Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis (Idiopathic Inflammatory Myopathy)

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

95 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-02-27

Study Completion Date

2019-11-05

Brief Summary

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Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study")

Detailed Description

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Conditions

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Dermatomyositis

Keywords

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DM

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Placebo

Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Patients to be treated with a Placebo

Octagam10%

Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.

Group Type EXPERIMENTAL

Octagam 10%

Intervention Type DRUG

Patients to be treated with Octagam 10%

Interventions

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Octagam 10%

Patients to be treated with Octagam 10%

Intervention Type DRUG

Placebo

Patients to be treated with a Placebo

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Subjects with diagnosis of definite or probable DM according to the Bohan and Peter criteria.
2. Subjects under treatment with corticosteroids and/or maximally 2 immune-suppressants and being on stable therapy for at least 4 weeks (see Section 4.2.1) OR Subjects with previous failure of response or previous intolerance to corticosteroid and at least 1 additional immunosuppressive drug, and with steroid/immunosuppressive drugs washed out as per Section 4.2.1 (Table 2).
3. Subjects with active disease, assessed and agreed upon by an independent adjudication committee.
4. Manual Muscle Testing-8 (MMT-8) score \<142, with at least 2 other abnormal Core Set Measures (CSM) (Visual Analogue Scale \[VAS\] of patient global activity ≥2 cm, physician's global disease activity ≥2 cm, extra-muscular activity ≥2 cm; at least one muscle enzyme \>1.5 times upper limit of normal, Health Assessment Questionnaire ≥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 to understand and comply with the relevant aspects of the study protocol.

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 and at least 1 or 5 years, respectively, have passed since excision).
2. 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.
3. Subjects with overlap myositis (except for overlap with Sjögren's syndrome), 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 have received IgG treatment within the last 6 months before enrolment.
7. Subjects who received blood or plasma-derived products (other than IgG) or plasma exchange within the last 3 months before enrolment.
8. Subjects starting or planning to start a physical therapy-directed exercise regimen during the trial.
9. Cardiac insufficiency (New York Heart Association III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease.
10. Severe liver disease, with signs of ascites and hepatic encephalopathy.
11. Severe kidney disease (as defined by estimated glomerular filtration rate (eGFR) \< 30 mL/min/1.73 m2).
12. Known hepatitis B, hepatitis C or HIV infection.
13. Subjects with a history of TEE such as deep vein thrombosis, pulmonary embolism, myocardial infarction, ischemic stroke, transient ischemic attack, peripheral artery disease (Fontaine IV) ever.
14. Body mass index ≥40 kg/m2.
15. Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).
16. Known IgA deficiency with antibodies to IgA.
17. History of hypersensitivity, anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products or any component of Octagam 10%.
18. Known blood hyperviscosity, or other hypercoagulable states.
19. Subjects with a history of drug abuse within the past 5 years prior to study enrollment.
20. Subjects unable or unwilling to understand or comply with the study protocol.
21. Participating in another interventional clinical study with investigational treatment within 3 months prior to study enrollment.
22. Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to apply an effective birth control method (such as implants, injectables, combined oral contraceptives, some intrauterine devices \[IUDs\], sexual abstinence or vasectomized partner) up to four weeks after the last IMP infusion.
23. Subjects who are accommodated in an institution or care facility based on an official directive or court order.
24. Subjects who are in any way dependent on the Sponsor, Investigator or Study Site.
25. Subjects who received forbidden medication within the washout period as defined in Section 4.2.2 (Table 3).

Minimum Eligible Age

18 Years

Maximum Eligible Age

79 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Wolfgang Frenzel

Role: STUDY_DIRECTOR

International Medical Director

PMID: 33429735 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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GAM10-08

Identifier Type: -

Identifier Source: org_study_id

Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis (Idiopathic Inflammatory Myopathy)

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

Placebo

Placebo

Octagam10%

Octagam 10%

Interventions

Octagam 10%

Placebo

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Octapharma

Responsible Party

Principal Investigators

Wolfgang Frenzel

Locations

Octapharma Research Site

Octapharma Research Site

University of California -Irvine

Octapharma Research Site

NeuroMedical Research Center

Octapharma Research Site

University of South Florida

Octapharma Research Site

Octapharma Research Site

Octapharma Research Site

Octapharma Research Site

Octapharma Research Site

Octapharma Research Site

Stones River Dermatology

Austin Neuromuscular Center

Octapharma Research Site

Arthritis & Osteoporosis Clinic

Octapharma Research Site

Revmatologický ústav

Charité-Universitätsmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie

Uniklinikum Münster, Klinik für Hautkrankheiten

Semmelweis University Dermatology Clinic

University of Debrecen Dept of Internal Medicine

University of Szeged Dermatology Clinic

Academic Medical Centre University of Amsterdam

Centrum Medyczne Plejady

Narodowy Instytut Geriatrii, Reumatologii I Rehabilitacji - Warsaw

Octapharma Research Site

Scientific Research Institute for Rheumatology (Moscow)

I.M. Sechenov First Moscow State Medical University

Orenburg State Medical University Based On Regional Clinical Hospital

3rd Rheumatology Department Of Clinical Rheumatology Hospital No. 25

AVA-Peter clinic (Saint-Petersburg)

Octapharma Research Site

Octapharma Research Site

Octapharma Research Site

Octapharma Research Site

Countries

References

Provided Documents

Document Type: Study Protocol

Document Type: Statistical Analysis Plan

Other Identifiers

GAM10-08