Trial Outcomes & Findings for Rilonacept for Deficiency of the Interleukin-1 Receptor Antagonist (DIRA) (NCT NCT01801449)

NCT ID: NCT01801449

Last Updated: 2017-10-16

Results Overview

Inflammatory remission criteria remission criteria were defined as meeting all of the following criteria: a diary score of \<0.5 (reflecting no fever, skin rash or bone pain), normal acute phase reactants (C-reactive protein (CRP) \<0.5 mg/dL), no objective skin rash or radiological evidence of active bone lesions on x-ray.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

6 months

Results posted on

2017-10-16

Participant Flow

Participant milestones

Participant milestones
Measure	Rilonacept Treatment Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week.Total duration of the study 24 months.
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Rilonacept for Deficiency of the Interleukin-1 Receptor Antagonist (DIRA)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Age, Categorical <=18 years	6 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: Analysis included all subjects

Inflammatory remission criteria remission criteria were defined as meeting all of the following criteria: a diary score of \<0.5 (reflecting no fever, skin rash or bone pain), normal acute phase reactants (C-reactive protein (CRP) \<0.5 mg/dL), no objective skin rash or radiological evidence of active bone lesions on x-ray.

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Number of Subjects Who Maintained Inflammatory Remission With Rilonacept	6 Participants

SECONDARY outcome

Timeframe: 24 months after rilonacept initiation

Population: Analysis included all subjects

Antropometric measurements - Height z-score

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Height	-1.22 z-score Standard Deviation 0.48

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects

Antropometric measurements - Height z-score

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Height	-1.1 z-score Standard Deviation 0.41

SECONDARY outcome

Timeframe: 24 months after rilonacept initiation

Population: Analysis included all subjects

Antropometric measurements - Weight z-score

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Weight	-0.48 z-score Standard Deviation 0.64

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects

Antropometric measurements - Weight z-score

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Weight	-0.59 z-score Standard Deviation 0.62

SECONDARY outcome

Timeframe: 24 months after rilonacept initiation

Population: Analysis included all subjects

The Childhood Health Assessment Questionnaire (CHAQ) is a patient self-assessment (or parent assessment) tool for how illness affects ability to function in daily life. Includes overall score, overall pain rating, overall global evaluation, subcategories for dressing and grooming, arising, eating, walking, hygiene, reach, grip, and activities. Measured on scale of 0-3 from 'Without any difficulty' (0) to 'Unable to do' (3). The scores are summed and divided by the number of total categories answered to arrive at a final value.

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Childhood Health Assessment Questionnaire (CHAQ)	0.25 Units on a scale Standard Deviation 0.38

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects

The Childhood Health Assessment Questionnaire (CHAQ) is a patient self-assessment (or parent assessment) tool for how illness affects ability to function in daily life. Includes overall score, overall pain rating, overall global evaluation, subcategories for dressing and grooming, arising, eating, walking, hygiene, reach, grip, and activities. Measured on scale of 0-3 from 'Without any difficulty' (0) to 'Unable to do' (3). The scores are summed and divided by the number of total categories answered to arrive at a final value.

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Childhood Health Assessment Questionnaire (CHAQ)	0.83 Units on a scale Standard Deviation 0.74

SECONDARY outcome

Timeframe: 12 months after rilonacept initiation

Population: Analysis included all subjects

Mean z-scores of anteroposterior lumbar (AP) spine were used for comparisons

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Dual-energy X-ray Absorptiometry (DEXA) Scores	-0.12 z-score Standard Deviation 1.26

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects

Mean z-scores of anteroposterior lumbar (AP) spine were used for comparisons

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Dual-energy X-ray Absorptiometry (DEXA) Scores	-0.08 z-score Standard Deviation 1.23

SECONDARY outcome

Timeframe: 24 months after rilonacept initiation

Population: Analysis included all subjects

Pediatric Quality of Life Inventory (PedsQL)-assess functional impairment and change in treatment and health-related quality of life respectively. Responses on this scale are transformed into a 0-100 scale, with 0 being the worst value for health-related quality of life and 100 being the best possible value for health-related quality of life.

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Pediatric Quality of Life Inventory (PedsQL)	90.6 Units on a scale Standard Deviation 4.85

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects

Pediatric Quality of Life Inventory (PedsQL)-assess functional impairment and change in treatment and health-related quality of life respectively. Responses on this scale are transformed into a 0-100 scale, with 0 being the worst value for health-related quality of life and 100 being the best possible value for health-related quality of life.

Outcome measures

Outcome measures
Measure	Rilonacept Treatment n=6 Participants Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Pediatric Quality of Life Inventory (PedsQL)	86.6 Units on a scale Standard Deviation 8.51

Adverse Events

Rilonacept Treatment

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Rilonacept Treatment n=6 participants at risk Rilonacept loading dose of 4.4 mg/kg/week followed by maintenace dose of 2.2 mg/kg/week. Total duration of the study 24 months.
Blood and lymphatic system disorders Thrombocytosis	16.7% 1/6 • 24 months
Ear and labyrinth disorders Otitis Externa	16.7% 1/6 • 24 months
Ear and labyrinth disorders Otitis Media	66.7% 4/6 • 24 months
Eye disorders Conjunctivitis allergic	16.7% 1/6 • 24 months
Gastrointestinal disorders Abdominal pain	33.3% 2/6 • 24 months
Gastrointestinal disorders Constipation	16.7% 1/6 • 24 months
Gastrointestinal disorders Gastroenteritis	33.3% 2/6 • 24 months
Gastrointestinal disorders Pharyngitis	50.0% 3/6 • 24 months
Gastrointestinal disorders Vomiting	33.3% 2/6 • 24 months
General disorders Pyrexia	33.3% 2/6 • 24 months
Immune system disorders Allergy to chemicals	16.7% 1/6 • 24 months
Immune system disorders Food allergy	16.7% 1/6 • 24 months
Immune system disorders Skin reaction	16.7% 1/6 • 24 months
Infections and infestations Pneumonia	16.7% 1/6 • 24 months
Infections and infestations Sinusitis	16.7% 1/6 • 24 months
Infections and infestations Upper respiratory tract infection	100.0% 6/6 • 24 months
Investigations Blood creatine phosphokinase increased	33.3% 2/6 • 24 months
Investigations C-reactive protein increased	16.7% 1/6 • 24 months
Metabolism and nutrition disorders Hypercholesterolaemia	16.7% 1/6 • 24 months
Metabolism and nutrition disorders Hyperkalaemia	16.7% 1/6 • 24 months
Musculoskeletal and connective tissue disorders Arthralgia	33.3% 2/6 • 24 months
Musculoskeletal and connective tissue disorders Myalgia	16.7% 1/6 • 24 months
Nervous system disorders Headache	33.3% 2/6 • 24 months
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	50.0% 3/6 • 24 months
Respiratory, thoracic and mediastinal disorders Upper respiratory tract congestion	50.0% 3/6 • 24 months
Skin and subcutaneous tissue disorders Dry skin	16.7% 1/6 • 24 months
Skin and subcutaneous tissue disorders Eczema	16.7% 1/6 • 24 months
Skin and subcutaneous tissue disorders Photosensitivity reaction	16.7% 1/6 • 24 months
Skin and subcutaneous tissue disorders Pruritis	16.7% 1/6 • 24 months
Skin and subcutaneous tissue disorders Rash	83.3% 5/6 • 24 months
Skin and subcutaneous tissue disorders Seborrhoeic dermatitis	16.7% 1/6 • 24 months

Additional Information

Montealegre, Gina

National Inst of Arthritis and Musculoskeletal and Skin Diseases

Phone: +1 301 761 7747

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place