Clinical Trials Register

Summary
EudraCT Number:	2017-002710-31
Sponsor's Protocol Code Number:	SCGAM-02
National Competent Authority:	Czechia - SUKL
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-06-01
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Czechia - SUKL

A.2

EudraCT number

2017-002710-31

A.3

Full title of the trial

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

Dvojitě zaslepená randomizovaná placebem kontrolovaná studie fáze III hodnotící účinnost a bezpečnost subkutánního lidského imunoglobulinu (octanorm) u pacientů s dermatomyozitidou.

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

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

A.4.1

Sponsor's protocol code number

SCGAM-02

A.5.4

Other Identifiers

Name:

IND

Number:

17515

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Octapharma Pharmazeutika Produktionsges.m.b.H
B.1.3.4	Country	Austria
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Octapharma Pharmazeutika Produktionsges.m.b.H.
B.4.2	Country	Austria
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Octapharma Pharmazeutika Produktionsges.m.b.H.
B.5.2	Functional name of contact point	Global Clinical Project Manager
B.5.3	Address:
B.5.3.1	Street Address	Oberlaaer Str. 235
B.5.3.2	Town/ city	Vienna
B.5.3.3	Post code	1100
B.5.3.4	Country	Austria
B.5.4	Telephone number	431610321202
B.5.5	Fax number	431610329249
B.5.6	E-mail	clinical.department@octapharma.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Octanorm 16.5%
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Human Normal Immunoglobulin
D.3.9.1	CAS number	308067-58-5
D.3.9.2	Current sponsor code	Octanorm 16.5%
D.3.9.3	Other descriptive name	HUMAN NORMAL IMMUNOGLOBULIN
D.3.9.4	EV Substance Code	SUB14196MIG
D.3.10	Strength
D.3.10.1	Concentration unit	% percent
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	16.5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	Yes
D.3.11.7	Plasma derived medicinal product	Yes
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Dermatomyositis

E.1.1.1

Medical condition in easily understood language

Subject with Dermatomyositis

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10012503
E.1.2	Term	Dermatomyositis
E.1.2	System Organ Class	10040785 - Skin and subcutaneous tissue disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of this study is to determine the efficacy of subcutaneous immunoglobulin octanorm in the maintenance treatment of DM patients who have previously responded to IGIV therapy.

E.2.2

Secondary objectives of the trial

The secondary objectives of this study are:
•to assess other efficacy outcomes at the end of study (Week 32 or Drop-out Visit;
•to assess the effect of octanorm on Quality of Life measures;
•to assess the treatment compliance of home treatment with octanorm;
to evaluate the safety and tolerability of octanorm in subjects with Dermatomyositis

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Subjects with diagnosis of definite or probable DM according to the Bohan and Peter criteria.
2.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%) prior to study enrolment.
3.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).
4.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.
5.Males or females 18 to <80 years of age.
6.Voluntarily given, fully informed written consent obtained from subject before any study-related procedures are conducted.
7.Subject must be capable and willing to understand and comply with the relevant aspects of the study protocol

E.4

Principal exclusion criteria

1-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 at least 1 year for basal or squamous cell skin cancer and 5 years for carcinoma in situ of the cervix must have passed since excision).
2-Evidence of active malignant disease or malignancies diagnosed within the previous 5 years (including hematological malignancies and solid tumors).
3-Subjects with overlap myositis connective tissue disease associated DM, inclusion body myositis, polymyositis, juvenile dermatomyositis or drug-induced myopathy.
4-Subjects with immune-mediated necrotizing myopathy with absence of typical DM rash.
5-Subjects with generalized, severe musculoskeletal conditions other than DM that prevent a sufficient assessment of the subject by the physician.
6-Subjects who received blood or plasma-derived products (other than IGIV) or plasma exchange within the last 3 months before enrolment.
7-Subjects with administration of permitted concomitant medications exceeding the maximally allowed conditions as per section 4.2.1
8-Subjects with administration of forbidden concomitant medications within the washout periods as defined in Table 3
9-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.
10-Cardiac insufficiency (New York Heart Association III/IV).
11-Severe liver disease, with signs of ascites and hepatic encephalopathy.
12-Severe kidney disease (as defined by estimated glomerular filtration rate (eGFR)
< 30 mL/min/1.73 m2).
13-Known active or chronic hepatitis B, hepatitis C or HIV infection. Past hepatitis B or C infection that has been cured is allowed.
14-Subjects with a history of deep vein thrombosis within the last year prior to study enrolment or pulmonary embolism.
15-Body mass index >40 kg/m2 and/or body weight >120 kg.
16-Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization.
17-Known IgA deficiency with antibodies to IgA.
18-History of hypersensitivity, anaphylaxis or severe systemic response to immuno- globulin, blood or plasma derived products or any component of octanorm 16.5% such as polysorbate 80 or to sodium chloride.
19-Known blood hyperviscosity, or other hypercoagulable states.
20-Subjects with a history of drug abuse within the past 5 years prior to study enrolment.
21-Participating in another interventional clinical study with investigational treatment within 3 months prior to study enrolment except study GAM10-08.
22-Women who are breast feeding, pregnant, or planning to become pregnant during study.

E.5 End points

E.5.1

Primary end point(s)

Proportion of patients with clinically important deterioration during the treatment period up to Week 32. A patient with clinically important deterioration is defined as a patient with 1) MMT-8 worsening ≥ 6 points (scale of 150) OR CDASI (Total Activity Score)worsening ≥ 5 points, AND 2) a Physician’s Global Disease Activity VAS worsening 2 cm.

E.5.1.1

Timepoint(s) of evaluation of this end point

End of the study

E.5.2

Secondary end point(s)

•Mean change from baseline (Week 0-defined as end of IGIV therapy) to end of the treatment period (Week 32 or Drop-Out Visit) in:
oModified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI);
oSix Myositis CSM: MMT-8, Physician’s Global Disease Activity, Patient’s Global Disease Activity, Extra-Muscular Disease Activity, Muscle enzymes, Health Assessment Questionnaire (HAQ);
oSF-36v2 Health Survey.
•Mean change in TIS from Baseline (Week 0) to end of treatment period (Week 32 or Drop-Out Visit).
•Time to clinically important deterioration during the treatment period.
•Number and type of deviations from protocol requirements relating to home treatment (dosing, timing).

E.5.2.1

Timepoint(s) of evaluation of this end point

End of the study

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Canada

Russian Federation

United States

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

After end of the study each subject will be treated at the discretion of their physician, with other most suitable standard therapies and medical care, currently available locally for his/her condition.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2018-06-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-06-27

P. End of Trial
P.	End of Trial Status	Completed

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