Trial · 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
NCT01302860, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2015-08-18
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 NCT01302860. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01302860
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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
- NCT01302860
- 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
- 2011-02-24
- Last updated
- 2017-03-29
- Results first posted
- 2015-08-18
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56 | Week 56 | Complete response was defined as clinical remission and serological remission. Clinical remission was defined as Physician global assessment of auto-inflammatory disease activity as absent or minimal (using a 5-point scale ranging from absent to severe) and assessment of skin disease as absent or minimal (using a 5-point scale ranging from absent to severe). Serological remission was defined as C reactive protein (CRP) or Serum amyloid A protein (SAA) to be less than (\<) 15 milligram per liter (mg/L) and \<10 mg/L respectively. |
| Secondary | Percentage of Participants Aged 2 Years or Younger With at Least One Complete Response at Week 56 | Week 56 | Complete response was defined as clinical remission and serological remission. Clinical remission was defined as Physician global assessment of auto-inflammatory disease activity as absent or minimal (using a 5-point scale ranging from absent to severe) and assessment of skin disease as absent or minimal (using a 5-point scale ranging from absent to severe). Serological remission was defined as CRP or SAA to be \<15 mg/L and \<10 mg/L respectively. |
| Secondary | Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | Week 56 | 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 | Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56 | Week 56 | Participants were assessed by physician for skin disease (urticarial skin rash) measured on a 5--point scale as: 0 = None/absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe. |
| Secondary | Change From Baseline in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations at Week 56 | Baseline, Week 56 | The CRP and SAA were used as 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 | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 (start of study treatment) up to Week 56 (end of study) | Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. |
| Secondary | Percentage of Participants Receiving a Concomitant Vaccination During the Study | Day 1 (start of study treatment) to Week 56 (end of study) | Participants received any one of the following inactivated vaccines as per the immunization program: Corynebacterium diphtheria, Bordetella pertussis, Neisseria meningitidis, Clostridium tetani, Influenza type A, Influenza type B, Haemophilus influenza B, Streptococcus pneumoniae, or Hepatitis B were determined. |
| Secondary | Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines | Day -14 (prior-vaccination), Day 0 (vaccination), Day 28, Day 57 (post-vaccination) | 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. |
| Secondary | Number of Participants With Anti-canakinumab Antibodies at Week 56 | Week 56 (End of study) | Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using BIAcore system, with detection based on surface plasmon resonance technique. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56 | Week 56 | The analysis was performed in Full analysis set (FAS), defined as all participants who received at least one dose of study drug under this study protocol. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Aged 2 Years or Younger With at Least One Complete Response at Week 56 | Week 56 | The analysis was performed in FAS population. Here, Number of participants analysed signifies participants aged 2 years or younger. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | Week 56 | The analysis was performed in FAS population. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56 | Week 56 | The analysis was performed in FAS population. | Percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations at Week 56 | Baseline, Week 56 | The analysis was performed in FAS population. Here 'n' signifies those participants with evaluable measurements at both baseline and the post-baseline visit. | mg/L | MEAN |
| SECONDARY | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 (start of study treatment) up to Week 56 (end of study) | The analysis was performed in the safety set population defined as participants who received at least one dose of study drug. Here, 'n' signifies participants evaluable for this measure at specified time points for each group, respectively. | participants | NUMBER |
| SECONDARY | Percentage of Participants Receiving a Concomitant Vaccination During the Study | Day 1 (start of study treatment) to Week 56 (end of study) | The analysis was performed in the FAS population. | Percentage of participants | NUMBER |
| SECONDARY | Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines | Day -14 (prior-vaccination), Day 0 (vaccination), Day 28, Day 57 (post-vaccination) | The analysis was performed in the FAS population. Here, Number of participants analysed signifies evaluable participants who received a total of 31 vaccinations during the study. | vaccination cases | NUMBER |
| SECONDARY | Number of Participants With Anti-canakinumab Antibodies at Week 56 | Week 56 (End of study) | The analysis was performed in the safety set population. Here, 'Number of participants analysed' signifies participants who had immunogenicity samples taken and analyzed during the study. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group22
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Aged 4 Years or Younger With at Least One Complete Response at Week 56 | Canakinumab | 94.1 | Percentage of participants | ||
| Percentage of Participants Aged 2 Years or Younger With at Least One Complete Response at Week 56 | Canakinumab | 90 | Percentage of participants | ||
| Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | Canakinumab | Absent | 70.6 | Percentage of participants | |
| Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | Canakinumab | Minimal | 23.5 | Percentage of participants | |
| Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | Canakinumab | Mild | 0 | Percentage of participants | |
| Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | Canakinumab | Moderate | 5.9 | Percentage of participants | |
| Percentage of Participants With Defined Grades in Physician's Global Assessment Score at Week 56 | Canakinumab | Severe | 0 | Percentage of participants | |
| Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56 | Canakinumab | Absent | 82.4 | Percentage of participants | |
| Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56 | Canakinumab | Minimal | 5.9 | Percentage of participants | |
| Percentage of Participants With Defined Grades in Physician Assessment of Skin Disease at Week 56 | Canakinumab | Mild | 11.8 | Percentage of participants |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 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 | 17 | |
| Overall Study | NOT COMPLETED | Canakinumab | 0 |
Source ClinicalTrials.gov
Adverse events61
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 | Cryopyrin associated periodic syndrome | Congenital, familial and genetic disorders | Canakinumab | 2 | 17 | |
| other | Conductive deafness | Ear and labyrinth disorders | Canakinumab | 1 | 17 | |
| other | Iridocyclitis | Eye disorders | Canakinumab | 1 | 17 | |
| other | Papilloedema | Eye disorders | Canakinumab | 1 | 17 | |
| other | Abdominal pain | Gastrointestinal disorders | Canakinumab | 3 | 17 | |
| other | Diarrhoea | Gastrointestinal disorders | Canakinumab | 4 | 17 | |
| other | Gastritis | Gastrointestinal disorders | Canakinumab | 1 | 17 | |
| other | Stomatitis | Gastrointestinal disorders | Canakinumab | 1 | 17 | |
| other | Toothache | Gastrointestinal disorders | Canakinumab | 1 | 17 |
1 to 10 of 61Source ClinicalTrials.gov
See all 61 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 | 17 | 17 |
| Total, serious adverse events | Canakinumab | 4 | 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/NCT01302860
GET https://conductscience.org/api/trials/v1/NCT01302860/results?page=2