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

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.

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.