Open-label Study of Anakinra in MPS III

• Register: ClinicalTrials.gov
• Last refreshed on: 4 March 2024
• Main ID: NCT04018755
• Date of registration: 01/07/2019
• Prospective Registration: Yes
• Primary sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Public title: Open-label Study of Anakinra in MPS III
• Scientific title: Open-label Pilot Study of the Effects of Anakinra in Mucopolysaccharidosis (MPS) III
• Date of first enrolment: January 30, 2020
• Target sample size: 24
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04018755
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

United States

Contacts

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

Key inclusion & exclusion criteria

Inclusion Criteria:

- MPS III

- = 4 years of age

- 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 when cognitively possible.

- If on Genistein, must have been on a stable dose for 6 months prior to enrollment

- If on melatonin or other sleep medications, must have been on stable doses for the
past 3 months

Exclusion Criteria:

- Currently enrolled in another ongoing clinical treatment trial

- Previous or current treatment with anakinra, canakinumab or any other IL-1 inhibitor.

- Use of the following therapies prior to enrollment:

- Narcotic analgesics within 24 hours prior to enrollment.

- Tocilizumab, dapsone or mycophenolate mofetil within 3 weeks prior to enrollment.

- Etanercept, leflunomide, thalidomide, or cyclosporine or intraarticular,
intramuscular, intravenous, or oral administration of glucocorticoids within 4
weeks prior to enrollment.

- Intravenous immunoglobulin (IVIG), adalimumab, or methotrexate within 8 weeks
prior to enrollment.

- Infliximab, 6-mercaptopurine, azathioprine, cyclophosphamide or chlorambucil
within 12 weeks prior to enrollment.

- Rituximab within 26 weeks prior to enrollment

- Live vaccines within 1 month prior to enrollment.

- Known presence or suspicion of active, chronic or recurrent serious bacterial, fungal
or viral infections, including tuberculosis, HIV infection or hepatitis B or C
infection.

- Clinical evidence of liver disease or liver injury as indicated by presence of
abnormal liver tests:

- AST or ALT > 5 x ULN, or

- AST or ALT > 3 x ULN accompanied by elevated bilirubin >2 x ULN.

- Presence of severe renal function impairment (estimated creatinine clearance < 30
mL/min/1.73m2).

- Presence of neutropenia.

- History of malignancy.

- Known hypersensitivity to E coli-derived proteins, or any components of Kineret®
(anakinra).

- Pregnant or lactating women.

- 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

- Requirement for live vaccine exposure that would be expected to occur during the time
frame of the study



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis III

Intervention(s)

Biological: anakinra

Primary Outcome(s)

The percent of participants who required an increase in anakinra dose from 100 mg SC daily to 200 mg SC daily at Week 8 or Week 16 [Time Frame: up to 8 weeks of treatment]

Secondary ID(s)

31834-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cure Sanfilippo Foundation

Swedish Orphan Biovitrum