Study of PTC844 in Participants With Active Rheumatoid Arthritis

NCT07779616 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: PTC844-RA-201
• Study Type: interventional
• Target: 75
• Locations: 0 countries
• Sites: N/A

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

Keywords

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

Outcome Measures

Primary Outcomes (2)

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

  Baseline up to Week 16

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

  Baseline, Week 2

Secondary Outcomes (12)

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

  Baseline, Week 12

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

  Baseline, Week 12

• Change From Baseline in DHO Over Time

  Baseline up to Week 16

• Maximum Observed Plasma Concentration (Cmax) of PTC844

  Baseline up to Week 16

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

  Baseline up to Week 16

Study Arms (3)

PTC844 Low Dose

EXPERIMENTAL

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

Drug: PTC844

PTC844 High Dose

EXPERIMENTAL

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

Drug: PTC844

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

PTC844 DRUG

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

PTC844 High Dose; PTC844 Low Dose

Placebo DRUG

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

Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• PTC Therapeutics: lead

MeSH Terms

Conditions

Arthritis, Rheumatoid; Autoimmune Diseases

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Immune System Diseases

Central Study Contacts

Patient Advocacy

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 18, 2026
• First Posted: August 21, 2026
• Study Start: August 31, 2026
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): October 29, 2027
• Last Updated: August 21, 2026

Record last verified: 2026-08