Trial · 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
NCT01576367, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2016-08-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. 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. Trials, record NCT01576367. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01576367
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
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
- NCT number
- NCT01576367
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 17 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Interventions
- ACZ885
- First posted
- 2012-04-12
- Last updated
- 2018-09-11
- Other registries' IDs
- eudract:2011-005154-57
- Results first posted
- 2016-08-03
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers. | Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months) | Disease relapse following complete response is defined as inflammation markers: C-Reactive Protein (CRP) and/or Serum Amyloid A (SAA) result \> 30 mg/L AND Physician's Global Assessment of Autoinflammatory Disease Activity \> minimal or Physician's Global Assessment \>= minimal AND Skin Disease Assessment \> minimal. Physician's Global Assessment of Autoinflammatory Disease Activity and Skin Disease Assessment (urticarial skin rash) are completed by the investigator using a 5 point rating scale: absent, minimal, mild, moderate and severe. |
| Secondary | Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies | minimum of 6 months and maximum of 24 months | Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique. |
| Secondary | Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Week 0, 80, 104, 128 and 152, last assessment | CRP and SAA were used as serologic inflammatory markers. The target level concentrations for CRP and SAA was ≤15 mg/L and ≤10 mg/L, respectively. Negative change in concentration of inflammatory markers indicated improvement. |
| Secondary | Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease | minimum of 6 months and maximum of 24 months | Participants were assessed based by physician on Physician's Global Assessment measured on a 5--point scale for auto inflammatory disease activity as: 0 = None/absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe |
| Secondary | Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines | pre-vaccine dose, Day 28 post-vaccine | Participants who received any inactivated vaccines during the study were assessed for their ability to attain protective antibody levels against the vaccine (antigen) post immunization. Participants vaccinations were not assessed for a response if the antibody titre was already sufficient at pre-dose and maintained during the study. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers. | Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months) | Extension Full analysis set (FAS) consisted of all patients who received at least one dose of study drug in the extension study | Percentage of participants | NUMBER |
| SECONDARY | Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies | minimum of 6 months and maximum of 24 months | Extension Safety set consisted of all patients from the core study who received at least one dose of study drug in the extension study and had at least one post-treatment safety assessment. Of note, the statement that a patient had no AE also constituted a safety assessment. | Participants | NUMBER |
| SECONDARY | Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Week 0, 80, 104, 128 and 152, last assessment | Extension Full analysis set (FAS) consisted of all patients who received at least one dose of study drug in the extension study | (mg/L) | MEAN |
| SECONDARY | Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease | minimum of 6 months and maximum of 24 months | Extension Full analysis set (FAS) consisted of all patients who received at least one dose of study drug in the extension study | Percentage of participants | NUMBER |
| SECONDARY | Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines | pre-vaccine dose, Day 28 post-vaccine | Extension Full analysis set (FAS) consisted of all patients who received at least one dose of study drug in the extension study. Out of 20 unique patient-vaccination cases, 17 cases were assessable for a vaccination response due to availability of pre dose antibody titer. | vaccination cases | NUMBER |
Source ClinicalTrials.gov
Results by group26
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Serological Inflammation Markers. | Canakinumab | 94.1 | Percentage of participants | ||
| Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies | Canakinumab | 0 | Participants | ||
| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Canakinumab | CRP at Core End of Study (last assessment) (n=14) | -5.4 | Standard Deviation 6.28 | (mg/L) |
| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Canakinumab | CRP at week 80 (n=16) | -14.7 | Standard Deviation 35.8 | (mg/L) |
| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Canakinumab | CRP at Week 104 (n=11) | -3.8 | Standard Deviation 14.4 | (mg/L) |
| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Canakinumab | CRP at Week 128(n=12) | -4.1 | Standard Deviation 10.3 | (mg/L) |
| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Canakinumab | CRP at Week 152 (n=12) | -4.3 | Standard Deviation 11 | (mg/L) |
| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Canakinumab | CRP at End of Study (last assessment) (n=16) | -10.4 | Standard Deviation 30.3 | (mg/L) |
| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Canakinumab | SAA at Core End of Study (last assessment) (n=16) | -54.4 | Standard Deviation 133.8 | (mg/L) |
| Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations | Canakinumab | SAA at Week 80 (n=12) | -79.1 | Standard Deviation 224.1 | (mg/L) |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Canakinumab | 17 | |
| Overall Study | COMPLETED | Canakinumab | 14 | |
| Overall Study | NOT COMPLETED | Canakinumab | 3 |
Source ClinicalTrials.gov
Adverse events97
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 | Eosinophilia | Blood and lymphatic system disorders | Canakinumab | 1 | 17 | |
| other | Angina pectoris | Cardiac disorders | Canakinumab | 1 | 17 | |
| other | Cryopyrin associated periodic syndrome | Congenital, familial and genetic disorders | Canakinumab | 1 | 17 | |
| other | Conductive deafness | Ear and labyrinth disorders | Canakinumab | 1 | 17 | |
| other | Ear pain | Ear and labyrinth disorders | Canakinumab | 2 | 17 | |
| other | Eye pruritus | Eye disorders | Canakinumab | 1 | 17 | |
| other | Iridocyclitis | Eye disorders | Canakinumab | 1 | 17 | |
| other | Papilloedema | Eye disorders | Canakinumab | 2 | 17 | |
| other | Uveitis | Eye disorders | Canakinumab | 1 | 17 | |
| other | Abdominal pain | Gastrointestinal disorders | Canakinumab | 2 | 17 |
1 to 10 of 97Source ClinicalTrials.gov
See all 97 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Canakinumab | ||
| Total, other adverse events | Canakinumab | 16 | 17 |
| Total, serious adverse events | Canakinumab | 8 | 17 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 31161734 | 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/NCT01576367
GET https://conductscience.org/api/trials/v1/NCT01576367/results?page=2