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

• Register: ClinicalTrials.gov
• Last refreshed on: 3 April 2023
• Main ID: NCT03153319
• Date of registration: 11/05/2017
• Prospective Registration: Yes
• Primary sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Public title: Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
• Scientific title: Phase 1/2 Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I, II, and VI
• Date of first enrolment: June 5, 2017
• Target sample size: 14
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03153319
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Lynda Polgreen, MD
• Affiliation: The Lundquist Institute at Harbor-UCLA Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female =5 years of age;

- Diagnosis of MPS I, II or VI;

- 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.



Age minimum: 5 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis II

Mucopolysaccharidosis I

Mucopolysaccharidosis VI

Intervention(s)

Drug: Adalimumab Injection [Humira]

Drug: Saline Solution for Injection

Primary Outcome(s)

Adalimumab trough [Time Frame: 32 weeks]

Pain - 16 weeks [Time Frame: 16 weeks]

Secondary Outcome(s)

Pain - 52 weeks [Time Frame: 52 weeks]

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability [Time Frame: 52 weeks]

Joint range-of-motion - 16 weeks [Time Frame: 16 weeks]

Joint range-of-motion - 52 weeks [Time Frame: 52 weeks]

Secondary ID(s)

31041-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors