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Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II

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ClinicalTrials.gov Identifier: NCT03153319
Recruitment Status : Recruiting
First Posted : May 15, 2017
Last Update Posted : August 15, 2017
Sponsor:
Information provided by (Responsible Party):
Los Angeles Biomedical Research Institute

Brief 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 or II.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis I Mucopolysaccharidosis II Drug: Adalimumab Injection [Humira] Drug: Saline Solution for Injection Phase 1 Phase 2

Detailed 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 or II. Children and adults diagnosed with MPS I or II, 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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 14 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase 1/2 Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I and II
Actual Study Start Date : June 5, 2017
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020


Arm Intervention/treatment
Experimental: Adalimumab
20 mg subQ every other week (weight 15to <30 kg) 40 mg subQ every other week (weight ≥30 kg). Non-responders will be escalated to weekly dosing.
Drug: Adalimumab Injection [Humira]
Investigational Drug

Placebo Comparator: Placebo
Saline placebo comparator
Drug: Saline Solution for Injection
Placebo Comparator

Experimental: Open-label adalimumab
Open-label extension of adalimumab dose
Drug: Adalimumab Injection [Humira]
Investigational Drug




Primary Outcome Measures :
  1. Pain - 16 weeks [ Time Frame: 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

  2. Adalimumab trough [ Time Frame: 32 weeks ]
    Percentage of subjects who achieve a goal trough concentration of adalimumab with every other week dosing


Secondary Outcome Measures :
  1. Joint range-of-motion - 16 weeks [ Time Frame: 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.

  2. Pain - 52 weeks [ Time Frame: 52 weeks ]
    Mean difference in bodily pain measured by the CHQ-PF50 or the SF-36 at 52 weeks compared to baseline.

  3. Joint range-of-motion - 52 weeks [ Time Frame: 52 weeks ]
    Percentage of subjects who achieve a 5 degree or more improvement in joint range-ot-motion at 52 weeks compared to baseline.

  4. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability [ Time Frame: 52 weeks ]
    Percentage of subjects who develop an AE and/or SAE



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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female ≥5 years of age;
  • Diagnosis of MPS I or II;
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03153319


Contacts
Contact: Lynda Polgreen, MD (310) 222-1961 lpolgreen@labiomed.org
Contact: Nathalia Cressey (310) 781-3682 ncressey@labiomed.org

Locations
United States, California
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center Recruiting
Torrance, California, United States, 90502
Contact: Lynda Polgreen, MD, MS    310-222-1961    lpolgreen@labiomed.org   
Principal Investigator: Lynda Polgreen, MD, MS         
Sponsors and Collaborators
Los Angeles Biomedical Research Institute
Investigators
Principal Investigator: Lynda Polgreen, MD Los Angeles Biomedical Research Institute

Responsible Party: Los Angeles Biomedical Research Institute
ClinicalTrials.gov Identifier: NCT03153319     History of Changes
Other Study ID Numbers: 31041-01
First Posted: May 15, 2017    Key Record Dates
Last Update Posted: August 15, 2017
Last Verified: July 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis I
Mucopolysaccharidosis II
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Adalimumab
Anti-Inflammatory Agents
Antirheumatic Agents