A randomized, double blind, placebo-controlled, phase II, multicentre trial to explore the efficacy and safety of oral AP1189 tablets administered at the dose 100 mg/day for 12 weeks in patients diagnosed with polymyalgia rheumatica and in remission on glucocorticoid

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Synact Pharma ApS

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Decision date (initial)

2026-03-19

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To explore the safety and tolerability of oral AP1189 tablets in patients with PMR. To explore the efficacy of oral 100 mg/day AP1189 tablets over a 12-week treatment period in patients with PMR.

Secondary objectives 1

1. To investigate the effect of 100 mg/day AP1189 tablets against placebo tablets by evaluating the proportion of patients in glucocorticoid free remission at week.

Conditions and MedDRA coding

Polymyalgia rheumatica

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1. Signed and dated informed consent obtained before undergoing any trial-specific procedure. 2. Male or female aged ≥50 years. 3. Patients diagnosed with PMR fulfilling the 2010 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) classification criteria. 4. Treatment with glucocorticoid for PMR initiated at diagnosis 4 weeks beforefore baseline. 5. Patients in clinical remission at baseline, defined as absence of PMR activity evaluated by the investigator based on symptoms and clinical examination combined with a CRP level be-low 8.0 mg/l. 6. No Giant cell arteritis (GCA) on vascular ultrasound at diagnosis or screening. 7. Patients with C-Reactive Protein (CRP) ≥8 mg/L at the time of diagnosis. 8. Willing and able to comply with the scheduled study visits, the treatment plan, and all study procedures. 9. Females of childbearing potential must have a negative pregnancy test at screening and again at baseline. 10. Sexually active female patients of childbearing potential and male patients must use a highly effective method of birth control (hormonal contraceptives, intrauterine device, vasectomy, bilateral tubal occlusion, sexual abstinence) with their partner during the study and for 90 days after the last dose of study drug or who will remain abstinent during the study and for 90 days after the last dose.

Exclusion criteria 1

1. 1. Previous treatment with glucocorticoids for GCA. 2. Ongoing treatment with oral/intravenous/intramuscular glucocorticoids for other diseases than PMR. 3. Other inflammatory rheumatic diseases (e.g., rheumatoid arthritis, polymyositis, spondyloar-thritis, psoriatic arthritis, gout). 4. Symptoms or findings of GCA (newly onset-headache, tenderness of the temporal artery, jaw claudication, vision disturbances, limb claudication). 5. Non-inflammatory type of musculoskeletal condition (e.g., osteoarthritis or fibromyalgia) that in the Investigator's opinion is symptomatic and severe enough to interfere with the subject's primary diagnosis of PMR or the evaluation of the effect of the study drug. 6. Gastrointestinal diseases that, in the opinion of the Investigator, may interfere with the absorption or excretion of medications. 7. Severe, progressive, or uncontrolled renal (including CKD stage 4 or higher), hepatic, hematologic, gastrointestinal, metabolic, endocrine, pulmonary, cardiac or neurologic disease. 8. Malignancy (with the exception of adequately treated or excised non-metastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ) active during the 5 years preceding the Screening Visit. 9. Any other clinically relevant disease and condition that, in the opinion of the Investigator, may jeopardize efficacy or safety assessments or may compromise the subject’s safety during trial participation. 10. Females who are pregnant or lactating. 11. Participation in any other study involving investigational drug(s) within 4 weeks prior to study entry.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety evaluations include adverse event monitoring, physical examinations, vital sign measurements, and clinical laboratory testing.

Secondary endpoints 12

1. To investigate the effect of 100 mg/day AP1189 tablets against placebo tablets by evaluating the proportion of patients in glucocorticoid free remission at week
2. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: • Accumulated glucocorticoid dosage from baseline to week 12. • Time to first relapse from baseline to week 12. • Glucocorticoid free remission at week 12 (Remission defined as PMR-AS<10).
3. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: PMR activity score at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest.
4. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: Patient global VAS at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest
5. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: SF-36 MCS at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest
6. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: SF-36 PCS at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest
7. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: HAQ-DI at week 4, 8, and 12 with the change from baseline to week 12 being of primary in-terest.
8. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: Patient reported PMR VAS at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest
9. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: Patient reported global VAS at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest.
10. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: Patient reported fatigue VAS at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest
11. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: Patient reported morning stiffness VAS at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest.
12. To investigate the effects of 100 mg/day AP1189 tablets against placebo tablets by evaluating: Patient reported duration of morning stiffness at week 4, 8, and 12 with the change from baseline to week 12 being of primary interest

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Synact Pharma ApS

Sponsor organisation

Synact Pharma ApS

Address

Dronninggaards Alle 136

City

Holte

Postcode

2840

Country

Denmark

Scientific contact point

Organisation

Synact Pharma ApS

Contact name

Thomas Jonassen

Public contact point

Organisation

Synact Pharma ApS

Contact name

Thomas Jonassen

Third parties 1

Locations

1 EU/EEA country · 5 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 10 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications