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

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ClinicalTrials.gov Identifier: NCT02728752
Recruitment Status : Recruiting
First Posted : April 5, 2016
Last Update Posted : March 5, 2018
Sponsor:
Information provided by (Responsible Party):
Octapharma

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

Condition or disease Intervention/treatment Phase
Dermatomyositis Drug: Octagam 10% Other: Placebo Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 94 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM Study")
Actual Study Start Date : February 27, 2017
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : March 2019


Arm Intervention/treatment
Placebo Comparator: Placebo
Subjects eligible after screening, randomized to placebo arm, will receive 4 infusions of placebo every 4 weeks during the First Period (16 weeks). In case of deterioration at or after Week 6, subjects will be crossed-over to the alternate treatment, and will not drop-out before the primary endpoint assessment at Week 16 to keep the blind unless they deteriorate also on the alternate IMP after starting it at least 6 weeks before. After response assessment at Week 16, patients will be unblinded. From Week 16 onwards, all subjects, except subjects randomized to Octagam 10% who deteriorate in the First Period and who will drop-out, will enter the open-label Extension Period. In the Extension Period they will receive six infusions of 2.0 g/kg Octagam 10% every 4 weeks (± 4 days). 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.
Other: Placebo
Patients to be treated with a Placebo
Experimental: Octagam10%
Subjects eligible after screening, randomized to Octagam10% arm, will receive 4 infusions of Octagam10% every 4 weeks during the First Period (16 weeks). In case of deterioration at or after Week 6, subjects will be crossed-over to the alternate treatment, and will not drop-out before the primary endpoint assessment at Week 16 to keep the blind unless they deteriorate also on the alternate IMP after starting it at least 6 weeks before. After response assessment at Week 16, patients will be unblinded. From Week 16 onwards, all subjects, except subjects randomized to Octagam10% who deteriorate in the First Period and who will drop-out, will enter the open-label Extension Period. In the Extension Period they will receive six infusions of 2.0 g/kg Octagam 10% every 4 weeks (± 4 days). 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.
Drug: Octagam 10%
Patients to be treated with Octagam 10%



Primary Outcome Measures :
  1. Measure the number of patients who had an increase of ≥20 points on the Total Improvement Score (TIS) [ Time Frame: At week 16 ]
    Proportion of responders in the 2.0 g/kg Octagam 10% and placebo arms at Week 16 relative to baseline (Week 0). A responder being defined as a patient with an increase of ≥20 points on the Total Improvement Score (TIS, a scale from 0 to 100; 20-39 points being minimal improvement, 40-59 points being moderate improvement, and ≥60 points being major improvement


Secondary Outcome Measures :
  1. Mean change from baseline (Week 0) to end of first period (Week 16) in: Total Improvement Score (TIS) [ Time Frame: First 16 weeks ]
  2. Mean change from baseline (Week 0) to Week 40 in: Total Improvement Score (TIS) [ Time Frame: First 40 weeks ]
  3. Mean change from baseline (Week 0) to end of first period (Week 16) in the modified: Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) [ Time Frame: First 16 weeks ]
  4. Mean change from end of first period (Week 16) to end of extension period (Week 40) in: Total Improvement Score (TIS) [ Time Frame: From week 16 to Week 40 ]
  5. Mean change from end of first period (Week 16) to end of extension period (Week 40) in the modified: Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) [ Time Frame: From week 16 to Week 40 ]
  6. Mean change from baseline (Week 0) to end of first period (Week 16) and extension period (Week 40) in: SF-36v2 Health Survey [ Time Frame: From start of the trial till Week 40 ]
  7. Mean change from baseline (Week 0) to end of first period (Week 16) and extension period (Week 40) in: Individual six core set measures (CSM) from Total Improvement Score (TIS) [ Time Frame: From start of the trial till Week 40 ]
  8. Proportion of Total Improvement Score (TIS) responders at the end of the extension period (Week 40) relative to Week 16 or Week 0 [ Time Frame: Week 40 only ]

Other Outcome Measures:
  1. Observe the occurrence of adverse events with a particular emphasis on Thromboembolic events (TEE) and Hemolytic Transfusion Reactions (HTR) [ Time Frame: throught the length of the trial ]
  2. Measurement of Vitals (blood pressure, heart rate, body temperature and respiratory rate) [ Time Frame: throught the length of the trial ]
  3. Physical examination [ Time Frame: at Screening and every 12 weeks from Week 4 on ]
  4. Laboratory parameters - hematology, clinical chemistry [ Time Frame: throught the length of the trial ]


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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  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 5 years 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 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, E.g. hepatitis or cirrhosis.
  11. Severe kidney disease (creatinine 1.5 times above the upper limit of normal).
  12. Known hepatitis B, hepatitis C or HIV infection.
  13. Subjects with a history of deep vein thrombosis within the last year prior to study enrollment or pulmonary embolism 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 while on study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02728752


Contacts
Contact: Mikaela Grupp 866-337-1868 ctgov@clinicalresearchmgt.com

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Sponsors and Collaborators
Octapharma
Investigators
Study Director: Wolfgang Frenzel International Medical Monitor

Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT02728752     History of Changes
Other Study ID Numbers: GAM10-08
First Posted: April 5, 2016    Key Record Dates
Last Update Posted: March 5, 2018
Last Verified: March 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Octapharma:
DM

Additional relevant MeSH terms:
Muscular Diseases
Dermatomyositis
Myositis
Polymyositis
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases
gamma-Globulins
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Physiological Effects of Drugs