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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03153319
Other study ID # 31041-01
Secondary ID
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date June 5, 2017
Est. completion date June 2026

Study information

Verified date March 2023
Source Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

This study is a 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. Children and adults diagnosed with MPS I, II or VI, with significant joint restrictions and pain will be randomized to adalimumab treatment or placebo treatment for the first 16 weeks. This will be followed by a 32-week open label adalimumab treatment phase.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 14
Est. completion date June 2026
Est. primary completion date December 2025
Accepts healthy volunteers No
Gender All
Age group 5 Years and older
Eligibility Inclusion Criteria: - Male or female =5 years of age; - Diagnosis of MPS I, II or VI; - Treatment with ERT for =1 year or no treatment with ERT for =1 year; - Weight =15 kg; - Significant bodily pain reported by the CHQ-PF50 or SF-36 (> 1 SD more severe [below] than the general population mean); - = 3 joints with limitations in motion; and Patient or parent/legal guardian is able and willing to provide informed consent. For patients 7 to 17 years of age, assent must also be provided. Exclusion Criteria: - History of HCT less than 2 years prior to enrollment; - Immune suppression therapy less than 1 year prior to enrollment; - Active graft versus host disease; - Current diagnosis or history of lymphoma or other malignancy; - Current active infection; - History of serious opportunistic infection (e.g., bacterial [Legionella and Listeria]; tuberculosis [TB]; invasive fungal infections; or viral, parasitic, and other opportunistic infections); - Positive TB skin test, positive Quantiferon-TB Gold TB test, positive chest X-ray, or a recent exposure to TB - Congestive heart failure defined by an ejection fracture <50% measured by ECHO; - Demyelinating disorders (e.g., central nervous system [CNS] disorders including multiple sclerosis and optic neuritis and peripheral nervous system disorders including Guillain-Barre syndrome); - Hematologic abnormalities (e.g., pancytopenia, aplastic anemia); - Hepatitis B infection (active or chronic carrier); - Latex sensitivity; - Pregnancy or breastfeeding; - Known or suspected allergy to adalimumab or related products; - Participation in simultaneous therapeutic study that involves an investigational study drug or agent within 4 weeks of study enrollment; - Requirement for live vaccine exposure that would be expected to occur during the time frame of the study; or - Any other social or medical condition that the Investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study.

Study Design


Intervention

Drug:
Adalimumab Injection [Humira]
Investigational Drug
Saline Solution for Injection
Placebo Comparator

Locations

Country Name City State
United States The Lundquist Institute at Harbor-UCLA Medical Center Torrance California

Sponsors (1)

Lead Sponsor Collaborator
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Pain - 16 weeks 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 16 weeks
Primary Adalimumab trough Percentage of subjects who achieve a goal trough concentration of adalimumab with every other week dosing 32 weeks
Secondary Joint range-of-motion - 16 weeks Percentage of subjects who achieve a 5 degree or more improvement in joint range-ot-motion in treatment versus placebo at 16 weeks. 16 weeks
Secondary Pain - 52 weeks Mean difference in bodily pain measured by the CHQ-PF50 or the SF-36 at 52 weeks compared to baseline. 52 weeks
Secondary Joint range-of-motion - 52 weeks Percentage of subjects who achieve a 5 degree or more improvement in joint range-ot-motion at 52 weeks compared to baseline. 52 weeks
Secondary Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability Percentage of subjects who develop an AE and/or SAE 52 weeks
See also
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