Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

Conditions

• Mucopolysaccharidosis Type I
• Mucopolysaccharidosis Type II
• Mucopolysaccharidosis Type VI

Eligibility Criteria

Sex

ALL

Age

5 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Adalimumab — DRUG
  Subjects will be treated with adalimumab (20 mg \[weight 15-\<30 kg\] or 40 mg \[weight ≥30 kg\] administered subcutaneously \[subQ\] every other week) or placebo for 16 weeks, then cross-over to the other group for 16 weeks.
• Placebo — OTHER
  Saline placebo

Study Details

The purpose of the study is to collect preliminary data on whether the drug adalimumab (also called Humira) can decrease pain and stiffness, improve quality of life, and is safe in people with mucopolysaccharidosis type I, II, or VI. In this study people will be randomly assigned to one of two groups. One group will be treated with adalimumab the first 16 weeks of the study and then with a saline injection for the last 16 weeks of the study. The other group will start with the saline injection for 16 weeks and then switch to adalimumab for the last 16 weeks. The study subject and the study doctor and study coordinator will not know what group a subject is in until the study is done. Adalimumab is given as an injection, just under the skin, every 2 weeks. Both groups will have blood drawn at a screening visit, and then 7 more times over the 32 week study. There will be safety labs done (liver and immune function tests). Other safety tests include a chest X-ray and screening for tuberculosis exposure - these will be done at the screening visit and later in the study if there is concern for tuberculosis exposure or a persistent cough. The following will also be done at screening, the first, middle, and last study visits: 1) a pregnancy test in all girls 8 and older, 2) questionnaires that ask about pain, how MPS impacts social and physical function, and other quality of life questions, 3) height and weight. Finally, a physical exam, that includes for children and adolescents a check of where they are in puberty, will be done by a study physician at the first, middle, and last visits. There are risks to taking adalimumab that include redness and pain where the injection is given, a decreased ability to fight off infections, and others. The safety tests are designed to identify and decrease the risk associated with adalimumab. The study physicians believe that the potential benefit of adalimumab on pain, quality of life, and other MPS related problems outweigh the potential risks of treatment.

Key Dates

Start date

May 31, 2015

Status verified

May 2017

Primary completion

Jul 31, 2016

Completion

Jul 31, 2016

Study Design

Enrollment

2 participants (actual)

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Arms

• Experimental: Adalimumab first, then Placebo
  Participants first received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
• Experimental: Placebo first, then Adalimumab
  Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to \<30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.

Primary Outcome Measure

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

Locations (1)

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