Phase 2
N=9
Canakinumab in Patients With Active Hyper-IgD Syndrome
Mevalonate Kinase Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT01303380 ↗Enrolled (actual)
9
Serious AEs
44.4%
Results posted
Aug 2015
Primary outcome: Primary: Number of Flares Per Participant During Historical Period and Treatment Period — 5; 0 Number of flares
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Canakinumab (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Jul 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Flares Per Participant During Historical Period and Treatment Period |
5; 0 | — |
| SECONDARY Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period |
0; 0 | — |
| SECONDARY Number of Participants Who Flared at Month 6, Month 24 and Month 36 |
2; 4; 2 | — |
| SECONDARY Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score |
0; 0; 0; 1; 0; 2 | — |
| SECONDARY Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score |
0; 0; 1; 1; 0; 0 | — |
| SECONDARY Percentage of Participants With Defined Grades of Participants Assessed Symptom Control |
0; 0; 11.11; 33.33; 55.56; 0 | — |
| SECONDARY Percentage of Participants With Defined Grades of Physician Assessed Symptom Control |
0; 0; 0; 44.44; 55.56; 0 | — |
| SECONDARY Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment |
88.89; 11.11; 0; 0; 0; 100 | — |
| SECONDARY Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment |
88.89; 0; 11.11; 0; 0; 100 | — |
| SECONDARY Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment |
88.89; 11.11; 0; 0; 0; 77.78 | — |
| SECONDARY Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment |
100; 0; 0; 0; 0; 100 | — |
| SECONDARY Time to Resolution of the Initial Flare After First Canakinumab Treatment |
3 | — |
| SECONDARY Change From Baseline in Inflammation Markers Over Time up to Month 24 |
-120.5; -111; -113.55; -125.2; -624.2; -685.95 | — |
| SECONDARY Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time |
0; 0; 0; 0 | — |
| SECONDARY Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time |
0; 0.1; 0.1; 0.1 | — |
| SECONDARY Percentage of Participants Who Received Dose Up-titration During 6-month Treatment Period |
22.22 | — |
| SECONDARY Duration of Flares Experienced During the Study |
3; 4; 3.5; 8.5 | — |
| SECONDARY Time to Flare After the Last Dose of Canakinumab During the Follow-up Period |
110 | — |
| SECONDARY Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
9; 4 | — |
| SECONDARY Participants Who Received Rescue Treatment |
11.11 | — |
| SECONDARY Serum Concentration-time Profile of Canakinumab |
28.8; 26.9; 12.7; 19.4; 23.8; 24 | — |
| SECONDARY Serum Concentration of Total Interleukin-1β Antibody (IL-1β) |
27.3; 22.2; 21.8; 27.3; 24.3; 32.5 | — |
| SECONDARY Number of Participants Exhibiting Anti-canakinumab Antibodies at Any Visit |
— | — |
Summary
This pilot study is designed to evaluate the efficacy, the safety, and the pharmacokinetics (PK) / pharmacodynamics (PD) of canakinumab treatment in patients with HIDS.
Eligibility Criteria
Inclusion Criteria
- Patients with a diagnosis of HIDS proven by DNA analysis and/or enzymatic studies.
- At time of start of drug treatment: active HIDS as evidenced by a physician global assessment of HIDS flare severity ≥ 2 and CRP values >10 mg/L (normal CRP or = 3 febrile acute HIDS flares in a 6-month period when not receiving prophylaxis treatment (e.g. anakinra daily treatment) with a duration of each flare lasting > or = 4 days and limiting the normal daily activities.
Exclusion Criteria
- Pregnant or nursing (lactating) women.
- History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result.
- Positive Hepatitis B or Hepatitis C.
- Live vaccinations within 3 months prior to the start of the trial
- Positive tuberculosis screening test.
Other protocol-defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT01303380). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.