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

study id #: NCT03153319

condition: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI

status: Recruiting

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, II or VI.

intervention: Adalimumab Injection [Humira], Saline Solution for Injection

results: https://clinicaltrials.gov/ct2/show/results/NCT03153319

last updated: February 26, 2022

start date: June 5, 2017

estimated completion: February 2023

last updated: December 14, 2021

phase of development: Phase 1/Phase 2

size / enrollment: 14

study description: 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, II or VI. Children and adults diagnosed with MPS I, II or VI, 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.

primary outcomes:

• Pain - 16 weeks
  Mean difference in bodily pain measured by the Children's Health Questionnaire - Parent Form 50 (CHQ-PF50) or the Medical Outcomes Study - Short Form 36 (SF-36) in treatment versus placebo at 16 weeks
• 16 weeks
  Adalimumab trough
  Percentage of subjects who achieve a goal trough concentration of adalimumab with every other week dosing
• 32 weeks
  

secondary outcomes:

• Joint range-of-motion - 16 weeks
  16 weeks
• Pain - 52 weeks
  52 weeks
• Joint range-of-motion - 52 weeks
  52 weeks
• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability
  52 weeks

inclusion criteria:

exclusion criteria: 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.

sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

contacts: Adolfo Morales, 310-357-9023, adolfo.morales@lundquist.org

investigators: Lynda Polgreen, MD,The Lundquist Institute At Harbor-UCLA Medical Center

trial center locations: United States

• United States, California
  The Lundquist Institute at Harbor-UCLA Medical Center
  Adolfo Morales, 310-357-9023, adolfo.morales@lundquist.org