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NCT Number: NCT07779616

Study of PTC844 in Participants With Active Rheumatoid Arthritis

This study is designed to assess the safety, pharmacokinetics (PK), and biomarker effects of PTC844 compared to placebo in participants with active rheumatoid arthritis (RA).

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Key information

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Historical diagnosis of RA as per the American College of Rheumatology (ACR)/European Alliance of Associations for Rheumatology (EULAR) diagnostic criteria.
  • Active RA disease with a threshold of moderate activity or higher as confirmed via DAS28-CRP (score of ≥3.2) at screening.
  • Naïve to disease-modifying treatments for RA or have had an inadequate response to background treatment for RA and/or inability to tolerate disease-modifying antirheumatic drugs, as per investigator's discretion.
  • Disease-modifying antirheumatic drugs should be stable and unchanged from 30 days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study.
  • CRP of ≥5 milligrams (mg)/liter (L) at screening.

Key Exclusion Criteria:

  • Presence of any clinically significant abnormality at screening, or prior or ongoing medical condition (for example, concomitant illness, psychiatric condition), surgical procedure, medical history, or physical findings that, in the investigator's opinion, could adversely affect the safety of the participant or could impair the assessment of study results.
  • Participants with Class IV RA.
  • Past medical history of blood dyscrasia, bone marrow abnormality and/or suppression, drug induced thrombocytopenia or idiopathic thrombocytopenia, or bleeding diathesis.
  • Past medical history of inflammatory joint disease other than RA or fibromyalgia.
  • Rheumatoid arthritis onset at <17 years of age.
  • Past medical history of malignancy within 5 years prior to screening, except for nonmelanoma skin cancers cursed via local resection.
  • Participant has a known hypersensitivity to any of the ingredients of PTC844 or to any excipients of the study drug.
  • Use or intended use of any prescribed disease-modifying treatments for RA within 30 days or 5 half-lives (whichever is longer) before the Screening Period until completion of the Follow-Up Visit, except for methotrexate, low dose prednisone (<10 mg/day), hydroxychloroquine, or sulfasalazine, which are permitted as established background single-therapy medications.
  • Use or intended use of a combination of methotrexate, low dose prednisone (<10 mg/day), hydroxychloroquine, or sulfasalazine within 30 days or 5 half-lives (whichever is longer) before the Screening Period until completion of the Follow-Up Visit.

Note: Other protocol-defined inclusion/exclusion criteria may apply.

Treatment and study plan

PTC844

Drug

PTC844 will be administered per schedule specified in the arm description.

Placebo

Drug

Placebo matching to PTC844 will be administered per schedule specified in the arm description.

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: Baseline up to Week 16

  2. Change From Baseline to Week 2 in Dihydroorotate (DHO)

    Time frame: Baseline, Week 2

Secondary outcomes

  1. Change From Baseline to Week 12 in C-reactive Protein (CRP)

    Time frame: Baseline, Week 12

  2. Change From Baseline to Week 12 in Erythrocyte Sedimentation Rate (ESR)

    Time frame: Baseline, Week 12

  3. Change From Baseline in DHO Over Time

    Time frame: Baseline up to Week 16

  4. Maximum Observed Plasma Concentration (Cmax) of PTC844

    Time frame: Baseline up to Week 16

  5. Area Under the Concentration-time Curve From Time 0 to the Time of Last Quantifiable Concentration (AUClast)

    Time frame: Baseline up to Week 16

  6. Time to Reach Cmax (Tmax)

    Time frame: Baseline up to Week 16

  7. Number of Participants Achieving ≥20% Improvement in American College of Rheumatology Score (ACR20)

    Time frame: Week 12

  8. Number of Participants Achieving ≥50% Improvement in American College of Rheumatology Score (ACR50)

    Time frame: Week 12

  9. Number of Participants Achieving ≥70% Improvement in American College of Rheumatology Score (ACR70)

    Time frame: Week 12

  10. Change From Baseline to Week 12 in 28-Joint Disease Activity Score-CRP (DAS28-CRP)

    Time frame: Baseline, Week 12

  11. Change From Baseline to Week 12 in 28-Joint Disease Activity Score-ESR (DAS28-ESR)

    Time frame: Baseline, Week 12

  12. Change From Baseline to Week 12 in 20- Item Disability Scale and Heath Assessment Questionnaire (HAQ-DI)

    Time frame: Baseline, Week 12

Study contacts

Contact information is provided by the study sponsor or research team.

Patient Advocacy

CONTACT

[email protected]

1-866-562-4620

Sponsors and collaborators

Lead sponsor

PTC Therapeutics

Industry

Registry information

Official study title

A Phase 2A, Proof-of-Concept, Randomized, Parallel, Double-Blind, Placebo-Controlled Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of PTC844 in Participants With Active Rheumatoid Arthritis

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Aug 21, 2026
Registry last updated
Aug 21, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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