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Interleukin-1 (IL-1) Inhibitor
anakinra for Mucopolysaccharidosis
Phase 2 & 3
Waitlist Available
Led By Lynda Polgreen, MD, MS
Research Sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 8 weeks of treatment
Awards & highlights
Study Summary
This trial is testing whether a drug called anakinra can help treat symptoms in children with Sanfilippo syndrome, a disorder that causes progressive, irreversible neurodegeneration.
Eligible Conditions
- Mucopolysaccharidosis
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 8 weeks of treatment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 8 weeks of treatment
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
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
Side effects data
From 2017 Phase 2 trial • 33 Patients • NCT0227856214%
right side weakness
14%
access related
14%
arrhythmia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo 1 and 2
Actos
Anakinra
Trial Design
1Treatment groups
Experimental Treatment
Group I: treatmentExperimental Treatment1 Intervention
anakinra 100 mg subcutaneous once daily
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
anakinra
2012
Completed Phase 3
~240
Find a Location
Who is running the clinical trial?
Cure Sanfilippo FoundationUNKNOWN
1 Previous Clinical Trials
175,000 Total Patients Enrolled
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterLead Sponsor
101 Previous Clinical Trials
46,277 Total Patients Enrolled
Swedish Orphan BiovitrumIndustry Sponsor
92 Previous Clinical Trials
12,490 Total Patients Enrolled
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Can you give some examples of other research that has been done on anakinra?
"There are currently 34 active clinical trials studying anakinra, 6 of which have reached Phase 3. Many of these studies originate from Paris and Ile-De-France; however, 440 different locations around the world are running similar investigations."
Answered by AI
How is anakinra typically employed in medical settings?
"Systemic juvenile idiopathic arthritis (sjia) is most often treated with the medication anakinra. However, anakinra can also help patients manage neonatal-onset multisystem inflammatory disease (NOMID), receptors, interleukin-1, and steroids."
Answered by AI
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