Trial · NCT00288704
Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)
NCT00288704, Phase 3, Completed, Regeneron Pharmaceuticals; results first posted 2009-11-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS). Trials, record NCT00288704. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00288704
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)
- NCT number
- NCT00288704
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 104 participants, actual
- Lead sponsor
- Regeneron Pharmaceuticals
- Conditions
- Familial Cold Autoinflammatory Syndrome (FCAS), Familial Cold Urticaria, Muckle-Wells Syndrome (MWS), Genetic Diseases, Inborn
- Interventions
- rilonacept 160 mg, Placebo, rilonacept 160 mg
- First posted
- 2006-02-08
- Last updated
- 2011-12-06
- Results first posted
- 2009-11-13
- Limitations and caveats
- Cryopyrin Associated Periodic Syndrome (CAPS) is a rare disease with only a few hundred cases in the US.
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) | Baseline (Days -21 to -1) and Week 6 (Days 21-42) | The mean Key Symptom Score (KSS --from the validated, patient-administered Daily Health Assessment Form(DHAF)) was the average on a 0-10 scale (0=None, 10=Very Severe) of 5 separate scales -- rash, feeling of fever/chills, joint pain, eye redness/pain, and fatigue). KSS was averaged over two 21-day daily reporting periods (the 3 weeks prior to both baseline and week 6). In part A, a negative change in mean values indicated improvement under treatment with rilonacept in symptoms. The DHAF was used because it is a validated instrument to collect subject's self-reported responses. |
| Primary | Mean Change in Key Symptom Score (KSS) From Week 15 to Week 24 (During the Randomized Withdrawal Phase or Part B) | Week 15 through Week 24 (randomized withdrawal) | The mean Key Symptom Score (KSS --from the validated, patient-administered DHAF) was the average on a 0-10 scale (0=None, 10=Very Severe) of 5 separate scales -- rash, feeling of fever/chills, joint pain, eye redness/pain, and fatigue). Subjects all received rilonacept 160 mg from week 6 through week 14. At week 15, subjects were re-randomized in a 1:1 ratio between Placebo and rilonacept 160 mg. Subjects baseline period was the 21-day period prior to week 15 randomization. A positive score indicated a worsening of symptoms versus an active treatment rilonacept baseline period. |
| Other | Mean Change From Baseline to Endpoint (Week 6) in Number of Disease Flare Days Per Patient | Baseline to Week 6 (Part A) | A Disease flare day was any day where the mean Key Symptom Score (KSS) was greater than 3. The KSS was the average on a 0-10 scale (0=None, 10=Very Severe) of 5 separate scales -- rash, feeling of fever/chills, joint pain, eye redness/pain, and fatigue). KSS was calculated for 21 Day Periods at baseline and at the endpoint (from Weeks 3 - 6). The difference in the number of flares between the two periods was averaged for all subjects. The DHAF was used because it is a validated instrument to collect subject's self-reported responses. It was the basis for the KSS and the flare day count. |
| Other | Change From Baseline to Endpoint (Week 6) in Physician's Global Assessment | Baseline to Week 6 (Part A) | The Physician's Global Assessment was an evaluation at each visit on a scale of 0=no disease activity to 10=severe disease activity. A negative value in change in Physician's Global Assessment is indicative of an improvement. |
| Other | Mean Change From Baseline to Endpoint (Week 6) in Patient's Global Assessment | Baseline to Week 6 (Part A) | The Patient's Global Assessment was a question on the Daily Health Assessment Form "Considering all the ways that Familial Cold Autoinflmatory Syndrome (FCAS) /Muckle-Wells Syndrome (MWS) affects you, please rate how you are doing based on the following scale" 0=very well to 10=very poor. A negative value in change in Patient's Global Assessment is indicative of an improvement. |
| Other | Median Change From Baseline to Week 6 (Part A) Endpoint in C-Reactive Protein (mg/L) | Baseline to Endpoint of Part A | An abnormal value for CRP was considered \> 8.4 mg/L. |
| Other | Median Change From Baseline to Week 6 (Part A) Endpoint in Serum Amyloid A (mg/L) | Baseline to Endpoint of Part A | An abnormal value for SAA was considered \> 6.4 mg/L. |
| Other | Number of Subjects With at Least 30% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | Baseline to Endpoint (Week 6) | The mean Key Symptom Score (KSS --from the validated, patient-administered Daily Health Assessment Form(DHAF)) was the average on a 0-10 scale (0=None, 10=Very Severe) of 5 separate scales -- rash, feeling of fever/chills, joint pain, eye redness/pain, and fatigue). |
| Other | Number of Subjects With at Least 50% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | Baseline to Week 6 (Part A) | The mean Key Symptom Score (KSS --from the validated, patient-administered Daily Health Assessment Form(DHAF)) was the average on a 0-10 scale (0=None, 10=Very Severe) of 5 separate scales -- rash, feeling of fever/chills, joint pain, eye redness/pain, and fatigue). |
| Other | Number of Subjects With at Least 75% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | Baseline to Week 6 (Part A) | The mean Key Symptom Score (KSS --from the validated, patient-administered Daily Health Assessment Form(DHAF)) was the average on a 0-10 scale (0=None, 10=Very Severe) of 5 separate scales -- rash, feeling of fever/chills, joint pain, eye redness/pain, and fatigue). |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) | Baseline (Days -21 to -1) and Week 6 (Days 21-42) | Cryopyrin Associated Autoinflammatory Syndrome (CAPS) is a rare, orphan, hereditary disease. There are several hundred CAPS cases in the United States. | Units of a Scale | MEAN |
| PRIMARY | Mean Change in Key Symptom Score (KSS) From Week 15 to Week 24 (During the Randomized Withdrawal Phase or Part B) | Week 15 through Week 24 (randomized withdrawal) | Subjects were re-randomized as part of the randomized withdrawal period (Part B). Subjects were not necessarily assigned the same treatment as in the first double-blind portion (Part A). Subjects were analyzed using last observation carried forward. | Units of a Scale | MEAN |
| OTHER_PRE_SPECIFIED | Mean Change From Baseline to Endpoint (Week 6) in Number of Disease Flare Days Per Patient | Baseline to Week 6 (Part A) | Days | MEAN | |
| OTHER_PRE_SPECIFIED | Change From Baseline to Endpoint (Week 6) in Physician's Global Assessment | Baseline to Week 6 (Part A) | Units of a Scale | MEAN | |
| OTHER_PRE_SPECIFIED | Mean Change From Baseline to Endpoint (Week 6) in Patient's Global Assessment | Baseline to Week 6 (Part A) | Visual Analog Scale | MEAN | |
| OTHER_PRE_SPECIFIED | Median Change From Baseline to Week 6 (Part A) Endpoint in C-Reactive Protein (mg/L) | Baseline to Endpoint of Part A | Milligrams per Liter | MEDIAN | |
| OTHER_PRE_SPECIFIED | Median Change From Baseline to Week 6 (Part A) Endpoint in Serum Amyloid A (mg/L) | Baseline to Endpoint of Part A | Milligrams per Liter | MEDIAN | |
| OTHER_PRE_SPECIFIED | Number of Subjects With at Least 30% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | Baseline to Endpoint (Week 6) | Participants | NUMBER | |
| OTHER_PRE_SPECIFIED | Number of Subjects With at Least 50% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | Baseline to Week 6 (Part A) | Participants | NUMBER | |
| OTHER_PRE_SPECIFIED | Number of Subjects With at Least 75% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | Baseline to Week 6 (Part A) | Participants | NUMBER |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group57
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) | Placebo | Baseline Part A | 2.4 | Standard Deviation 1.5 | Units of a Scale |
| Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) | Rilonacept 160 mg | Baseline Part A | 3.1 | Standard Deviation 1.9 | Units of a Scale |
| Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) | Placebo | Endpoint Part A (Week 6) in KSS | 2.1 | Standard Deviation 1.6 | Units of a Scale |
| Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) | Rilonacept 160 mg | Endpoint Part A (Week 6) in KSS | 0.5 | Standard Deviation 0.5 | Units of a Scale |
| Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) | Placebo | Change to Week 6 in KSS | -0.3 | Standard Deviation 0.7 | Units of a Scale |
| Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) | Rilonacept 160 mg | Change to Week 6 in KSS | -2.6 | Standard Deviation 1.9 | Units of a Scale |
| Mean Change in Key Symptom Score (KSS) From Week 15 to Week 24 (During the Randomized Withdrawal Phase or Part B) | Placebo | Baseline Part B | 0.2 | Standard Deviation 0.4 | Units of a Scale |
| Mean Change in Key Symptom Score (KSS) From Week 15 to Week 24 (During the Randomized Withdrawal Phase or Part B) | Rilonacept 160 mg | Baseline Part B | 0.3 | Standard Deviation 0.3 | Units of a Scale |
| Mean Change in Key Symptom Score (KSS) From Week 15 to Week 24 (During the Randomized Withdrawal Phase or Part B) | Placebo | Endpoint Part B (Week 24) in KSS | 1.2 | Standard Deviation 1.0 | Units of a Scale |
| Mean Change in Key Symptom Score (KSS) From Week 15 to Week 24 (During the Randomized Withdrawal Phase or Part B) | Rilonacept 160 mg | Endpoint Part B (Week 24) in KSS | 0.4 | Standard Deviation 0.5 | Units of a Scale |
1 to 10 of 57Source ClinicalTrials.gov
See all 57 results by groupParticipant flow27
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part A, Double Blind, Weeks 1-6 | STARTED | Placebo | 24 | |
| Part A, Double Blind, Weeks 1-6 | STARTED | Rilonacept 160 mg | 23 | |
| Part A, Double Blind, Weeks 1-6 | STARTED | Open-Label Extension (OLE) Rilonacept 160 mg | 0 | |
| Part A, Double Blind, Weeks 1-6 | COMPLETED | Placebo | 24 | |
| Part A, Double Blind, Weeks 1-6 | COMPLETED | Rilonacept 160 mg | 22 | |
| Part A, Double Blind, Weeks 1-6 | COMPLETED | Open-Label Extension (OLE) Rilonacept 160 mg | 0 | |
| Part A, Double Blind, Weeks 1-6 | NOT COMPLETED | Placebo | 0 | |
| Part A, Double Blind, Weeks 1-6 | NOT COMPLETED | Rilonacept 160 mg | 1 | |
| Part A, Double Blind, Weeks 1-6 | NOT COMPLETED | Open-Label Extension (OLE) Rilonacept 160 mg | 0 | |
| Part B, Randomized Withdrawal, Wks 15-24 | STARTED | Placebo | 23 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 participant flowAdverse events40
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Injection Site Reaction | General disorders | Rilonacept 160 mg | 11 | 23 | |
| other | Injection Site Reaction | General disorders | Placebo | 3 | 24 | |
| other | Sinusitis | Infections and infestations | Rilonacept 160 mg | 2 | 23 | |
| other | Sinusitis | Infections and infestations | Placebo | 1 | 24 | |
| other | Urinary Tract Infection | Infections and infestations | Rilonacept 160 mg | 1 | 23 | |
| other | Urinary Tract Infection | Infections and infestations | Placebo | 1 | 24 | |
| other | Upper Respiratory Tract Infection | Infections and infestations | Rilonacept 160 mg | 6 | 23 | |
| other | Upper Respiratory Tract Infection | Infections and infestations | Placebo | 1 | 24 | |
| other | Diarrhea | Gastrointestinal disorders | Rilonacept 160 mg | 1 | 23 | |
| other | Diarrhea | Gastrointestinal disorders | Placebo | 3 | 24 |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Rilonacept 160 mg | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Rilonacept 160 mg | 17 | 23 |
| Total, other adverse events | Placebo | 13 | 24 |
| Total, serious adverse events | Rilonacept 160 mg | 7 | 104 |
| Total, serious adverse events | Placebo | 0 | 24 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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 comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Rilonacept 160 mg vs Placebo: Number of Subjects With at Least 75% Improvement in Key Symptoms Scores (KSS) From Baseline to Endpoint (Week 6) | 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) | 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) | Trial comparisons |
| Rilonacept 160 mg vs Placebo: serious adverse events | Trial comparisons |
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.
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GET https://conductscience.org/api/trials/v1/NCT00288704
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