Disease · MONDO_0016168
cryopyrin-associated periodic syndrome
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. cryopyrin-associated periodic syndrome. Diseases, record MONDO_0016168. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0016168
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Highest stage of its drugs
- Approval, for 2 of its 8 drugs, from DailyMed, EMA Human Drugs and TTD
- Open Targets
Drugs8
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| CANAKINUMAB | CHEMBL1201834 | Antibody | Approval | DailyMed, EMA Human Drugs and TTD | 10 | 7 | 7 | Drugs and targets |
| RILONACEPT | CHEMBL1201830 | Protein | Approval | DailyMed | 3 | 1 | 1 | Drugs and targets |
| ZUNSEMETINIB | CHEMBL3704901 | Small molecule | Phase 2 | ClinicalTrials.gov and TTD | 2 | 1 | 1 | Drugs and targets |
| TRANILAST | CHEMBL415324 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| USNOFLAST | CHEMBL5314548 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| INZOMELID | CHEMBL4650350 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SELNOFLAST POTASSIUM | CHEMBL5095351 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ANAKINRA | CHEMBL1201570 | Protein | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets2
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| IL1B | Interleukin-1 beta | 2 | Interleukin-1 beta inhibitor | Approval | https://conductscience.org/library/genes/3553 | Drugs and targets |
| IL1R1 | Interleukin-1 receptor type 1 | 1 | Interleukin-1 receptor antagonist | Unknown | https://conductscience.org/library/genes/3554 | Drugs and targets |
Trials14
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT00288704 | Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) | Phase 3 | 104 | 2009-11-13 | Familial Cold Autoinflammatory Syndrome (FCAS), Familial Cold Urticaria, Muckle-Wells Syndrome (MWS), Genetic Diseases, Inborn | RILONACEPT | Its record names the disease | ClinicalTrials.gov |
| NCT00465985 | Efficacy, Safety, and Tolerability of ACZ885 in Patients With Muckle-Wells Syndrome | Phase 3 | 35 | 2011-02-11 | Muckle Wells Syndrome | CANAKINUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT01302860 | Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease | Phase 3 | 17 | 2015-08-18 | Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease | CANAKINUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT01576367 | Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease | Phase 3 | 17 | 2016-08-03 | Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease | CANAKINUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04524858 | Study of ATI-450 in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS) | Phase 2 | 1 | 2023-09-21 | Cryopyrin-Associated Periodic Syndrome | ZUNSEMETINIB | Its record names the disease | ClinicalTrials.gov |
| NCT04015076 | Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With Inzomelid | Phase 1 | 80 | Healthy | INZOMELID | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT03923140 | A Clinical Study of Tranilast in the Treatment of Cryopyrin-Associated Periodic Syndrome (CAPS) | Phase 2 | 71 | Cryopyrin-Associated Periodic Syndromes | TRANILAST | Its record names the disease | ClinicalTrials.gov | |
| NCT04086602 | Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334 | Phase 1 | 64 | Healthy Volunteers, Cryopyrin Associated Periodic Syndrome | SELNOFLAST POTASSIUM | Its record names the disease | ClinicalTrials.gov | |
| NCT00487708 | Safety, Efficacy, Pharmacokinetics, Pharmacodynamics of ACZ885 in Patients With NALP3 Mutations and Clinical Symptoms | Phase 2 | 34 | NALP3 Mutation | CANAKINUMAB | Its record names the disease | ClinicalTrials.gov | |
| NCT06838143 | Ilaris NIS in Korea | 25 | Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS), Colchicine Resistance Familial Mediterranean Fever (crFMF), TNF Receptor Associated Periodic Syndrome (TRAPS), Hyper-IgD Syndrome / Mevalonate Kinase Deficiency (HIDS/MKD), Systemic Juvenile Idiopathic Arthritis (sJIA) | CANAKINUMAB | Open Targets' reading of its record | ClinicalTrials.gov |
1 to 10 of 14
See all 14 trialsComparisons in its trials5
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Rilonacept 160 mg vs Placebo: serious adverse events | 7 of 104 | 0 of 24 | 128 | NCT00288704 | Trial comparisons |
| Rilonacept 160 mg vs Placebo: Number of Subjects With at Least 75% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | 16 of 23 | 0 of 24 | 47 | NCT00288704 | Trial comparisons |
| Rilonacept 160 mg vs Placebo: Number of Subjects With at Least 50% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | 20 of 23 | 2 of 24 | 47 | NCT00288704 | Trial comparisons |
| Rilonacept 160 mg vs Placebo: Number of Subjects With at Least 30% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | 22 of 23 | 7 of 24 | 47 | NCT00288704 | Trial comparisons |
| ACZ885 vs Placebo: Number of Participants Who Experienced a Disease Flare in Part II | 0 of 15 | 13 of 16 | 31 | NCT00465985 | Trial comparisons |
Papers from its trials7
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
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
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0016168
GET https://conductscience.org/api/diseases/v1/MONDO_0016168/drugs