NCT07779616尚未招募2 期
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
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 75
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
PTC844 Low Dose
Experimental
Participants will receive PTC844 at a low dose once daily (QD) orally for 12 weeks.
干预措施: PTC844 (Drug)
PTC844 High Dose
Experimental
Participants will receive PTC844 at a high dose QD orally for 12 weeks.
干预措施: PTC844 (Drug)
Placebo
Placebo Comparator
Participants will receive placebo matching to PTC844 QD orally for 12 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
时间窗: Baseline up to Week 16
Change From Baseline to Week 2 in Dihydroorotate (DHO)
时间窗: Baseline, Week 2
次要结局
- 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)
- Time to Reach Cmax (Tmax)(Baseline up to Week 16)
- Number of Participants Achieving ≥20% Improvement in American College of Rheumatology Score (ACR20)(Week 12)
- Number of Participants Achieving ≥50% Improvement in American College of Rheumatology Score (ACR50)(Week 12)
- Number of Participants Achieving ≥70% Improvement in American College of Rheumatology Score (ACR70)(Week 12)
- Change From Baseline to Week 12 in 28-Joint Disease Activity Score-CRP (DAS28-CRP)(Baseline, Week 12)
- Change From Baseline to Week 12 in 28-Joint Disease Activity Score-ESR (DAS28-ESR)(Baseline, Week 12)
- Change From Baseline to Week 12 in 20- Item Disability Scale and Heath Assessment Questionnaire (HAQ-DI)(Baseline, Week 12)
研究者
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