A Study of Transition Regimens to KPL-387 Monotherapy in Participants with Well-Controlled Recurrent Pericarditis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Kiniksa Pharmaceuticals GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutics [E02], Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

17 Apr 2026 → ongoing

Decision date (initial)

2026-03-19

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Kiniksa Pharmaceuticals GmbH

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Pharmacodynamic, Safety, Therapy, Efficacy, Pharmacokinetic, Pharmacogenomic

Primary Objective – Posology Study:
To characterize the efficacy of the dosing regimens used to transition from prior pericarditis therapies to KPL-387 monotherapy in participants with well-controlled recurrent pericarditis (RP) on standard therapies.

Primary Objective – LTE:
To evaluate the long-term efficacy of KPL-387

Conditions and MedDRA coding

Well-Controlled Recurrent Pericarditis

Study design 2 periods

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 13

1. Each participant must meet all of the following criteria to be enrolled in this study: Is capable of understanding the written ICF, has provided signed written informed consent, and agrees to comply with protocol requirements.
2. Is > 18 years of age and < 81 years of age at Screening.
3. Has a body weight of at least 40 kg at Screening.
4. Has a history of pericarditis that in the opinion of the Investigator was consistent with the 2015 ESC Guidelines for the Diagnosis and Management of Pericardial Diseases (Adler 2015) based on the documented available data, fulfilling at least 2 of the following 4 criteria: • Pericarditic chest pain • Pericardial rub • New widespread ST segment elevation or PR segment depression according to ECG findings • Pericardial effusion (new or worsening)
5. Currently has RP which is well-controlled, i.e., including having CRP < 0.5 mg/dL within 14 days of Baseline and a pericarditis pain NRS score ≤ 3 at Baseline.
6. Has a documented history of CRP elevation (> 1 mg/dL) associated with at least one prior acute pericarditis episode, whether the incident event or any pericarditis recurrence.
7. Has received treatment for RP for at least 3 months prior to Baseline with standard therapy(ies) and is currently on a stable dosing regimen as listed in the protocol section 4.1, page 40
8. Participant is willing to and, in the opinion of the Investigator, is expected to be able to discontinue all permitted/prior pericarditis medications after initiation of study drug within protocol defined windows.
9. Routine adult vaccinations should be up to date or offered at least 2 weeks prior to first study drug administration according to regional and national guidelines based on medical history or presence of risk factors, in the opinion of the Investigator.
10. If female, must be either postmenopausal (defined as no menses for 12 months without other medical cause), permanently surgically sterile (i.e., removal of ovaries, fallopian tubes, and/or uterus), or, for women of childbearing potential, must: Be nonpregnant, nonlactating, and agree to remain abstinent or use a highly effective method of contraception with a failure rate of < 1% per year from the Screening Visit until after the end of study participation and at least 8 weeks after the last KPL-387 dose. Examples of contraception methods with a failure rate of < 1% per year include: a. Hormonal contraceptives associated with inhibition of ovulation (stable dose for at least 4 weeks prior to first dose of KPL-387); hormonal contraceptive methods must be supplemented by a barrier method b. Hormone-releasing or copper intrauterine device c. Bilateral tubal occlusion d. Vasectomized male partner e. Abstinence from heterosexual intercourse; the reliability of sexual abstinence should be evaluated based on duration of the study and usual lifestyle of the participant. Intermittent abstinence (such as calendar, ovulation, symptothermal or post-ovulation) and withdrawal are not considered acceptable contraceptive methods *The definition of childbearing potential may be adapted for alignment with local guidelines or regulations.
11. Sexually active male participants must have documented vasectomy or must agree to use a condom or method of contraception with a failure rate of <1% per year, as defined above with their partners of childbearing potential through the end of study participation and at least 8 weeks after last KPL-387 dose.
12. Male participants must agree to refrain from donating sperm through the end of study participation and at least 8 weeks after last study drug dose. Female participants must agree to refrain from donating eggs through the end of study participation and at least 8 weeks after last KPL-387 dose.
13. Agrees to refrain from lifestyle changes that may affect pericarditis symptoms (e.g., significant changes to exercise pattern) except as recommended by the Investigator from the time the ICF is signed through the end of the Posology Study.

Exclusion criteria 19

1. Participants meeting any of the following criteria will be excluded from this study: Has a diagnosis of pericarditis that is secondary to specific prohibited etiologies, including: a. Tuberculosis (TB) b. Neoplastic, purulent, or radiation etiologies c. Post-thoracic blunt trauma (e.g., motor vehicle accident) d. Systemic autoimmune diseases e. Concurrent extensive myocardial inflammation and/or injury as evidenced by: • New regional wall motion abnormalities or new global left ventricular systolic dysfunction • Cardiac Troponin level > 5 × upper limit of normal (ULN) during Screening
2. Has had a pericarditis recurrence in the last 3 months prior to Baseline.
3. Has clinical worsening of pericarditis signs/symptoms (e.g., more than mild pericardial pain) or clinical suspicion of impending pericarditis recurrence during Screening.
4. Has had prior lack of efficacy while receiving anakinra (100 mg SC once-daily dosing regimen) or rilonacept (160 mg SC once-weekly dosing regimen), or discontinuation of therapy due to safety concerns (other than local injection site reactions [ISRs])
5. Is receiving/has received concurrent or prior treatments within the washout periods prior to first dose of study drug, defined in the table on page 43 section 4.2
6. Has a positive (or 2 indeterminate) QuantiFERON® or other interferon gamma release assay (IGRA) test results unless confirmation of prior completion of appropriate treatment for latent TB and no evidence of active TB. a. IGRA test will be performed during Screening unless participant has a previously documented negative IGRA result within 8 weeks prior to Screening or documented prior positive IGRA at any time. b. An indeterminate IGRA test should be repeated. c. A positive or two successive indeterminate IGRA results should be considered a positive diagnostic TB test. d. An indeterminate followed by a negative IGRA test should be considered a negative diagnostic TB test.
7. Has a history of immunodeficiency.
8. Has a positive human immunodeficiency virus (HIV) test result. HIV test will be performed during Screening unless participant has a previously documented negative HIV result within 8 weeks prior to Screening.
9. Has chest x-ray at Screening (or history of results within 12 weeks before receiving first study drug administration), with evidence of malignancy, abnormality consistent with prior or active TB infection, active infection, or any other medical condition that could adversely affect study participation or interfere with study evaluations, in the opinion of the Investigator.
10. Has positive or intermediate results for hepatitis C virus (HCV) infection or chronic active hepatitis B infection at Screening as defined below. a. Hepatitis C antibody positive unless confirmed to have negative polymerase chain reaction (PCR) test of HCV RNA b. Hepatitis B surface antigen positive c. Hepatitis B anti-core antibody positive and anti-surface antibody negative d. Consultation with a liver disease expert is recommended prior to enrollment of any participants with hepatitis B anti-core antibody positive and anti-surface antibody positive results.
11. Has an estimated glomerular filtration rate < 30 mL/min, by laboratory standard practice (e.g., multidimensional regression discontinuity formula).
12. Has a history of malignancy of any organ system within the past 5 years before Screening (other than a successfully treated non-metastatic cutaneous squamous cell carcinoma or basal cell carcinoma and/or localized carcinoma in situ of the cervix).
13. Has a known or suspected current active infection or a history of chronic or recurrent infectious disease (> 3 episodes in prior 12 months), including, but not limited to, genitourinary infection, chest infection, sinusitis, or skin/soft tissue infection.
14. Has had a serious infection, has been admitted to the hospital for an infection, or has been treated for a documented infection requiring more than one dose of parenteral (IV or intramuscular) antibiotics within 8 weeks prior to first study drug administration or has been treated with oral (or single dose parenteral antibiotics for a documented infection within 2 weeks prior to first study drug administration).
15. Has had an organ transplant (except corneal transplant performed more than 3 months prior to first study drug administration).
16. Has most recent Screening laboratory test results indicating any of the following criteria: a. Hemoglobin level <10.0 g/dL b. WBC count <3.0 × 103/μL c. Neutrophil count <1.5 × 103/μL d. Platelet count <100 × 103/μL e. Total bilirubin level >1.5 × the ULN unless the test results are consistent with those for Gilbert’s syndrome f. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) values >2 × ULN
17. Has a known hypersensitivity to KPL-387 or to any of its excipients.
18. Has any medical condition that, in the opinion of the Investigator, could interfere with protocol compliance, evaluation of the study drug effects, or interpretation of participant safety events or confound the study results. This includes but is not limited to significant concomitant cardiac, renal, neurological, endocrinological, metabolic, pulmonary, or gastrointestinal disease, and psychiatric or substance use disorders.
19. Is not likely to be compliant with the study protocol, in the opinion of the Investigator.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Primary Efficacy Endpoint – Posology Study: Proportion of participants free from Pericarditis Recurrence by Week 16. Only Clinical Endpoint Committee (CEC) confirmed Pericarditis Recurrences will be considered as events for establishment of freedom from Pericarditis Recurrence.
2. Primary Efficacy Endpoint – LTE: Annualized rate of Pericarditis Recurrence through the end of the LTE. Only CEC-confirmed Pericarditis Recurrences will be considered as events for the analysis in the LTE.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Kiniksa Pharmaceuticals GmbH

Sponsor organisation

Kiniksa Pharmaceuticals GmbH

Address

Grafenaustrasse 5

City

Zug

Postcode

6300

Country

Switzerland

Scientific contact point

Organisation

Kiniksa Pharmaceuticals GmbH

Contact name

Medical Information Team

Public contact point

Organisation

Kiniksa Pharmaceuticals GmbH

Contact name

Medical Information Team

Third parties 11

Sponsor responsibilities

Article 77 implementation

Kiniksa Pharmaceuticals GmbH

Locations

6 EU/EEA countries · 25 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 76 files

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

Application history

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