Trial Outcomes & Findings for Human Anti-Tac (Daclizumab) to Treat Juvenile Idiopathic Arthritis (JIA)-Associated Uveitis (NCT NCT00130637)

Last Updated: 2017-01-30

Results Overview

Number of participants with a two-step reduction (or down to 0 out of a scale of 0 to 4+) of anterior chamber (AC) inflammation according to Standardization of Uveitis Nomenclature (SUN) criteria, while on a topical corticosteroid schedule of less than 3 times a day. Grade 0 is the best score on this scale with \<1 cell in the field and 4+ is the worst score on this scale with \>50 cells in the field.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

12 weeks

Results posted on

2017-01-30

Participant Flow

single center, 6 participants

pilot, non-randomized, open-label trial

Participant milestones

Participant milestones
Measure	Daclizumab An induction regimen of intravenous (IV) daclizumab at 8 mg/kg was given on Day 0 followed by another IV dose of 4 mg/kg at Day 14, provided the safety endpoint was not met. Participants who showed a two-step reduction in their ocular inflammation or a decrease to inactivity, without serious adverse events, had the option to receive extended treatments of 2 mg/kg IV daclizumab treatments at 4-week intervals, beginning day 28, for up to a total of 52 weeks.
Overall Study STARTED	6
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Daclizumab An induction regimen of intravenous (IV) daclizumab at 8 mg/kg was given on Day 0 followed by another IV dose of 4 mg/kg at Day 14, provided the safety endpoint was not met. Participants who showed a two-step reduction in their ocular inflammation or a decrease to inactivity, without serious adverse events, had the option to receive extended treatments of 2 mg/kg IV daclizumab treatments at 4-week intervals, beginning day 28, for up to a total of 52 weeks.
Overall Study Adverse Event	2
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Human Anti-Tac (Daclizumab) to Treat Juvenile Idiopathic Arthritis (JIA)-Associated Uveitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Daclizumab n=6 Participants An induction regimen of intravenous (IV) daclizumab at 8 mg/kg was given on Day 0 followed by another IV dose of 4 mg/kg at Day 14, provided the safety endpoint was not met. Participants who showed a two-step reduction in their ocular inflammation or a decrease to inactivity, without serious adverse events, had the option to receive extended treatments of 2 mg/kg IV daclizumab treatments at 4-week intervals, beginning day 28, for up to a total of 52 weeks.
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
Age, Continuous	10.2 years STANDARD_DEVIATION 4.8 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Outcome measures

Outcome measures
Measure	Daclizumab n=6 Participants An induction regimen of intravenous (IV) daclizumab at 8 mg/kg was given on Day 0 followed by another IV dose of 4 mg/kg at Day 14, provided the safety endpoint was not met. Participants who showed a two-step reduction in their ocular inflammation or a decrease to inactivity, without serious adverse events, had the option to receive extended treatments of 2 mg/kg IV daclizumab treatments at 4-week intervals, beginning day 28, for up to a total of 52 weeks.
Number of Participants With a Two-step Reduction in Inflammation	5 participants

PRIMARY outcome

Timeframe: 52 weeks

Safety of acute daclizumab use in JIA-associated uveitis was assessed through serious adverse events (SAE).

Outcome measures

Outcome measures
Measure	Daclizumab n=6 Participants An induction regimen of intravenous (IV) daclizumab at 8 mg/kg was given on Day 0 followed by another IV dose of 4 mg/kg at Day 14, provided the safety endpoint was not met. Participants who showed a two-step reduction in their ocular inflammation or a decrease to inactivity, without serious adverse events, had the option to receive extended treatments of 2 mg/kg IV daclizumab treatments at 4-week intervals, beginning day 28, for up to a total of 52 weeks.
Number of Participants Reporting a Serious Adverse Event (SAE)	1 participant

Adverse Events

Daclizumab

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Daclizumab n=6 participants at risk An induction regimen of intravenous (IV) daclizumab at 8 mg/kg was given on Day 0 followed by another IV dose of 4 mg/kg at Day 14, provided the safety endpoint was not met. Participants who showed a two-step reduction in their ocular inflammation or a decrease to inactivity, without serious adverse events, had the option to receive extended treatments of 2 mg/kg IV daclizumab treatments at 4-week intervals, beginning day 28, for up to a total of 52 weeks.
Cardiac disorders palpitations	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".

Other adverse events

Other adverse events
Measure	Daclizumab n=6 participants at risk An induction regimen of intravenous (IV) daclizumab at 8 mg/kg was given on Day 0 followed by another IV dose of 4 mg/kg at Day 14, provided the safety endpoint was not met. Participants who showed a two-step reduction in their ocular inflammation or a decrease to inactivity, without serious adverse events, had the option to receive extended treatments of 2 mg/kg IV daclizumab treatments at 4-week intervals, beginning day 28, for up to a total of 52 weeks.
Infections and infestations herpes zoster skin infection	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders lower extremity edema	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Immune system disorders Allergy symptoms	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Musculoskeletal and connective tissue disorders Ankle stiffness	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Aphtous ulcer on right lower gum	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Back Pain	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Musculoskeletal and connective tissue disorders Bilateral Hand and wrists inflammation, Mild Pain	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Cold Symptoms/Sore throat	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Gastrointestinal disorders Decreased appetite with associated nausea	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Blood and lymphatic system disorders Decreased Hgb and Hct	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Metabolism and nutrition disorders Elevated AST	50.0% 3/6 • Number of events 3 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Metabolism and nutrition disorders Elevated Cholesterol level	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Metabolism and nutrition disorders Elevated CPK	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Musculoskeletal and connective tissue disorders Elevated ESR	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Metabolism and nutrition disorders Elevated potassium level	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Renal and urinary disorders Elevated Urine WBC	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Blood and lymphatic system disorders Elevated WBC	33.3% 2/6 • Number of events 2 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Erythematous area of Lower Eyelid	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Stomach virus	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Gum abscess	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Headache	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Headaches/Back Pain	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Hot Flashes	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Knee Pain	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Blood and lymphatic system disorders Low Hgb and Hct	33.3% 2/6 • Number of events 2 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Blood and lymphatic system disorders Low platelets	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Blood and lymphatic system disorders Low White Blood Count	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Mild myalgia in legs	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Mild pain left knee and hip after playing soccer.	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders mild right shoulder pain	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Mild shoulder pain	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Mouth Sores	16.7% 1/6 • Number of events 2 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Mucous membrane Ulcers	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Nasal congestion causing right ear pain	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Neck and Lower back pain	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Blood and lymphatic system disorders Nose bleed	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Renal and urinary disorders Occasional dysuria	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Cardiac disorders Occasional Light headiness	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Pain Right great toe	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Painful Right Wrist	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Persistent cough	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Photophobia	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Hepatobiliary disorders Planter tenderness	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Pustules over abdominal area	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Right knee pain without inflammation	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Musculoskeletal and connective tissue disorders Right Thumb sore and swollen	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Right Knee Pain few hrs after prolonged exertion	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Runny Nose	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Gastrointestinal disorders Ruptured blister inner lower lip	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Rash	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Metabolism and nutrition disorders Slight increase in total bilirubin	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Slightly Swollen right anterior pilla of tonsil	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Sore Throat, fever, body ache	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Sore Throat, Low grade fever, Malaise	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Eye disorders Squinting OU	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Stomach Flu	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Stuffy Nose and Sore Throat	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Musculoskeletal and connective tissue disorders Systemic Joint Inflammation, & assoc. joint pain.	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Tonsilitis with associated fever	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
General disorders Tooth Extraction	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Renal and urinary disorders Slight Leukocyte esterase increase	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Upper respiratory Infection	33.3% 2/6 • Number of events 2 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Gastrointestinal disorders Vomiting	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Throat infection	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".
Infections and infestations Slight cold	16.7% 1/6 • Number of events 1 • Adverse event data were collected during the 52 week study period Event descriptions were not mapped to a standardized term. General disorders was chosen as the organ system for events categorized as "Other" or "Pain".

Additional Information

H. Nida Sen, MD, MHS

National Eye Institute, NIH

Phone: 301-435-5139

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place