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New pill could ease rare muscle disease

NCT ID NCT05695950

Disease control Sponsor: Lakefront Biotherapeutics NV Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study tested an oral drug called GLPG3667 (Cadefrecitinib) in 40 adults with dermatomyositis, a rare disease causing muscle weakness and skin rash. Participants took the drug or a placebo daily for 24 weeks to see if it improves symptoms. The trial is complete, and results will show if the drug is safe and effective.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
GLPG3667 (also known as Cadefrecitinib)
What this could lead to
If successful, this could point toward a new oral treatment option for dermatomyositis, potentially reducing symptoms and improving quality of life.
What could go wrong
This is a small Phase 2 trial with only 40 participants, so results may not apply to everyone. The drug may not work better than placebo, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

40 people

The number who actually took part.

Started

Feb 2023

Finished

Apr 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Participant has probable or definite DM in accordance with the ACR/EULAR criteria for at least 3 months. * Participant with DM diagnosed in the 3 years prior to screening must have undergone cancer screening (according to local standard of care or applicable guidelines) within 1 year prior to screening. Note: The evidence of cancer screening must be documented. * Participant must present objective evidence of active disease as defined by fulfilling 1 of the criteria below (as confirmed by the sponsor): * DM rash as defined by modified-Cutaneous Dermatomyositis Disease Area and Severity Index Activity Score (m-CDASI-A) ≥ 6 at screening, or * Creatine kinase (CK) \> 4x upper limit of normal (ULN) at screening, or * muscle biopsy evidence of active disease within 3 months prior to screening (defined as presence of active inflammation in muscle biopsy), or * muscle magnetic resonance imaging showing active inflammation (edema) of the proximal skeletal muscles within 3 months prior to screening, or * electromyography showing acute changes, such as spontaneous activity and myopathic changes not explained by other diseases within 3 months prior to screening, or * any other clinical evidence of active disease as confirmed by the steering committee. * Participant has reduced muscle strength (defined as Manual Muscle Test-8 \< 142/150) and at least 2 additional abnormal core set measurements out of the following 5 at screening: * Physician's Global Disease Activity score \> 2/10 cm on the visual analog scale (VAS), * Patient's Global Disease Activity score \> 2/10 cm on VAS, * extra-muscular disease activity \> 2/10 cm on VAS, * Health Assessment Questionnaire-Disability Index score \> 0.25, * elevated muscle enzymes (e.g. aldolase, CK, ALT, AST, and lactate dehydrogenase) with at least 1 muscle enzyme \> 1.5x ULN. * Participant previously demonstrated failure to or intolerance to first-line treatment (defined as oral corticosteroid\[s\] and at least 1 other immunosuppressant/ hydroxychloroquine) OR active disease despite treatment with first-line drugs. Currently, the participant is receiving maximum 3 treatments for DM (oral corticosteroid\[s\] and/or allowed immunosuppressant\[s\]/hydroxychloroquine) for at least 3 months and is on a stable dose (defined as no change in dose, type of administration, or dose regimen) for at least 4 weeks prior to screening and during screening within maximum allowed doses as specified in the study protocol. Note: Participants receiving 1 or no concomitant treatment for DM are also eligible. * Open Label Extension : Participant must meet both of the following inclusion criteria at Visit 8 to be eligible for participation in the OLE period of the study: participant who may benefit from open-label treatment with GLPG3667, according to the investigator's judgment; participant who completed the 24-week double-blind treatment period on investigational product. Key Exclusion Criteria: * Participant has cancer-associated myositis (defined as myositis diagnosed within 2 years of cancer diagnosis with the exception of basal cell carcinoma, squamous cell carcinoma of the skin, or in situ uterine cervical carcinoma that has been excised and cured). Note: At least 1 year for basal cell carcinoma and squamous cell carcinoma or 5 years for in situ uterine cervical carcinoma must have passed since the excision. * Participant has other causes of myositis (e.g. connective tissue disease) associated DM, polymyositis, juvenile DM, inclusion body myositis, or necrotizing idiopathic inflammatory myopathies (with or without rash) with the exception of overlap with secondary Sjogren's syndrome. * Participant has permanent muscle weakness due to muscle damage (e.g. participant is wheelchair bound or has significant muscle atrophy on magnetic resonance imaging \[MRI\]) or a non-DM cause (drug-induced myopathy, including glucocorticoid-induced myopathy as primary cause of muscle weakness), according to investigator's judgement. * Participant has taken any prohibited therapies within the defined washout periods before screening, and during screening as listed in the study protocol. * Open Label Extension : Participant meeting one or more of the criteria at Visit 8 as defined in the protocol, cannot be selected for the OLE period of this clinical study. 1. Participant has total bilirubin \>1.5x ULN; however, participant with an isolated increase in total bilirubin \<3 x ULN due to Gilbert's Syndrome, with normal direct bilirubin, can be enrolled in the OLE period. 2. Participant has AST or ALT \>1.5 x ULN (hepatic injury), or AST or ALT \>=5 x ULN if judged to be of muscular origin (and confirmed by the steering committee) at Visit 7 and Visit 8.

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Conditions

The condition(s) this trial relates to.

dermatomyositis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Dermatomyositis

Contacts and locations

Locations

  • Altoona Center for Clinical Research, P.C.

    Duncansville, Pennsylvania, 16635, United States

  • Augusta University

    Augusta, Georgia, 30912, United States

  • Azienda Ospedaliero Universitaria Pisana U.O. Reumatologia Universitaria

    Pisa, 56100, Italy

  • Azienda Ospedaliero Universitaria Policlinico. PO San Marco

    Catania, 95100, Italy

  • Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (Presidio Spedali Civili) Medicina Interna

    Brescia, 25123, Italy

  • BioMedica Research Group Psicomedica Clinical and Research Group

    Santiago, 7500710, Chile

  • CHU Strasbourg - Hôpital Hautepierre service de rhumatologie

    Strasbourg, 67091, France

  • CHU de Nice Hôpital Pasteur 2 Centre de Réf des Maladies Neuromusculaires et SLA

    Nice, 06001, France

  • Centro de Alta Especialidad en Reumatología e Investigación del Potosí S.C.

    San Luis Potosí City, 78213, Mexico

  • Centro de Investigacion Clínica GRAMEL S.C

    México, 03720, Mexico

  • Centro de Investigacion Medico Asistencial S.A.S

    Barranquilla, 80020, Colombia

  • Centrum Medyczne Plejady

    Krakow, 30-363, Poland

  • Charité - Campus Charité Mitte - Klinik für Dermatologie, Venerologie und Allergologie

    Berlin, 10117, Germany

  • Clinica de la Costa S.A.S

    Barranquilla, 080020, Colombia

  • Consultorio de Reumatologia Hospital Angeles Lindavista

    México, 07760, Mexico

  • Enroll SpA

    Santiago, 7500587, Chile

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 00168, Italy

  • Framingham Centro Medico

    La Plata, B1902, Argentina

  • Fundacion Respirar Consultorios Médicos Dr. Doreski

    Buenos Aires, C1426ABO, Argentina

  • Groupe Hospitalier Pitie-Salpetriere service de médecine interne et immunologie cliniqu

    Paris, 75651, France

  • Healthy Medical Center

    Zipaquirá, 250252, Colombia

  • Helios Fachklinik Vogelsang-Gommern

    Gommern, 39245, Germany

  • HonorHealth Neurology

    Scottsdale, Arizona, 85251, United States

  • Hospital Clinic de Barcelona Servicio de Medicina Interna

    Barcelona, 08036, Spain

  • Hospital Cordoba

    Córdoba, X5004CDT, Argentina

  • Hospital Italiano de La Plata

    La Plata, B1900, Argentina

  • Hospital Universitari Vall d'Hebron Internal Medicine Dept.

    Barcelona, 08035, Spain

  • Hospital Universitari de Bellvitge Servicio de Cardiologia

    L'Hospitalet de Llobregat, 08907, Spain

  • Hospital Universitario Fundacion Jimenez Diaz

    Madrid, 28040, Spain

  • Inland Rheumatology Clinical Trials, Inc.

    Upland, California, 91786, United States

  • Instituto Medico CER

    Quilmes, B1878DVB, Argentina

  • Istituto Clinico Humanitas U.O. Reumatologia

    Rozzano, 20089, Italy

  • King's College Hospital Dept of Rheumatology

    London, SE5 9RS, United Kingdom

  • Klinika Ambroziak ESTEDERM

    Warsaw, 02-953, Poland

  • Medical Care & Research SA de CV

    Mérida, 97070, Mexico

  • New Access Research and Medical Center

    Kendall, Florida, 33186, United States

  • Northwell Health, LLC PRIME

    Lake Success, New York, 11042, United States

  • Nova Reuma Domysławska i Rusiłowicz, Spółka Partnerska Lekarza Reumatologa i Fizjoterapeuty

    Bialystok, 15-707, Poland

  • Omega Research Orlando, LLC

    Orlando, Florida, 32808, United States

  • Ospedale San Giovanni Bosco

    Torino, 10154, Italy

  • Ospedale San Raffaele U.O. di Medicina Gen. Ind. Immunologico-Clinica

    Milan, 20132, Italy

  • Polyclinic Bonifarm

    Zagreb, 10000, Croatia

  • Revmatologicky Ustav

    Prague, 12850, Czechia

  • Salford Care Organisation Dept of Rheumatology

    Salford, M6 8HD, United Kingdom

  • Sc Medaudio-Optica SRL

    Râmnicu Vâlcea, 240762, Romania

  • Solmed Polyclinic

    Zagreb, 10000, Croatia

  • Spitalul Clinic 'Sf. Maria' Clinica de Medicina Interna si Reumatologie

    Bucharest, 11172, Romania

  • Spitalul Clinic Colentina parent

    Bucharest, 020125, Romania

  • St. Paul Rheumatology

    Eagan, Minnesota, 55121, United States

  • St. Peter´s Hospital Dept of Rheumatology

    Chertsey, KT16 OPZ, United Kingdom

  • UZ Leuven

    Leuven, 3000, Belgium

  • Universitätsklinikum Tübingen - Universitäts-Hautklinik

    Tübingen, 72076, Germany

  • Western General Hospital Dept of Rheumatology

    Edinburgh, EH4 2XU, United Kingdom

  • Zespol Poradni Specjalistycznych Reumed

    Lublin, 20-582, Poland

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