Tofacitinib for Juvenile Arthritis

No longer recruiting at 200 trial locations

Overseen By Pfizer CT.gov Call Center

Age: < 65

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Pfizer

Must be taking: Tofacitinib

Disqualifiers: Chronic infections, HIV, Hepatitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: Variable, up to 1 year or until marketing approval

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the long-term safety and tolerability of tofacitinib, a treatment for juvenile arthritis, which causes joint pain and swelling in children. All participants will receive tofacitinib to evaluate its effects over time. Suitable candidates include children and teens who previously participated in a tofacitinib study for juvenile arthritis and still experience active symptoms requiring treatment. As a Phase 3 trial, this is the final step before FDA approval, providing participants an opportunity to contribute to the potential availability of a new treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications before starting the study drug, but it doesn't specify which ones. You can continue taking medications that are allowed by the study protocol.

Is there any evidence suggesting that tofacitinib is likely to be safe for humans?

A previous study showed that tofacitinib helps people with juvenile idiopathic arthritis (JIA). This treatment is approved for children aged 2 and older with a type of arthritis called pcJIA, indicating it is safe enough for some patients who do not respond well to other treatments.

However, serious risks must be considered. Tofacitinib carries warnings for serious infections, increased risk of death, cancer, and blood clots. These risks are important to consider when thinking about joining a trial.

Long-term studies have demonstrated that tofacitinib can be effective and generally well-tolerated. Discuss these risks with a doctor to decide if joining a trial is appropriate. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for juvenile arthritis?

Tofacitinib is unique because it works by targeting and inhibiting Janus kinases (JAKs), which play a crucial role in the inflammatory process associated with juvenile arthritis. Unlike standard treatments such as methotrexate or biologics like TNF inhibitors, which have broader immune-suppressing effects, tofacitinib offers a more targeted approach. Researchers are excited about this treatment because it has the potential to reduce inflammation more precisely and may offer an oral administration option, making it easier for young patients to take compared to injectable therapies.

What is the effectiveness track record for tofacitinib in treating juvenile arthritis?

Research has shown that tofacitinib can help treat juvenile idiopathic arthritis (JIA). In one study, 76.9% of children who started taking tofacitinib continued to see benefits compared to those who switched to a placebo, a pill with no active medicine. Another study found that 27.3% of children reached "clinically inactive disease," where symptoms were well controlled, compared to 17.6% who took a placebo. These findings suggest that tofacitinib can improve symptom control and physical function in children with JIA. All participants in this trial will receive tofacitinib. ^{2 6 7 8 9}

Who Is on the Research Team?

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Are You a Good Fit for This Trial?

This trial is for pediatric patients aged 2 to less than 18 with Juvenile Idiopathic Arthritis (JIA) who've been in previous tofacitinib studies. They should have active JIA needing treatment and use effective contraception if applicable. Excluded are those with recent serious infections, certain viral infections like HIV or hepatitis, recurrent herpes zoster, or chronic infections.

Inclusion Criteria

I am under 18, have JIA, and need tofacitinib as per my doctor's advice.

I have stopped taking certain medications as required and only take approved ones.

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.

Exclusion Criteria

I have a long-term infection.

I am infected with HIV, hepatitis B, or hepatitis C.

You have had an infected joint replacement in the past and still have the replacement in place.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 28 days

1 visit (in-person)

Baseline

Baseline assessments are conducted before starting the treatment

1 day

1 visit (in-person)

Treatment

Participants receive tofacitinib treatment with regular follow-up visits

Variable, up to 1 year or until marketing approval

Visits at 1 month, 3 months, then every 3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tofacitinib

Trial Overview The study tests the long-term safety of a medication called Tofacitinib in young patients with JIA. It's for those who've already taken part in earlier Tofacitinib trials and focuses on how well they tolerate the drug over an extended period.

How Is the Trial Designed?

Group I: Tofacitinib Experimental Treatment 1 Intervention

All patients will be in tofacitinib treatment group.

Tofacitinib is already approved in United States, European Union for the following indications:

Approved in United States as Xeljanz for:

• Rheumatoid Arthritis
• Psoriatic Arthritis
• Ulcerative Colitis
• Ankylosing Spondylitis
• Polyarticular Course Juvenile Idiopathic Arthritis

Approved in European Union as Xeljanz for:

• Rheumatoid Arthritis
• Psoriatic Arthritis
• Ulcerative Colitis
• Ankylosing Spondylitis

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38849152/

Safety and efficacy of tofacitinib for the treatment of patients ... Results: Of 225 patients with JIA (median (range) duration of treatment, 41.6 (1-103) months), 201 (89.3%) had AEs; 34 (15.1%) had serious AEs.

2. ard.bmj.com ard.bmj.com/content/83/11/1561

Safety and efficacy of tofacitinib for the treatment of patients ... Efficacy outcomes included improvement since tofacitinib initiation per the JIA-American College of Rheumatology (ACR)70/90 criteria, JIA flare rate and disease ...

3. jrheum.org jrheum.org/content/52/9/919

Clinically Inactive Disease and Remission in Patients With ... Results Of 225 patients with JIA in part 1, 173 (76.9%) were randomized in part 2 to continue tofacitinib or switch to placebo. Rates of JADAS10 ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT02592434

Efficacy Study Of Tofacitinib In Pediatric JIA Population This is a randomized withdrawal, double blind, placebo controlled study of pediatric subjects (2 to <18 years of age) with JIA.

5. rheumatologyadvisor.com rheumatologyadvisor.com/news/tofacitinib-improves-physical-function-in-jia/

Tofacitinib Improves Disease Control, Function in Juvenile ... The percentage of patients who achieved JADAS10-CID plus normal physical function reached 27.3% with tofacitinib vs 17.6% with placebo.

6. xeljanz.com xeljanz.com/pcjia/

Home Page | XELJANZ® (tofacitinib) For pcJIA | Safety Info The first and only JAK inhibitor approved to treat active pcJIA in patients 2 and older when TNF blockers did not work well or could not be tolerated.

7. ema.europa.eu ema.europa.eu/en/medicines/human/EPAR/xeljanz

Xeljanz | European Medicines Agency (EMA) Tofacitinib is indicated for the treatment of active polyarticular juvenile idiopathic arthritis (rheumatoid factor positive [RF+] or negative [RF ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT01500551?term=TOFACITINIB&rank=10

Long-Term Safety Study Of Tofacitinib In Patients With ... This is a Phase 2/3, long term, open-label, follow-up study. Subjects will have previously participated in qualifying/index JIA studies of tofacitinib.

9. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2020/203214s026lbl.pdf

Tofacitinib - accessdata.fda.gov WARNING: SERIOUS INFECTIONS, MORTALITY, MALIGNANCY. AND THROMBOSIS. See full prescribing information for complete boxed warning.