Study Evaluating Efficacy and Safety of Octanorm in Patie... | NCT03686969

NCT03686969

Protocol Section

Identification Module

Results Section

Participant Flow Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module

Status Module

Label	Type	Description	Intervention Names
Octanorm	Experimental	0.5g/kg/week octanorm 16.5%	Drug: Octanorm
Placebo	Placebo Comparator	Placebo	Other: Placebo

Name	Type	Description	Arm Group Labels	Other Names
Octanorm	Drug	Octanorm 0.5g/kg/week	Octanorm
Placebo

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Measure	Description	Time Frame
Extra-Muscular Disease Activity	Extra-muscular activity (part of MDAAT; a combined tool that captures the physician's assessment of disease activity of various organ systems using a scale from 0 = "Not present in the last 4 weeks" to 4 = "New - in the last 4 weeks [compared to the previous 4 weeks]" and a VAS).	32 weeks

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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.

Exclusion Criteria:

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 overlap myositis (except for overlap with Sjögren's syndrome), connective tissue disease associated DM, inclusion body myositis, polymyositis or drug-induced myopathy.
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).
Known IgA deficiency with antibodies to IgA.
History of hypersensitivity, anaphylaxis or severe systemic response to immunoglobulin, blood or plasma derived products or any component of octanorm 16.5% such as polysorbate 80 or to sodium chloride.
Known blood hyperviscosity, or other hypercoagulable states.
Subjects with a history of drug abuse within the past 5 years prior to study enrolment.
Participating in another interventional clinical study with investigational treatment within 3 months prior to study enrolment. Subjects who participated in the Octagam 10% Dermatomyositis Study (GAM10-08) can be included.
Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to apply an effective birth control method (as per protocol section 7.3.9 b) up to four weeks after the last IMP infusion received.

Facility	Status	City	State	ZIP	Country	Contacts
I.M. SECHENOV FIRST MOSCOW STATE MEDICAL UNIVERSITY Rheumatology Department Of, Clinici Of Nephrology		Moscow		119992	Russia

ID	Title	Description
FG000	Octanorm	0.5g/kg/week octanorm 16.5% Octanorm: Octanorm 0.5g/kg/week
FG001	Placebo	Placebo Placebo: Placebo

ID	Title	Description
BG000	Octanorm	0.5g/kg/week octanorm 16.5% Octanorm: Octanorm 0.5g/kg/week
BG001	Placebo	Placebo Placebo: Placebo

Units	Counts
Participants	BG000

Title	Description	Population Description	Parameter Type	Dispersion Type	Unit of Measure	Calculate Percentage	Denominator Units Selected	Denominators	Classes
Age, Customized			Number

Adverse Events Module

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Octanorm	0.5g/kg/week octanorm 16.5% Octanorm: Octanorm 0.5g/kg/week	0

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Jul 16, 2018	Mar 12, 2021	Prot_000.pdf
SAP	No	Yes	No	Statistical Analysis Plan	Jul 20, 2018	Mar 12, 2021	SAP_001.pdf

ID	Term
D017285	Polymyositis
D009220	Myositis
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases