Study Evaluating Efficacy and Safety of Octanorm in Patients With Dermatomyositis

NCT03686969 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: SCGAM-02
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED PHASE III STUDY EVALUATING EFFICACY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH DERMATOMYOSITIS

Conditions

Dermatomyositis

Outcome Measures

Primary Outcomes (3)

• MMT-8

  MMT-8; a set of 8 designated muscles tested bilaterally \[potential score 0 - 150\]

  32 weeks

• CDASI

  The CDASI is a clinician-scored single page instrument that separately measures activity and damage in the skin of DM patients for use in clinical practice or clinical/therapeutic studies.

  32 weeks

• Physician's Global Disease Activity VAS Worsening

  Physician's Global Disease Activity (10 cm VAS assessing global disease activity from "No evidence of disease activity" to "Extremely active or severe disease activity"; Disease Activity being defined as potentially reversible pathology or physiology resulting from the myositis).

  32 weeks

Secondary Outcomes (56)

• Extra-Muscular Disease Activity

  32 weeks

• Muscle Enzymes - Aldolase

  32 weeks

• Muscle Enzymes - Creatine Kinase

  32 weeks

• Muscle Enzymes - Alanine Aminotransferase

  32 weeks

• Muscle Enzymes - Aspartate Aminotransferase

  32 weeks

Study Arms (2)

Octanorm

EXPERIMENTAL

0.5g/kg/week octanorm 16.5%

Drug: Octanorm

Placebo

PLACEBO COMPARATOR

Placebo

Other: Placebo

Interventions

Octanorm DRUG

Octanorm 0.5g/kg/week

Octanorm

Placebo OTHER

Placebo

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects with diagnosis of definite or probable DM according to the Bohan and Peter criteria.
• Subjects who have responded to IGIV treatment as assessed by the treating physician and being on a stable dose for at least 3 months on 2 g/kg bodyweight (+/- 10%).
• For subjects being on other medication(s) for the treatment of DM (immunosuppressants, corticosteroids): a) subject was on such medication(s) at the start of IGIV treatment in the first place, and b) received such medication(s) for at least 3 months prior to study enrolment and at a stable dose for at least 4 weeks prior to study enrolment at the maximally allowed conditions as per Table 2 (see section 4.2.1).
• MMT-8 score ≥144, with at least 3 other CSM to be normal or near normal as per the following criteria: Visual Analogue Scale \[VAS\] of patient global disease activity ≤2 cm, physician's global disease activity ≤2 cm, extra-muscular disease activity ≤2 cm; no muscle enzyme \>4 times upper limit of normal due to myositis, Health Assessment Questionnaire \[HAQ\] ≤0.25.
• Males or females ≥ 18 to \<80 years of age.
• Voluntarily given, fully informed written consent obtained from subject before any study-related procedures are conducted.
• Subject must be capable and willing to understand and comply with the relevant aspects of the study protocol.

You may not qualify if:

• Cancer-associated myositis, defined as the diagnosis of myositis within 2 years of the diagnosis of cancer (except basal or squamous cell skin cancer or carcinoma in situ of the cervix that has been excised and cured and at least 1 or 5 years, respectively, have passed since excision).
• Evidence of active malignant disease or malignancies diagnosed within the previous 5 years (including hematological malignancies and solid tumors) or breast cancer diagnosed within the previous 10 years. Subjects \>5 years (\>10 years for breast cancer) of cancer diagnosis who have been treated and are in remission are allowed.
• Subjects with immune-mediated necrotizing myopathy with absence of typical DM rash.
• Subjects with generalized, severe musculoskeletal conditions other than DM that prevent a sufficient assessment of the subject by the physician.
• Subjects who received blood or plasma-derived products (other than IGIV) or plasma exchange within the last 3 months before enrolment.
• Subjects with administration of permitted concomitant medications exceeding the maximally allowed conditions as per section 4.2.1.
• Subjects with administration of forbidden concomitant medications within the washout periods as defined in Table 3: see section 4.2.2.
• Subjects starting or planning to start a physical therapy-directed exercise regimen during the trial. Subjects on stable physical therapy for \>4 weeks are allowed but the regimen should remain the same throughout the trial.
• Cardiac insufficiency (New York Heart Association III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease.
• Severe liver disease, with signs of ascites and hepatic encephalopathy.
• Severe kidney disease (as defined by estimated glomerular filtration rate (eGFR) \< 30 mL/min/1.73 m2).
• Known hepatitis B, hepatitis C or HIV infection.
• Subjects with a history of deep vein thrombosis within the last year prior to study enrolment or pulmonary embolism ever.
• Body mass index \>40 kg/m2 and/or body weight \>120 kg.
• Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).

Sponsors & Collaborators

• Octapharma: lead

Study Sites (1)

I.M. SECHENOV FIRST MOSCOW STATE MEDICAL UNIVERSITY Rheumatology Department Of, Clinici Of Nephrology

Moscow, 119992, Russia

Location

MeSH Terms

Conditions

Dermatomyositis

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Results Point of Contact

• Title: Mikaela Raymond
• Organization: CRMG

ctgov@cllinicalresearchmgt.com

866-337-1868

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 31, 2018
• First Posted: September 27, 2018
• Study Start: August 2, 2018
• Primary Completion: November 22, 2018
• Study Completion: November 29, 2018
• Last Updated: April 23, 2021
• Results First Posted: April 23, 2021

Record last verified: 2021-04

Locations

RU (1)