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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01500551




Registration number
NCT01500551
Ethics application status
Date submitted
22/12/2011
Date registered
28/12/2011
Date last updated
8/07/2019

Titles & IDs
Public title
Long-Term Safety Study Of Tofacinib In Patients With Juvenile Idiopathic Arthritis
Scientific title
A LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA)
Secondary ID [1] 0 0
2011-004915-22
Secondary ID [2] 0 0
A3921145
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Juvenile Idiopathic Arthritis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Osteoarthritis
Inflammatory and Immune System 0 0 0 0
Rheumatoid arthritis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tofacitinib
Treatment: Drugs - Tofacitinib
Treatment: Drugs - Tofacitinib

Experimental: Tofacitinib - All patients will be in tofacitinib treatment group.


Treatment: Drugs: Tofacitinib
Tofacitinib will be administered orally BID (twice daily) approximately 12 hours (±2 hours) apart, once in the morning and once in the evening, based on body weight.
5 mg BID Dose Level:
Body Weight (Dose in tablet [mg BID] or solution [ml BID]) 5 - < 7 kg (2 mg or 2 ml) 7 - < 10 kg (2.5 mg or 2.5 ml) 10 - <15 kg (3 mg or 3 ml) 15 - <25 kg (3.5 mg or 3.5 ml) 25 - <40 kg (4 mg or 4 ml) >=40 kg (5 mg or 5 ml)
Oral solution (1 mg/mL concentration) will be used for subjects weighing <40 kg. Oral tablets (5 mg) will be used for subjects weighing >=40 kg; subjects who are unable to swallow tablets will have the option of taking oral solution.
Subjects will swallow study tablets whole and will not manipulate or chew tablets prior to swallowing.

Treatment: Drugs: Tofacitinib
For subjects rolling over from study A3921103 and actively participating in this study at the time of Protocol Amendment 6 and receiving a dosage of tofacitinib in accordance with the dosing scheme specified in Protocol Amendment 5, investigators will have the option of maintaining the subject's current dosage regimen from index study A3921103 (if the desired clinical response has been attained with no safety concern) or adjusting the dosage regimen in accordance with the dosing scheme specified in this section.

Treatment: Drugs: Tofacitinib
For participants rolling over from study A3921165: 10 mg BID Dose Level
Body Weight (Dose in tablet [mg BID] or solution [ml BID])
5 - < 7 kg (4 mg or 4 ml) 7 - < 10 kg (5 mg or 5 ml) 10 - <15 kg (6 mg or 6 ml) 15 - <25 kg (7 mg or 7 ml) 25 - <40 kg (8 mg or 8 ml) >=40 kg (10 mg or 10 ml)
Oral solution (1 mg/mL concentration) will be used for subjects weighing <40 kg. Oral tablets (5 mg) will be used for subjects weighing >=40 kg; subjects who are unable to swallow tablets will have the option of taking oral solution.
Subjects will swallow study tablets whole and will not manipulate or chew tablets prior to swallowing.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Standard laboratory safety data and adverse event (AE) reports. Body weight, height and Tanner Stages will collected to assess growth and physical development.
Timepoint [1] 0 0
up to 8 years
Secondary outcome [1] 0 0
Physician global evaluation of disease activity at each visit.
Timepoint [1] 0 0
up to 8 years
Secondary outcome [2] 0 0
Number of joints with active arthritis at each visit.
Timepoint [2] 0 0
up to 8 years
Secondary outcome [3] 0 0
Number of joints with limitation of motion at each visit.
Timepoint [3] 0 0
up to 8 years
Secondary outcome [4] 0 0
Index of inflammation (C-reactive protein [CRP] and Erythrocyte Sedimentation Rate [ESR]) at each visit.
Timepoint [4] 0 0
up to 8 years
Secondary outcome [5] 0 0
Parent's Assessment of Physical Function (Childhood Health Assessment Questionnaire [CHAQ]Disability Index)at each visit.
Timepoint [5] 0 0
up to 8 years
Secondary outcome [6] 0 0
Parent's Assessment of Child's Arthritis Pain (Childhood Health Assessment Questionnaire [CHAQ] Discomfort Index, Visual Analog Scale [VAS])at each visit.
Timepoint [6] 0 0
up to 8 years
Secondary outcome [7] 0 0
Parent's Global Assessment of Overall Wellbeing (Childhood Health Assessment Questionnaire [CHAQ] subsection, Visual Analog Scale [VAS])at each visit.
Timepoint [7] 0 0
up to 8 years
Secondary outcome [8] 0 0
JIA American College of Rheumatology (ACR) response and occurrence of JIA ACR disease flare at each visit.
Timepoint [8] 0 0
up to 8 years
Secondary outcome [9] 0 0
JIA ACR Clinical Inactive Disease status and Clinical Remission on Medication at each visit.
Timepoint [9] 0 0
up to 8 years
Secondary outcome [10] 0 0
Change from baseline in Juvenile Arthritis Disease Activity Score (JADAS) 27- CRP and JADAS 27-ESR, and occurrence of JADAS minimum disease activity and inactive disease at each visit.
Timepoint [10] 0 0
up to 8 years
Secondary outcome [11] 0 0
In subjects with Enthesitis Related Arthritis (ERA): Change from baseline in the Tender Entheseal Assessment, Modified Schober's Test, Overall Back Pain and Nocturnal Back Pain responses at various visits.
Timepoint [11] 0 0
up to 8 years
Secondary outcome [12] 0 0
In subjects with psoriatic arthritis (PsA): Change from baseline in body surface area (BSA) affected by psoriasis and Physician's Global Assessment (PGA) of psoriasis) at various visits.
Timepoint [12] 0 0
up to 8 years
Secondary outcome [13] 0 0
In subjects with sJIA: "Absence of Fever", defined as absence of fever due to sJIA in the week preceding the assessment at each visit.
Timepoint [13] 0 0
up to 8 years
Secondary outcome [14] 0 0
Eligibility of tapering defined per protocol for corticosteroids
Timepoint [14] 0 0
up to 8 years
Secondary outcome [15] 0 0
Eligibility of tapering defined per protocol for methotrexate
Timepoint [15] 0 0
up to 8 years
Secondary outcome [16] 0 0
Eligibility of tapering defined per protocol for leflunomide
Timepoint [16] 0 0
Up to 8 years
Secondary outcome [17] 0 0
Eligibility of tapering defined per protocol for tofacitinib
Timepoint [17] 0 0
Up to 8 years

Eligibility
Key inclusion criteria
- Pediatric subjects with JIA aged from 2 to less than 18 years who met entry criteria
for the qualifying/index study and in the opinion of the investigator have sufficient
evidence of JIA disease activity to warrant use of tofacitinib as a DMARD. Subjects
turning 18 years of age during participation in the qualifying/index study or
subsequently will be eligible for participation in this study.

- The subject has discontinued disallowed concomitant medications for the required time
prior to the first dose of study drug, as defined in Appendix 1, and is taking only
those concomitant medications in doses and frequency allowed by the protocol.

- Fertile male subjects and female subjects of childbearing potential who are, in the
opinion of the investigator, sexually active and at risk for pregnancy with their
partner(s) must be using a highly effective method of contraception as outlined in
this protocol throughout the study and for at least 28 days after the last dose of
study medication.

- Subjects must have previously completed participation in a qualifying study of
tofacitinib for the treatment of JIA. Subjects who have required earlier
discontinuation of treatment in a qualifying study for reasons other than tofacitinib
related serious adverse events may be eligible.
Minimum age
2 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Systemic JIA (sJIA) with active systemic features other than active joints and
elevated acute phase reactants, persistent oligoarthritis, and undifferentiated JIA.

- Infections:

1. Chronic infections.

2. Any infection requiring hospitalization, parenteral antimicrobial therapy or
judged to be opportunistic by the investigator within the 6 months prior to the
first dose of study drug.

3. Any treated infections within 2 weeks of baseline visit.

4. A subject known to be infected with human immunodeficiency virus (HIV), hepatitis
B or hepatitis C virus.

5. History of infected joint prosthesis with prosthesis still in situ.

- History of recurrent (more than one episode) herpes zoster or disseminated (a single
episode) herpes zoster or disseminated (a single episode) herpes simplex.

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
The Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
District of Columbia
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Georgia
Country [8] 0 0
United States of America
State/province [8] 0 0
Illinois
Country [9] 0 0
United States of America
State/province [9] 0 0
Indiana
Country [10] 0 0
United States of America
State/province [10] 0 0
Massachusetts
Country [11] 0 0
United States of America
State/province [11] 0 0
Minnesota
Country [12] 0 0
United States of America
State/province [12] 0 0
New Jersey
Country [13] 0 0
United States of America
State/province [13] 0 0
New York
Country [14] 0 0
United States of America
State/province [14] 0 0
North Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Ohio
Country [16] 0 0
United States of America
State/province [16] 0 0
Oregon
Country [17] 0 0
United States of America
State/province [17] 0 0
Pennsylvania
Country [18] 0 0
United States of America
State/province [18] 0 0
Texas
Country [19] 0 0
United States of America
State/province [19] 0 0
Utah
Country [20] 0 0
United States of America
State/province [20] 0 0
Washington
Country [21] 0 0
Argentina
State/province [21] 0 0
Buenos Aires
Country [22] 0 0
Argentina
State/province [22] 0 0
Santa FE
Country [23] 0 0
Argentina
State/province [23] 0 0
Tucuman
Country [24] 0 0
Belgium
State/province [24] 0 0
Leuven
Country [25] 0 0
Brazil
State/province [25] 0 0
BA
Country [26] 0 0
Brazil
State/province [26] 0 0
Minas Gerais
Country [27] 0 0
Brazil
State/province [27] 0 0
Parana
Country [28] 0 0
Brazil
State/province [28] 0 0
Paraná
Country [29] 0 0
Brazil
State/province [29] 0 0
RIO Grande DO SUL
Country [30] 0 0
Brazil
State/province [30] 0 0
SAO Paulo
Country [31] 0 0
Brazil
State/province [31] 0 0
SÃO Paulo
Country [32] 0 0
Brazil
State/province [32] 0 0
Rio de Janeiro
Country [33] 0 0
Brazil
State/province [33] 0 0
Sao Paulo
Country [34] 0 0
Canada
State/province [34] 0 0
Alberta
Country [35] 0 0
Canada
State/province [35] 0 0
British Columbia
Country [36] 0 0
Canada
State/province [36] 0 0
Ontario
Country [37] 0 0
Canada
State/province [37] 0 0
Quebec
Country [38] 0 0
Chile
State/province [38] 0 0
Concepcion
Country [39] 0 0
Chile
State/province [39] 0 0
Santiago
Country [40] 0 0
China
State/province [40] 0 0
Chongqing
Country [41] 0 0
China
State/province [41] 0 0
Shanghai
Country [42] 0 0
Germany
State/province [42] 0 0
Bad Bramstedt
Country [43] 0 0
Germany
State/province [43] 0 0
Bremen
Country [44] 0 0
Germany
State/province [44] 0 0
Hamburg
Country [45] 0 0
Germany
State/province [45] 0 0
Heidelberg
Country [46] 0 0
Germany
State/province [46] 0 0
Sankt Augustin
Country [47] 0 0
Israel
State/province [47] 0 0
Haifa
Country [48] 0 0
Israel
State/province [48] 0 0
Kfar Saba
Country [49] 0 0
Israel
State/province [49] 0 0
Ramat Gan
Country [50] 0 0
Mexico
State/province [50] 0 0
Jalisco
Country [51] 0 0
Mexico
State/province [51] 0 0
Nuevo LEON
Country [52] 0 0
Mexico
State/province [52] 0 0
San Luis Potosi
Country [53] 0 0
Mexico
State/province [53] 0 0
San Luis Potosí
Country [54] 0 0
Poland
State/province [54] 0 0
Bydgoszcz
Country [55] 0 0
Poland
State/province [55] 0 0
Krakow
Country [56] 0 0
Poland
State/province [56] 0 0
Lodz
Country [57] 0 0
Poland
State/province [57] 0 0
Sosnowiec
Country [58] 0 0
Russian Federation
State/province [58] 0 0
Republic OF Bashkortostan
Country [59] 0 0
Russian Federation
State/province [59] 0 0
Kazan
Country [60] 0 0
Russian Federation
State/province [60] 0 0
Moscow
Country [61] 0 0
Russian Federation
State/province [61] 0 0
Saint-Petersburg
Country [62] 0 0
Russian Federation
State/province [62] 0 0
Saratov
Country [63] 0 0
Russian Federation
State/province [63] 0 0
Tolyatti
Country [64] 0 0
Russian Federation
State/province [64] 0 0
Yekaterinburg
Country [65] 0 0
Slovakia
State/province [65] 0 0
Piestany
Country [66] 0 0
South Africa
State/province [66] 0 0
Cape Town
Country [67] 0 0
South Africa
State/province [67] 0 0
Gauteng
Country [68] 0 0
Spain
State/province [68] 0 0
Madrid
Country [69] 0 0
Spain
State/province [69] 0 0
Valencia
Country [70] 0 0
Turkey
State/province [70] 0 0
Ankara
Country [71] 0 0
Turkey
State/province [71] 0 0
Istanbul
Country [72] 0 0
Turkey
State/province [72] 0 0
Kayseri
Country [73] 0 0
Ukraine
State/province [73] 0 0
Dnipro
Country [74] 0 0
Ukraine
State/province [74] 0 0
Ivano-Frankivsk
Country [75] 0 0
Ukraine
State/province [75] 0 0
Kharkiv
Country [76] 0 0
Ukraine
State/province [76] 0 0
Lviv
Country [77] 0 0
Ukraine
State/province [77] 0 0
Vinnitsya
Country [78] 0 0
United Kingdom
State/province [78] 0 0
WEST Midlands

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Evaluate long-term safety and tolerability of tofacitinib in patients with JIA, who have
previously participated in tofacitinib JIA studies.
Trial website
https://clinicaltrials.gov/show/NCT01500551
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Country 0 0
Phone 0 0
1-800-718-1021
Fax 0 0
Email 0 0
ClinicalTrials.gov_Inquiries@pfizer.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT01500551