Dermatomyositis MedDRA version: 20.0 Level: PT Classification code 10012503 Term: Dermatomyositis System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
Conditions
Interventions
Product Name: Begelomab
Product Code: [Not applicable]
Pharmaceutical Form: Concentrate for solution for injection/infusion
CAS Number: 1403744-56-8
Current Sponsor code: Not applicable
Concentration
Sponsors
ADIENNE SA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Written informed consent before starting any study-related procedure; -Males and Females aged > or = 18 and or = 5; -Stable glucocorticoid treatment for DM at a stable dose 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
Exclusion criteria
Exclusion criteria: - 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 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of adding begelomab to glucocorticoid and/or immunosuppressant therapy (methotrexate, azathioprine, mycophenolate, cyclosporine) compared with glucocorticoid and/or immunosuppressant plus placebo in the treatment of patients with dermatomyositis (DM).;Secondary Objective: To assess the improvement of skin manifestations in begelomab arm compared to placebo; To assess the safety and tolerability of multiple doses of begelomab in patients with dermatomyositis compared to placebo; To assess if the treatment with begelomab is able to improve the quality of life in patients with dermatomyositis compared to placebo; To assess if the treatment with begelomab is able to reduce or maintain a stable glucocorticoid/immunosuppressant therapy;Primary end point(s): Mean difference (begelomab vs. placebo) in International Myositis Assessment and Clinical Studies (IMACS) total improvement score at Day 31 of each treatment period.;Timepoint(s) of evaluation of this end point: Day 31 of each treatment period | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Number of subjects who achieve IMACS Definition of Improvement (IMACS DOI) at Day 31 in each treatment period and at M2/M6 in the begelomab arm compared to placebo. The IMACS DOI is: - An improvement of >= 20% from baseline in 3 IMACS core set measures AND - No more than 2 IMACS core set measure scores worsening by >= 25% from baseline, AND - Manual Muscle Test (MMT-8) may not decrease by >= 25% from baseline. • The change from baseline of CDASI Activity Score (Cutaneous Dermatomyositis Disease Area and Severity Index) score at Day 31 in each treatment period and at M2/M6 in the begelomab arm compared to placebo. • Mean difference in International Myositis Assessment and Clinical Studies (IMACS) total improvement score at M2/M6 in the begelomab arm compared to placebo. • The change from baseline of HAQ (Health Assessment Questionnaire) or PGA (Patient Global Activity) score at Day 31 in each treatment period and at M2/M6 in the begelomab arm compared to placebo. • Number of subjects with a change (increase or taper) in glucocorticoid/ immunosuppressant therapy (begelomab vs placebo). • Proportion of participants who have a treatment-related AE (TRAE) at Day 15 and Day 31 in each treatment period.;Timepoint(s) of evaluation of this end point: Day 31 in each treatment period and at M2/M6 | — |
Countries
Italy
Contacts
Public ContactRenata Palmieri Clinical Developmen
ADIENNE SA
Outcome results
None listed