Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02437253
• Date of registration: 05/05/2015
• Prospective Registration: No
• Primary sponsor: Los Angeles Biomedical Research Institute
• Public title: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
• Scientific title: Pilot Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I, II and VI
• Date of first enrolment: May 2015
• Target sample size: 2
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02437253
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Lynda E Polgreen, MD, MS
• Affiliation: Los Angeles Biomedical Research Institute

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of MPS I, II or VI;

- Treatment with ERT for =1 year or no ERT for =1 year;

- Weight =15 kg;

- Bodily pain reported by the CHQ-PF50 or SF-36 > 1 SD below 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 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.



Age minimum: 5 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis Type I

Mucopolysaccharidosis Type VI

Mucopolysaccharidosis Type II

Intervention(s)

Drug: Adalimumab

Other: Placebo

Primary Outcome(s)

Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score [Time Frame: day 0 to week 16 of treatment with adalimumab versus placebo]

Secondary Outcome(s)

Anti-ERT Antibodies [Time Frame: Day 0 to week 16 of treatment with adalimumab versus placebo]

Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ) [Time Frame: Day 0 to week 16 of treatment with adalimumab versus placebo]

Children's Health Questionnaire - Parent Form 50 Physical Function (PF) Standardized Score [Time Frame: Day 0 to week 16 of treatment with adalimumab versus placebo]

Range of Motion - Bilateral Shoulder, Elbow, Hip, Knee [Time Frame: Day 0 to week 16 of treatment with adalimumab versus placebo]

Secondary ID(s)

30535

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 15/09/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02437253