Trial · NCT04018755
Open-label Study of Anakinra in MPS III
NCT04018755, Phase 1, Phase 2, Completed, Lynda E Polgreen
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Open-label Study of Anakinra in MPS III. Trials, record NCT04018755. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04018755
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-label Study of Anakinra in MPS III
- NCT number
- NCT04018755
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 24 participants, actual
- Lead sponsor
- Lynda E Polgreen
- Conditions
- Mucopolysaccharidosis III
- Interventions
- anakinra
- First posted
- 2019-07-12
- Last updated
- 2024-10-22
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | up to 8 weeks of treatment | Need for dose escalation was determined by within individual change over an 8-week treatment period compared to change over the 8-week pre-treatment observational period in the 2 most bothersome symptoms for each enrolled patient chosen by their caregiver and selected from 6 suverys included in the Multi-domian Responder Index (MDRI). These surveys were: * Sanfilippo Behavior Rating Scale (SBRS) * Child Sleep Health Questionnaire (CSHQ) * Autism Parenting Stress Index (APSI) * PROMIS Fatigue - Parent Proxy Custom Short Form * Movement disorder - parent reported frequency, duration, and severity (7-day log) * Non-communicating Children's Pain Checklist-Revised (NCCPC-R) |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38907160 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04018755
GET https://conductscience.org/api/trials/v1/NCT04018755/registered