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Study Evaluating Efficacy and Safety of Octanorm in Patients With Dermatomyositis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03686969
Recruitment Status : Terminated (Study complexity, low study recruitment)
First Posted : September 27, 2018
Last Update Posted : February 22, 2019
Sponsor:
Information provided by (Responsible Party):
Octapharma

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

Condition or disease Intervention/treatment Phase
Dermatomyositis Drug: Octanorm Other: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Dermatomyositis (SCGAM-02)
Actual Study Start Date : August 2, 2018
Actual Primary Completion Date : November 22, 2018
Actual Study Completion Date : November 29, 2018


Arm Intervention/treatment
Experimental: Octanorm
0.5g/kg/week octanorm 16.5%
Drug: Octanorm
Octanorm 0.5g/kg/week

Placebo Comparator: Placebo
Placebo
Other: Placebo
Placebo




Primary Outcome Measures :
  1. MMT-8 [ Time Frame: 32 weeks ]
    MMT-8; a set of 8 designated muscles tested bilaterally [potential score 0 - 150]

  2. CDASI [ Time Frame: 32 weeks ]
    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.

  3. Physician's Global Disease Activity VAS worsening [ Time Frame: 32 weeks ]
    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).


Secondary Outcome Measures :
  1. Extra-Muscular Disease Activity [ Time Frame: 32 weeks ]
    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).

  2. Muscle enzymes - aldolase [ Time Frame: 32 weeks ]
    Measurement of aldolase in blood

  3. Muscle enzymes - creatine kinase [ Time Frame: 32 weeks ]
    Measurement of creatine kinase in blood

  4. Muscle enzymes - alanine aminotransferase [ Time Frame: 32 weeks ]
    Measurement of alanine aminotransferase in blood

  5. Muscle enzymes - aspartate aminotransferase [ Time Frame: 32 weeks ]
    Measurement of aspartate aminotransferase in blood

  6. Muscle enzymes - lactate dehydrogenase [ Time Frame: 32 weeks ]
    Measurement of lactate dehydrogenase in blood

  7. Health Assessment Questionnaire [ Time Frame: 32 weeks ]
    • Health Assessment Questionnaire (HAQ; a generic rather than a disease-specific instrument; comprised of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 [without any difficulty] to 3 [unable to do]. For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8).

  8. SF-36v2 Health Survey [ Time Frame: 32 weeks ]
    The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index.

  9. Mean change in TIS [ Time Frame: 32 weeks ]
    Total Improvement Score

  10. Time to clinically important deterioration [ Time Frame: 32 weeks ]
    Time to clinically important deterioration

  11. Adverse Events [ Time Frame: 32 weeks ]
    Occurrence of all adverse events

  12. TEEs [ Time Frame: 32 weeks ]
    Monitoring safety with occurrence of all thromboembolic events (TEEs)

  13. HTRs [ Time Frame: 32 weeks ]
    Monitoring safety with occurrence of all hemolytic transfusion reactions (HTRs)

  14. Injection Site Reactions [ Time Frame: 32 weeks ]
    Monitoring safety by assessing local injection site reactions

  15. Blood Pressure [ Time Frame: 32 weeks ]
    Monitoring safety through blood pressure values

  16. Heart Rate [ Time Frame: 32 weeks ]
    Monitoring safety through heart rate values

  17. Body Temperature [ Time Frame: 32 weeks ]
    Monitoring safety through body temperature values

  18. Respiratory Rate [ Time Frame: 32 weeks ]
    Monitoring safety through respiratory rate values

  19. Physical Examination [ Time Frame: 33 Weeks32 weeks ]
    The physical examination outcome will be analyzed based on changes from baseline as adverse events.

  20. Sodium [ Time Frame: 32 weeks ]
    Monitoring safety through lab sodium levels

  21. Potassium [ Time Frame: 32 weeks ]
    Monitoring safety through lab potassium levels

  22. Glucose [ Time Frame: 32 weeks ]
    Monitoring safety through lab glucose levels

  23. ALAT [ Time Frame: 32 weeks ]
    Monitoring safety through lab ALAT levels

  24. ASAT [ Time Frame: 32 weeks ]
    Monitoring safety through lab ASAT levels

  25. LDH [ Time Frame: 32 weeks ]
    Monitoring safety through lab LDH levels

  26. Total Bilirubin [ Time Frame: 32 weeks ]
    Monitoring safety through lab total bilirubin levels

  27. Blood Urea Nitrogen [ Time Frame: 32 weeks ]
    Monitoring safety through lab blood urea nitrogen levels

  28. Urea [ Time Frame: 32 weeks ]
    Monitoring safety through lab urea levels

  29. Creatinine [ Time Frame: 32 weeks ]
    Monitoring safety through lab creatinine levels

  30. Albumin [ Time Frame: 32 weeks ]
    Monitoring safety through lab albumin levels

  31. Hematocrit [ Time Frame: 32 weeks ]
    Monitoring safety through lab hematocrit levels

  32. Hemoglobin [ Time Frame: 32 weeks ]
    Monitoring safety through lab hemoglobin levels

  33. Red Blood Cell Count [ Time Frame: 32 weeks ]
    Monitoring safety through lab red blood cell count levels

  34. White Blood Cell Count [ Time Frame: 32 weeks ]
    Monitoring safety through lab white blood cell count levels

  35. Platelets [ Time Frame: 32 weeks ]
    Monitoring safety through lab platelet levels

  36. Serum Haptoglobin [ Time Frame: 32 weeks ]
    Monitoring safety through lab serum haptoglobin levels

  37. Plasma-Free Hemoglobin [ Time Frame: 32 weeks ]
    Monitoring safety through lab plasma-free hemoglobin

  38. Direct Coombs' Test [ Time Frame: 32 weeks ]
    Monitoring safety through Direct Coombs' test

  39. D-dimers [ Time Frame: 32 weeks ]
    Monitoring safety through D-dimers test

  40. Serum IgG [ Time Frame: 32 weeks ]
    Monitoring safety through lab IgG levels

  41. Aldolase [ Time Frame: 32 weeks ]
    Monitoring safety through lab aldolase levels

  42. Creatine Kinase [ Time Frame: 32 weeks ]
    Monitoring safety through lab creatine kinase levels

  43. Pregnancy Test [ Time Frame: 32 weeks ]
    Monitoring safety through pregnancy test

  44. Urine Protein [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine protein levels

  45. Urine Glucose [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine glucose levels

  46. Urine pH [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine pH levels

  47. Urine Nitrite [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine nitrite levels

  48. Urine Ketones [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine ketone levels

  49. Urine Leukocytes [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine leukocyte levels

  50. Urine Hemoglobin [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine hemoglobin levels

  51. Urine Bilirubin [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine bilirubin levels

  52. Urine Urobilinogen [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine urobilinogen levels

  53. Urine Hemosiderin [ Time Frame: 32 weeks ]
    Monitoring safety through lab urine hemosiderin levels

  54. HIV [ Time Frame: 32 weeks ]
    Monitoring safety through HIV testing

  55. Hepatitis B [ Time Frame: 32 weeks ]
    Monitoring safety through hepatitis B testing

  56. Hepatitis C [ Time Frame: 32 weeks ]
    Monitoring safety through hepatitis C testing



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
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 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.

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 the 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. Subjects >5 years (>10 years for breast cancer) of cancer diagnosis who have been treated and are in remission are allowed.
  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 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 Table 2: see section 4.2.1.
  8. Subjects with administration of forbidden concomitant medications within the washout periods as defined in Table 3: see section 4.2.2.
  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), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease.
  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 ever.
  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 (e.g. protein-losing enteropathies, nephrotic syndrome).
  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. Subjects who participated in the Octagam 10% Dermatomyositis Study (GAM10-08) can be included.
  22. 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 7.3.9b) up to four weeks after the last IMP infusion received

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): NCT03686969


Locations
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Russian Federation
I.M. SECHENOV FIRST MOSCOW STATE MEDICAL UNIVERSITY Rheumatology Department Of, Clinici Of Nephrology
Moscow, Russian Federation, 119992
Sponsors and Collaborators
Octapharma
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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT03686969    
Other Study ID Numbers: SCGAM-02
First Posted: September 27, 2018    Key Record Dates
Last Update Posted: February 22, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Dermatomyositis
Polymyositis
Myositis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases