This study aims to investigate the efficacy and safety of efgartigimod PH20 SC in adult participants with active idiopathic inflammatory myopathy.
Zusammenfassung der Studie
This study is being conducted to verify whether the experimental drug "Efgartigimod" is safe and how effective it is in the disease known as idiopathic inflammatory myopathy, commonly referred to as myositis. The drug under investigation is "experimental". This means it has not been approved for use in treating myositis. In this study, efgartigimod will be combined with "rHuPH20". This is a protein used to enhance the distribution and absorption of drugs that are injected into the body. This study includes 2 phases (phase 2 and phase 3). In total, approximately 240 participants will take part in the study, of which about 2 will be recruited in Switzerland. The study will take place at various study centers in different countries around the world. Efgartigimod will be compared with an inert drug (placebo). The patient will be randomly assigned (like a coin toss) to the efgartigimod group or the placebo group. The placebo looks like efgartigimod but does not contain efgartigimod. However, the placebo contains rHuPH20. The purpose of this is to see the effect of efgartigimod. Researchers compare the results of efgartigimod with those of the placebo to verify whether efgartigimod works and is safe. The patient will not know whether they will receive efgartigimod or placebo, nor will the study doctor. The drug under study will be administered weekly during the treatment period. Between visits, the patient will receive the study drug at the study center or at home, depending on their preference. The patient will continue to take their baseline medication for myositis. This is the medication that the patient has already been taking for at least 8 weeks.
(BASEC)
Untersuchte Intervention
This study consists of a phase 2 and a phase 3. Each phase involves the enrollment of separate groups of participants. Participants will be randomly assigned (like a coin toss) to the efgartigimod group or the placebo group after a screening period of ≤4 weeks. In the meantime, they will continue to take their concomitant baseline treatment for IIM. In both phases, participants will receive weekly injections of 1000 mg of experimental medication (IMP) for 24 weeks (phase 2) or 52 weeks (phase 3). After the treatment period, participants will be enrolled in the open-label extension study ARGX-113-2011 or will complete a 56-day safety follow-up period.
(BASEC)
Untersuchte Krankheit(en)
Participants must have received a certain or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM), also known as myositis. Participants must also have one of the following medical histories: diagnosis of dermatomyositis or juvenile dermatomyositis diagnosed no more than 5 years prior to the screening date; diagnosis of polymyositis (including anti-synthetase antibody syndrome) or diagnosis of immune-mediated necrotizing myopathy.
(BASEC)
- Age over 18 years with clinical IIM - Muscle weakness and baseline pharmacological treatment* - Willingness to adhere to contraception guidelines - Provide informed consent (BASEC)
Ausschlusskriterien
- Active bacterial, viral, or fungal infection at the time of screening, including COVID-19 - Other known autoimmune disease or history of malignancy*. - Muscle damage, other myopathies, or uncontrolled manifestations of IIM, clinically significant disease, recent surgical intervention - Allergy to the experimental drug - No clinical response to plasmapheresis/plasma exchange - Received live or live-attenuated vaccine less than four weeks prior to screening - Received prior therapies with prohibited medications* - Participation in a clinical study on efgartigimod or concurrent participation in another clinical study - IgG < 4 g/l at screening - Alcohol, drug, or medication abuse - Pregnancy or breastfeeding or, if male, planning to procreate - Being hospitalized in an institution or in a dependent relationship with the sponsor or investigator. * As defined in the protocol (BASEC)
Studienstandort
Lugano
(BASEC)
Sponsor
ICON Clinical Research (Switzerland) GmbH
(BASEC)
Kontakt für weitere Auskünfte zur Studie
Kontaktperson Schweiz
Sabine Coppieters, MD
+1 857-350-4834
ClinicalTrials@clutterargenx.comargenx BV
(BASEC)
Name der bewilligenden Ethikkommission (bei multizentrischen Studien nur die Leitkommission)
Ethikkommission Tessin
(BASEC)
Datum der Bewilligung durch die Ethikkommission
04.04.2023
(BASEC)
ICTRP Studien-ID
NCT05523167 (ICTRP)
Offizieller Titel (Genehmigt von der Ethikkommission)
A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy (BASEC)
Wissenschaftlicher Titel
A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy (ICTRP)
Öffentlicher Titel
A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ICTRP)
Untersuchte Krankheit(en)
Active Idiopathic Inflammatory MyopathyMyositisDermatomyositisPolymyositisImmune-Mediated Necrotizing MyopathyAntisynthetase Syndrome (ICTRP)
Untersuchte Intervention
Biological: EFG PH20 SCOther: PBO (ICTRP)
Studientyp
Interventional (ICTRP)
Studiendesign
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator). (ICTRP)
Ein-/Ausschlusskriterien
Inclusion Criteria:
- Ability to consent in the jurisdiction in which the study is taking place and
capable of giving signed informed consent.
- A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM)
- One of the following medical histories: Diagnosis of dermatomyositis (DM) or
juvenile dermatomyositis (JDM), Diagnosis of polymyositis (PM) (including
antisynthetase syndrome (ASyS)), Diagnosis of immune-mediated necrotizing myopathy
(IMNM)
- Diagnosed with active disease as defined by the presence of at least 1 of the
following criteria: Abnormal levels of at least 1 of the following enzymes: creatine
kinase (CK), aldolase, lactate dehydrogenase, aspartate aminotransaminase (AST),
alanine aminotransferase (ALT), based on central laboratory results
Electromyography demonstrating active disease within the past 3 months Active
dermatomyositis (DM) skin rash Muscle biopsy indicative of active idiopathic
inflammatory myopathy (IIM) in the past 3 months Magnetic resonance imaging within
the past 3 months indicative of active inflammation
- Muscle weakness
- Receiving a permitted background treatment for idiopathic inflammatory myopathy.
- Contraceptive use consistent with local regulations, where available, for
individuals participating in clinical studies. Women of childbearing potential must
have a negative serum pregnancy test during screening and a negative urine pregnancy
test at baseline before receiving investigational medicinal product (IMP).
The full list of inclusion criteria can be found in the protocol.
Exclusion Criteria:
- A clinically significant active infection at screening
- A COVID-19 polymerase chain reaction (PCR)-positive test before enrollment
- Any other known autoimmune disease that, in the investigator's opinion, would
interfere with an accurate assessment of clinical symptoms of idiopathic
inflammatory myopathy (IIM) or put the patient at undue risk
- A history of malignancy unless considered cured by adequate treatment, with no
evidence of recurrence for = 3 years before the first administration of the
investigational medicinal product (IMP). Adequately treated participants with the
following cancers can be included at any time: Basal cell or squamous cell skin
cancer Carcinoma in situ of the cervix Carcinoma in situ of the breast
Incidental histological finding of prostate cancer
- Severe muscle damage
- Glucocorticoid-induced myopathy that the investigator considers the primary cause of
muscle weakness or permanent weakness linked to a non-idiopathic inflammatory
myopathy (IIM) cause
- Juvenile myositis (JDM) diagnosed > 5 years from screening or juvenile myositis with
extensive calcinosis or severe calcinosis.
- Uncontrolled interstitial lung disease or any other uncontrolled idiopathic
inflammatory myopathy (IIM) manifestation that, in the opinion of the investigator,
would be likely to require treatment with prohibited medication during the study
- Other inflammatory and noninflammatory myopathies: inclusion body myositis, overlap
myositis), metabolic myopathies, muscle dystrophies or a family history of muscle
dystrophy, drug-induced or endocrine induced myositis, and juvenile myositis (other
than juvenile dermatomyositis (JDM))
- Clinically significant disease, recent major surgery or intends to have surgery
during the study, or has any other condition in the opinion of the investigator that
could confound the results of the trial or put the patient at undue risk
- Known hypersensitivity reaction to investigational medicinal product (IMP) or 1 of
its excipients
- Received a live or live-attenuated vaccine less than 4 weeks before screening.
- Positive serum test at screening for active viral infection with any of the
following conditions: Hepatitis B virus (HBV) Hepatitis C virus (HCV) HIV
- Participant has previously participated in an efgartigimod clinical trial and
received at least 1 dose of investigational medicinal product (IMP).
- Participant is concurrently participating in any other clinical study, including a
noninterventional study.
- Participant has a current or history (ie, within 12 months of screening) of alcohol,
drug, or medication abuse.
- Participant is pregnant or lactating or intends to become pregnant during the study.
- Participant has severe renal impairment .
- Participant is institutionalized by a court or other governmental order or is in a
dependent relationship with the sponsor or investigator.
The full list of exclusion criteria can be found in the protocol. (ICTRP)
nicht verfügbar
Primäre und sekundäre Endpunkte
Total improvement score (TIS); measured on a [0,100] scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline). (ICTRP)
Time to reach TIS = 20 (first "minimal clinical improvement");Percentage of participants with TIS = 20;Time to reach TIS = 40 (first "moderate clinical improvement");Percentage of participants with TIS = 40;Change in manual muscle testing-8 (MMT8) score;Change in Patient Global Assessment of Disease Activity (PGA);Change in Physician Global Assessment of Disease Activity (MDGA);Proportion of participants achieving target dose of = 5 mg (prednisone equivalent) (ICTRP)
Registrierungsdatum
nicht verfügbar
Einschluss des ersten Teilnehmers
nicht verfügbar
Sekundäre Sponsoren
nicht verfügbar
Weitere Kontakte
Sabine Coppieters, MD, ClinicalTrials@argenx.com, 857-350-4834 (ICTRP)
Sekundäre IDs
2024-512785-33-00, ARGX-113-2007 (ICTRP)
Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
nicht verfügbar
Weitere Informationen zur Studie
https://clinicaltrials.gov/study/NCT05523167 (ICTRP)
Ergebnisse der Studie
Zusammenfassung der Ergebnisse
nicht verfügbar
Link zu den Ergebnissen im Primärregister
nicht verfügbar