Recruiting
Phase 2

PTC844

Sponsor:

PTC Therapeutics

Code:

NCT07779616

Conditions

Rheumatoid Arthritis

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

Interventions

PTC844

Placebo

Study Details

Brief summary:

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).

Conditions

Rheumatoid Arthritis

Study ID

NCT07779616

Start date

Sep 24, 2026

Status verified date

Oct, 2026

Completion date

Oct 29, 2027

Anticipated

Primary completion date

Sep 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

75 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: PTC844 Low Dose

Participants will receive PTC844 at a low dose once daily (QD) orally for 12 weeks.

experimental: PTC844 High Dose

Participants will receive PTC844 at a high dose QD orally for 12 weeks.

placebo comparator: Placebo

Participants will receive placebo matching to PTC844 QD orally for 12 weeks.

Interventions

PTC844

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

Placebo

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

Primary outcome measure

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Baseline up to Week 16 ]
  • Change From Baseline to Week 2 in Dihydroorotate (DHO) [ Time Frame: Baseline, Week 2 ]

Central Contacts and Locations

Central contacts

Patient Advocacy

1-866-562-4620medinfo@ptcbio.com

Locations

AARA Clinical Research - Arizona Arthritis and Rheumatology Associates - Chandler

Recruiting

Chandler, Arizona, United States, 85210

Principal Investigator:

Amer Al-Khoudari

Millennium Clinical Trials

Recruiting

Westlake Village, California, United States, 91361

Principal Investigator:

Tony Makhlouf

Nouvelle Clinical Research LLC

Recruiting

Cutler Bay, Florida, United States, 33189

Principal Investigator:

Rawan Jumean

Millennium Medical Research, LLC.

Recruiting

Miami, Florida, United States, 33126

Principal Investigator:

Ramon Moreda

Conquest Research

Recruiting

Winter Park, Florida, United States, 32789

Principal Investigator:

Anand Patel

Precision Comprehensive Clinical Research Solutions - Irving

Recruiting

Irving, Texas, United States, 76051

Principal Investigator:

Renuka Basavaraju

More Information

Sponsor

PTC Therapeutics

Last update posted

Nov 6, 2026

Last verified

Oct, 2026

Keywords

  • Immune-mediated disease
  • Autoimmune disease
  • Dihydroorotate dehydrogenase (DHODH) inhibitors
  • PTC844
  • Rheumatoid arthritis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-11-07. This information was provided to ClinicalTrials.gov by PTC Therapeutics on 2026-11-06. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.