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Canakinumab in Patients With Active Hyper-IgD Syndrome

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ClinicalTrials.gov Identifier: NCT01303380
Recruitment Status : Completed
First Posted : February 24, 2011
Results First Posted : August 19, 2015
Last Update Posted : November 5, 2015
Sponsor:
Information provided by (Responsible Party):

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Mevalonate Kinase Deficiency
Intervention: Drug: Canakinumab

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The study was conducted at 3 centres in Spain.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 10 participants were screened, 9 of which entered in the treatment period of the study, one participant was considered to be a screening failure.

Reporting Groups
  Description
Canakinumab Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new Hyper-IgD with periodic fever syndrome (HIDS) flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.

Participant Flow:   Overall Study
    Canakinumab
STARTED   9 
COMPLETED   8 
NOT COMPLETED   1 
Lack of compliance to study procedures                1 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The analysis was performed in the Full Analysis Set (FAS), defined as all participants who received at least one application of study treatment and had at least one post-baseline assessment for primary efficacy variable.

Reporting Groups
  Description
Canakinumab Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.

Baseline Measures
   Canakinumab 
Overall Participants Analyzed 
[Units: Participants]
 9 
Age 
[Units: Years]
Median (Full Range)
 17.3 
 (5 to 29) 
Age, Customized 
[Units: Participants]
 
Children (2­-11 years)   3 
Adolescents (12­-17 years)   3 
Adults (18-­64 years)   3 
Gender 
[Units: Participants]
 
Female   6 
Male   3 
Region of Enrollment 
[Units: Participants]
 
Spain   9 


  Outcome Measures

1.  Primary:   Number of Flares Per Participant During Historical Period and Treatment Period   [ Time Frame: Historical period, Month 6 (End of treatment period) ]

2.  Secondary:   Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period   [ Time Frame: Month 6 (End of treatment period), Month 36 (End of Long term treatment Period 2) ]

3.  Secondary:   Number of Participants Who Flared at Month 6, Month 24 and Month 36   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

4.  Secondary:   Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score   [ Time Frame: Any flare event [Baseline up to Month 36 (End of long term treatment period 2)] ]

5.  Secondary:   Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

6.  Secondary:   Percentage of Participants With Defined Grades of Participants Assessed Symptom Control   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

7.  Secondary:   Percentage of Participants With Defined Grades of Physician Assessed Symptom Control   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

8.  Secondary:   Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

9.  Secondary:   Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

10.  Secondary:   Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

11.  Secondary:   Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

12.  Secondary:   Time to Resolution of the Initial Flare After First Canakinumab Treatment   [ Time Frame: Day 1 (Baseline), Day 28 ]

13.  Secondary:   Change From Baseline in Inflammation Markers Over Time up to Month 24   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

14.  Secondary:   Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

15.  Secondary:   Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

16.  Secondary:   Percentage of Participants Who Received Dose Up-titration During 6-month Treatment Period   [ Time Frame: Day 1 up to Month 6 (End of follow up) ]

17.  Secondary:   Duration of Flares Experienced During the Study   [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]

18.  Secondary:   Time to Flare After the Last Dose of Canakinumab During the Follow-up Period   [ Time Frame: Last dose of canakinumab treatment in follow-up period to end of follow-up period (Day 337) ]

19.  Secondary:   Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)   [ Time Frame: Day 1 (Start of study treatment) up to Month 36 (End of study) ]

20.  Secondary:   Participants Who Received Rescue Treatment   [ Time Frame: Baseline up to Month 36 (End of study) ]

21.  Secondary:   Serum Concentration-time Profile of Canakinumab   [ Time Frame: Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose) ]

22.  Secondary:   Serum Concentration of Total Interleukin-1β Antibody (IL-1β)   [ Time Frame: Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose) ]

23.  Secondary:   Number of Participants Exhibiting Anti-canakinumab Antibodies at Any Visit   [ Time Frame: Baseline up to Month 36 (End of study) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862 ­778 ­8300



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01303380     History of Changes
Other Study ID Numbers: CACZ885D2402
2010-020904-31 ( EudraCT Number )
First Submitted: February 23, 2011
First Posted: February 24, 2011
Results First Submitted: July 15, 2015
Results First Posted: August 19, 2015
Last Update Posted: November 5, 2015