NCT03153319 is a Phase 1 study of Mucopolysaccharidosis II and Mucopolysaccharidosis VI that is active but no longer recruiting, run by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center. The registered enrollment target is 14 participants, below the 72-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target (81% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03153319, a Phase 1 study of Mucopolysaccharidosis II and Mucopolysaccharidosis VI, is active but no longer recruiting, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 14 participants
- Enrollment target
- 1
- Study location
Study Summary
Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.
Primary Outcome
Mean difference in bodily pain measured by the Children's Health Questionnaire - Parent Form 50 (CHQ-PF50) or the Medical Outcomes Study - Short Form 36 (SF-36) in treatment versus placebo at 16 weeks
Conditions Studied
Interventions
- DRUG Saline Solution for Injection
- DRUG Adalimumab Injection [Humira]
Study Locations (1)
California
- The Lundquist Institute at Harbor-UCLA Medical Center - Torrance
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 14 participants |
| Start Date | 2017-06-05 |
| Est. Completion | 2027-02 |
| Phase | Phase 1 |
What the registry record for NCT03153319 still lists
NCT03153319 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 14 participants, a relatively small participant target, below the 72-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target (81% lower).
The record links to 3 conditions, with Mucopolysaccharidosis II appearing as the primary indexed condition, and to 2 interventions - of which Saline Solution for Injection is the first listed.
NCT03153319 reports a single indexed study location in California.
Frequently Asked Questions
What is clinical trial NCT03153319 about?
NCT03153319 is a clinical study titled "Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI". Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.
What is the current status of trial NCT03153319?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 14 participants. The study started on 2017-06-05. Estimated completion is 2027-02.
What conditions does trial NCT03153319 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis II, Mucopolysaccharidosis VI, Mucopolysaccharidosis I.
What interventions are being tested in trial NCT03153319?
The interventions under investigation include: Saline Solution for Injection (DRUG), Adalimumab Injection [Humira] (DRUG).
Who is sponsoring clinical trial NCT03153319?
This trial is sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, which has 55 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03153319 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Mucopolysaccharidosis II
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.
-
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
RECRUITING · Phase 1
-
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
ACTIVE NOT RECRUITING · 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)
COMPLETED · Phase 1
-
Registry of Patients Diagnosed With Lysosomal Storage Diseases
RECRUITING
-
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
RECRUITING · Phase 2
-
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
ACTIVE NOT RECRUITING
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
- NCT02523092 · 14 participants · Phase 4
Use of CXCL9 as a Biomarker of Acthar Efficacy
- NCT04263285 · 14 participants · NA
Treatment of Depression Post-SCI
- NCT04743271 · 14 participants · NA
Food and Circadian Timing
- NCT05065671 · 14 participants · Phase 1
Microbiome Derived Metabolism and Pharmacokinetics
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
- NCT06358040 · started 2026-10 · NA
Opioid Dispenser for Microdiscectomy/Laminectomy