Status:
COMPLETED
Open-label Study of Anakinra in MPS III
Lead Sponsor:
Lynda E Polgreen
Collaborating Sponsors:
Cure Sanfilippo Foundation
Swedish Orphan Biovitrum
Conditions:
Mucopolysaccharidosis III
Eligibility:
All Genders
4+ years
Phase:
PHASE1
PHASE2
Brief Summary
Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a disorder of metabolism, associated with insufficient production of a lysosomal enzyme needed for normal cell function. As a conse...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 30 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 8 2023
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT04018755
Start Date
January 30 2020
End Date
March 8 2023
Last Update
October 22 2024
Active Locations (1)
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1
The Lundquist Institute at Harbor-UCLA Medical Center
Torrance, California, United States, 90502