Trial Outcomes & Findings for Anakinra for Behcet s Disease (NCT NCT01441076)

NCT ID: NCT01441076

Last Updated: 2017-05-12

Results Overview

Clinical remission was defined as no oral or vaginal ulcers on physical examination for 2 consecutive monthly visits from months 3-6.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 6 participants
• Primary outcome timeframe: Monthly study visits from months 3-6 during the trial
• Results posted on: 2017-05-12

Participant Flow

Participant milestones

Measure	Anakinra Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Overall Study STARTED	6
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Anakinra for Behcet s Disease

Baseline characteristics by cohort

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Age, Categorical LTE18	0 Participants n=5 Participants
Age, Categorical BTWN	6 Participants n=5 Participants
Age, Categorical GTE65	0 Participants n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 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) Hawaiian	0 Participants n=5 Participants
Race (NIH/OMB) Black	1 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Monthly study visits from months 3-6 during the trial

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Clinical remission was defined as no oral or vaginal ulcers on physical examination for 2 consecutive monthly visits from months 3-6.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Clinical Remission From Months 3-6	2 Participants

SECONDARY outcome

Timeframe: Month 6 study visit

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

The BDRQOL is a standardized assessment form in Behcet's disease composed of 30 items (answered true or not true) and each item is scored 0 or 1 (scoring range from 0 to 30). A total lower score indicates a better quality of life and a total higher score indicates a worse quality of life.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Behcet's Disease Related Quality of Life (BDRQOL) Assessment Score	9.5 Units on a scale Interval 0.0 to 22.0

SECONDARY outcome

Timeframe: Month 6 study visit

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

The BSAS is a standardized assessment form in Behcet's disease. The BSAS score comprises 10 items. The total score possible is between 0-100. A total lower score indicates a less syndrome activity and a higher total score indicates more syndrome activity.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Behcet's Syndrome Activity Scale (BSAS) Score	21.3 Units on a scale Interval 0.0 to 44.0

SECONDARY outcome

Timeframe: Month 6 study visit

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

The BDCAF is a standardized assessment form in Behcet's disease to measure patient activity. Scoring is based on the history of new clinical features present over the preceding 4 weeks prior to assessment. The range of score is 0 - 12. A total lower score indicates less disease activity and a higher total score indicates more disease activity.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Behcets Disease Current Activity Form (BDCAF) Score	5 Units on a scale Interval 4.0 to 6.0

SECONDARY outcome

Timeframe: Month 6 study visit

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Number of genital ulcers noted by physician evaluation

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Number of Genital Ulcers by Physician Evaluation	0 genital ulcers in participants Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Month 6 study visit

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Number of oral ulcers noted by physician evaluation

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Number of Oral Ulcers by Physician Evaluation	0.5 oral ulcers in participants Interval 0.0 to 2.0

SECONDARY outcome

Timeframe: Month 6 study visit

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Global visual analogue scale taken by patients with a range of score of 0-100. Lower scores indicate least symptoms and higher scores indicate worst symptoms faced by the patient.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Patient Global Score	51.5 Units on a scale Interval 3.0 to 91.0

SECONDARY outcome

Timeframe: Month 6 study visit

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Global visual analogue scale administered by physicians with a range of score of 0-100. Lower scores indicate least symptoms and higher scores indicate worst symptoms faced by the patient.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Physician Global Score	8.5 Units on a scale Interval 7.0 to 24.0

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

The BDRQOL is a standardized assessment form in Behcet's disease composed of 30 items (answered true or not true) and each item is scored 0 or 1 (scoring range from 0 to 30). A total lower score indicates a better quality of life and a total higher score indicates a worse quality of life.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Behcet's Disease Related Quality of Life (BDRQOL) Assessment Score	16 Units on a scale Interval 10.0 to 24.0

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

The BSAS is a standardized assessment form in Behcet's disease. The BSAS score comprises 10 items. The total score possible is between 0-100. A total lower score indicates a less syndrome activity and a higher total score indicates more syndrome activity.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Behcet's Syndrome Activity Scale (BSAS) Score	46 Units on a scale Interval 22.0 to 80.5

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

The BDCAF is a standardized assessment form in Behcet's disease to measure patient activity. Scoring is based on the history of new clinical features present over the preceding 4 weeks prior to assessment. The range of score is 0 - 12. A total lower score indicates less disease activity and a higher total score indicates more disease activity.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Behcets Disease Current Activity Form (BDCAF) Score	6 Units on a scale Interval 5.0 to 11.0

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Number of genital ulcers noted by physician evaluation

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Number of Genital Ulcers by Physician Evaluation	0 genital ulcers in participants Interval 0.0 to 3.0

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Number of oral ulcers noted by physician evaluation

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Number of Oral Ulcers by Physician Evaluation	3.5 oral ulcers in participants Interval 2.0 to 5.0

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Global visual analogue scale taken by patients with a range of score of 0-100. Lower scores indicate least symptoms and higher scores indicate worst symptoms faced by the patient.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Patient Global Score	73.5 Units on a scale Interval 9.0 to 90.0

SECONDARY outcome

Timeframe: Baseline

Population: Analysis included all subjects who enrolled in the trial, including those who terminated the study prior to month 6 due to perceived lack of efficacy of study drug.

Global visual analogue scale administered by physicians with a range of score of 0-100. Lower scores indicate least symptoms and higher scores indicate worst symptoms faced by the patient.

Outcome measures

Measure	Anakinra n=6 Participants Treatment with Anakinra 100mg subcutaneous daily with option to escalate dose up to 300mg subcutaneous daily
Physician Global Score	16.5 Units on a scale Interval 12.0 to 79.0

Adverse Events

Anakinra

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Measure	Anakinra n=6 participants at risk Treatment with Anakinra 100mg subcutaneous daily
Musculoskeletal and connective tissue disorders Chest wall pain	16.7% 1/6
Nervous system disorders Presyncope	16.7% 1/6
Respiratory, thoracic and mediastinal disorders Pulmonary hypertension	16.7% 1/6
Vascular disorders Thromboembolic event	16.7% 1/6

Other adverse events

Measure	Anakinra n=6 participants at risk Treatment with Anakinra 100mg subcutaneous daily
Blood and lymphatic system disorders Anemia	16.7% 1/6
Eye disorders Blurred vision	16.7% 1/6
Eye disorders Eye disorders - Other, specify	16.7% 1/6
Gastrointestinal disorders Abdominal pain	33.3% 2/6
General disorders Edema limbs	33.3% 2/6
General disorders Fatigue	16.7% 1/6
Immune system disorders Allergic reaction	66.7% 4/6
Infections and infestations Bronchial Infection	16.7% 1/6
Infections and infestations Infections and infestations - Other, specify	16.7% 1/6
Infections and infestations Pharyngitis	16.7% 1/6
Infections and infestations Rhinitis infective	33.3% 2/6
Infections and infestations Upper respiratory infection	33.3% 2/6
Infections and infestations Vaginal infection	16.7% 1/6
Infections and infestations Vulval infection	16.7% 1/6
Metabolism and nutrition disorders Hypoglycemia	16.7% 1/6
Musculoskeletal and connective tissue disorders Arthralgia	16.7% 1/6
Musculoskeletal and connective tissue disorders Pain in extremity	16.7% 1/6
Nervous system disorders Dizziness	16.7% 1/6
Psychiatric disorders Mania	16.7% 1/6
Renal and urinary disorders Urinary Frequency	16.7% 1/6
Respiratory, thoracic and mediastinal disorders Sore throat	16.7% 1/6
Skin and subcutaneous tissue disorders Alopecia	33.3% 2/6
Vascular disorders Phlebitis	16.7% 1/6

Additional Information

Grayson, Peter

National Inst of Arthritis and Musculoskeletal and Skin Diseases

Phone: +1 301 827 9187

Email: peter.grayson@nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place