Study aimed at evaluating the long-term safety and efficacy of a subcutaneous formulation of efgartigimod in adults with active idiopathic inflammatory myopathy
Summary description of the study
This is an open-label extension study (OLE) conducted following the ARGX-113-2007 study and provides participants from the ARGX-113-2007 study the opportunity to receive efgartigimod PH20 subcutaneously. The purpose of this study is to further analyze the efficacy, safety, and tolerability of the investigational drug (efgartigimod PH20 subcutaneously, being developed by Argenx BV) in people affected by myositis. It is hoped that the investigational drug may help improve the symptoms of myositis.
(BASEC)
Intervention under investigation
This is a phase 3 study. Each participant will receive the investigational drug for approximately 52 weeks, starting from the first visit. The treatment period will be followed by a 56-day safety follow-up period without treatment. Participants will enter this study with the same background medication for myositis that they received in ARGX-113-2007. Modification of the background medication for myositis will be allowed at the discretion of the investigator and based on the clinical conditions of the participant and the activity of the idiopathic myopathy.
(BASEC)
Disease under investigation
Participants must have received a definite clinical diagnosis of idiopathic inflammatory myopathy (IIM), also known as myositis, and must have completed participation in the ARGX-113-2007 clinical study.
(BASEC)
Inclusion criteria for participant selection, including age and sex. Other selection criteria may relate to clinical diagnosis and comorbidity conditions. - Completion of the ARGX-113-2007 study - Ability to provide signed informed consent and comply with protocol requirements. - Agreement to use contraceptive measures in accordance with local regulations, and women of childbearing potential must have a negative urine pregnancy test at baseline before receiving the investigational drug. (BASEC)
Exclusion criteria
Intention to undergo major surgery during the study period of ARGX-113-2011; or any other medical condition arising after enrollment in ARGX-113-2007, which, in the judgment of the investigator, could confound the study results or put the participant at excessive risk or known hypersensitivity to the investigational drug or any of its excipients. Development of any neoplasia, new or recurrent, other than basal cell carcinoma of the skin, regardless of relatedness or diagnosis of an unselected subtype of IIM based on the analysis of phase 2 data from ARGX-113-2007, unless the investigator determines that the participant is benefiting from the investigational drug, as defined by a score of "much better" or "moderately better" on the CGI-C and PGI-C assessments for at least 12 weeks, and that enrollment in the study is in the participant's best interest. Permanent discontinuation of the investigational drug in ARGX-113-2007, or meeting the criteria for permanent discontinuation at the rollover visit. (BASEC)
Trial sites
Lugano
(BASEC)
Sponsor
ICON Clinical Research (Switzerland) GmbH
(BASEC)
Contact
Contact Person Switzerland
Sabine Coppieters, MD
+1 857-350-4834
ClinicalTrials@clutterargenx.comargenx BV
(BASEC)
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee Ticino
(BASEC)
Date of authorisation
24.11.2023
(BASEC)
ICTRP Trial ID
NCT05979441 (ICTRP)
Official title (approved by ethics committee)
Estensione dello Studio ARGX-113-2007 di fase 3, a braccio singolo, multicentrico, in aperto, finalizzato a esaminare la sicurezza a lungo termine, la tollerabilità e l’efficacia di Efgartigimod PH20 SC, in partecipanti con un’età minima di 18 anni, affetti da miopatia infiammatoria idiopatica attiva (BASEC)
Academic title
A Phase 3, Single-Arm, Multicenter, Open-label Extension of Study ARGX-113-2007 to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy (ICTRP)
Public title
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy (ICTRP)
Disease under investigation
Myositis
(ICTRP)
Intervention under investigation
Biological: EFG PH20 SC
(ICTRP)
Type of trial
Interventional (ICTRP)
Trial design
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label). (ICTRP)
Inclusion/Exclusion criteria
Inclusion Criteria:
- Has completed trial ARGX-113-2007
- Being capable of providing signed informed consent and complying with protocol
requirements
- Agrees to use contraceptive measures consistent with local regulations and women of
childbearing potential must have a negative urine pregnancy test at baseline before
receiving the investigational medicinal product
Exclusion Criteria:
- Intention to have major surgery during the study period; or any other medical
condition that has arisen since enrollment in study ARGX-113-2007, that in the
investigator's opinion, would confound the results of the study or put the participant
at undue risk
- Known hypersensitivity reaction to investigational medicinal product or 1 of its
excipients Development of any malignancy, either new or recurrent, other than basal
cell carcinoma of the skin, regardless of relatedness
- Permanent discontinuation of IMP in ARGX-113-2007, or met the permanent
discontinuation criteria at the rollover visit
- Diagnosis with a deselected subtype of myositis based on the analysis of the phase 2
stage data in ARGX-113-2007, unless the investigator determines that the participant
is benefiting from IMP as defined by a score of "much better" or "moderately better"
on the 'Clinical Global Impression of Change' and 'Patient Global
- Impression of Change' assessments for at least 12 weeks, and that enrolling in the
study is in the participant's best interest
(ICTRP)
not available
Primary and secondary end points
Incidence treatment-emergent adverse events and adverse event of special interest
(ICTRP)
Percentage of participants with remission, defined as a clinically inactive disease for at least 24 weeks
Composite Glucocorticoid Toxicity Index (C-GTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening Score (CWS)
Individual core set measures (CSMs) of the TIS
Proportion of participants who discontinue corticosteroids
Percentage of participants with clinically inactive disease
Total improvement score (TIS)
Prednisone dose reduction (average monthly dose)
Proportion of TIS responders (minimal, moderate, major)
(ICTRP)
Registration date
28.07.2023 (ICTRP)
Incorporation of the first participant
12.09.2023 (ICTRP)
Secondary sponsors
not available
Additional contacts
Sabine Coppieters, MD, Clinicaltrials@argenx.com, 857-350-4834 (ICTRP)
Secondary trial IDs
ARGX-113-2011 (ICTRP)
Results-Individual Participant Data (IPD)
not available
Further information on the trial
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT05979441 (ICTRP)
Results of the trial
Results summary
not available
Link to the results in the primary register
not available