Phase II placebo controlled study, to evaluate the efficacy and safety of begelomab in combination with standard immunosuppressive therapy and/or corticosteroids in the treatment of patients with dermatomyositis
- Conditions
- DermatomyositisMedDRA version: 20.0Level: PTClassification code 10012503Term: DermatomyositisSystem Organ Class: 10040785 - Skin and subcutaneous tissue disordersTherapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2021-000898-83-IT
- Lead Sponsor
- ADIENNE SA
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 20
-Written informed consent before starting any study-related procedure;
-Males and Females aged > or = 18 and < or = 80 years old;
-Diagnosis of probable or definite DM according to Bohan and Peter criteria (Bohan and Peter, 1975) or ACR/EULAR criteria (Lundberg et al, 2017);
-Although not mandatory, patients with muscle weakness are eligible for enrollment. Those with active muscle weakness must have a Manual Muscle Testing (MMT-8) score < 142 out of 150;
-Active skin disease as defined by a CDASI score > or = 5;
-Stable glucocorticoid treatment for DM at a stable dose < or = 0.2 mg/kg/day methylprednisolone or prednisolone or equivalent for > or = 4 weeks before randomization;
-Stable immunosuppressant treatment for DM for > or = 4 weeks before randomization, the stable treatment is defined as follows:
o Patients currently treated with oral or subcutaneous methotrexate (MTX) must have been on a stable dose < or = 25 mg per week.
o Patients currently treated with oral azathioprine (AZA) must have been on a stable dose < or = 3 mg/kg/day.
o Patients currently treated with oral mycophenolate (MMF) must have been on a stable dose < or = 3 g/day.
o Patients currently treated with oral, subcutaneous or intravenous Cyclosporine (CsA) must have been on a stable dose of < or = 5 mg/kg/day.
-Patients that have received the following treatments can be enrolled only if the medications have been discontinued prior to screening visit:
o Rituximab: 9 months (Note: patients who received rituximab are only eligible for inclusion if B-cell counts are confirmed to be within normal limits)
o Intravenous immunoglobulin (IVIG): 3 months
-Female subjects must meet one of the following criteria:
o Surgical sterilization (complete hysterectomy, bilateral tubal ligation and/or bilateral ovariectomy or tubal occlusion at least 6 months prior to first dosing), or have documented congenital sterility.
o Postmenopausal: defined as at least 12 months with no menses prior to screening and a serum follicle-stimulating hormone (FSH) to confirm postmenopausal status at screening.
o Women of child-bearing potential must agree to take adequate contraceptive measures in order to avoid any pregnancies during the course of the study (or for at least 3 months following the last dose of study drug, whichever is longer). Acceptable methods of birth control include oral, injected or implanted hormonal methods of contraception, placement of an intrauterine device (IUD) or intrauterine system (IUS), barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/ film/ cream/suppository. Abstinence is only considered an acceptable form of contraception when
it is the usual life style of an individual.
-Male patient who is surgically sterile (vasectomy), or male patient who is willing to agree with the true abstinence (refrain from heterosexual intercourse) or who uses barrier contraceptive measures during the entire study treatment period and for 3 months after the last administration of study drug. It is also acceptable where patient partner is making use of oral contraceptive instead.
-Women must have a negative pregnancy test at Screening and at Baseline and must not be breastfeeding.
-Subject must be willing and able to comply with study requirements, remain at the clinic, and return to the clinic for the follow-up evaluation, as specified in this protocol during the study period.
Are the trial subjects under 18? no
Num
- Any systemic corticosteroid therapy for indication other than DM in the last 4 weeks before the enrolment; the application of topical steroids is allowed.
- Patients with other types of myositis or myopathies: polymyositis, inclusion body myositis, necrotizing myopathy, metabolic or drug induced myopathy or myositis in overlap with other rheumatic diseases such as lupus, scleroderma, Sjogren's, or vasculitis.
-Any of the following laboratory values at Screening:
o Haemoglobin < 9 g/dL in males and < 8 g/dL in females
o White blood cell count < 3.0 × 10^9/L
o Absolute neutrophil count (ANC) < 1.2 × 10^9 /L (1200/mm^3)
o Platelet count < 75 × 10^9 /L
o Serum AST or serum ALT > 2.5 × ULN (unless considered consistent with muscle origin)
-Major surgery within 12 weeks before Screening or planned during the study period.
-Any active or suspected malignancy, including myeloproliferative or lymphoproliferative disorder, or history of documented malignancy within the last 5 years before screening or within 3 years of diagnosis of myositis, except appropriately excised and cured cervical carcinoma in situ or basal or squamous cell carcinoma of the skin.
- Any underlying or current medical or psychiatric condition that, in the opinion of the investigator, would interfere with the evaluation of the subject including, but not limited to symptomatic congestive heart failure (New York Heart Association [NYHA] Class III to IV), unstable angina pectoris or cardiac arrhythmia. Any other serious medical condition, as judged by the investigator, which places the subject at an unacceptable risk if he or she were to participate in the study or confounds the ability to interpret data from the study.
-Patients with uncontrolled bacterial, viral or fungal infections.
-Administration of any other investigational agents (not approved by the United States Food and Drug Administration Agency [FDA] or European Medicines Agency [EMA] for any indication) within 4 weeks prior to enrolment.
-Clinically significant history of any drug sensitivity, drug allergy, or food allergy, as determined by the Investigator.
-History of chronic drug or alcohol abuse within the last 5 years
-Patients unable to understand the procedures and purposes of the study or unwilling to accept and meet study requirements.
-Unwillingness to use effective contraceptive measures up to 3 months after the end of study drug administration (females and males). Acceptable methods of birth control include oral, injected or implanted hormonal methods of contraception, placement of an intrauterine device IUD) or intrauterine system (IUS), barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository. Abstinence is only considered an acceptable form of contraception when it is the usual life style of an individual.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method