Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
Status:
Completed
Trial end date:
2016-07-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Los Angeles Biomedical Research Institute Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center