Retrospective chart review analysis of pairs of siblings with Mucopolysaccharidosis type II to evaluate the effectiveness of idursulfase started at 12 months of age and younger

Clinicaltrials.gov ID# N/A

This is a global, multicenter, non-interventional retrospective chart review study to evaluate the efficacy of Elaprase in sibling pairs with MPS II who initiated Elaprase treatment early at 12months of age or younger, compared to their sibling that initiated treatment at 36month of age or older. Assessments will include presence, age of onset, frequency of recurrence, and severity of clinical manifestations of MPS II, urine GAG levels, frequency and reasons for hospitalizations, emergency room visits and surgeries.  Male patients diagnosed with MPS II and sibling who received Elaprase for at least 2 years may be eligible for this study.  The younger sibling must have started Elaprase prior to 20 months old and be at least 12 months younger than the older sibling.  Patient who underwent bone marrow transplant, stem cell transplant, received ERT or other investigational product within 2 years of Elaprase treatment would be excluded.

Recruitment status: not yet active at BCH

PI:  Dr. Walla Al-Hertani

Contact information: 

Dr. Walla Al-Hertani  -- Walla.Al-Hertani@childrens.harvard.edu

Lora Pixley, study coordinator – Lora.Pixley@childrens.harvard.edu