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DermatomyositisPhase 3NCT03686969

Study Evaluating Efficacy and Safety of Octanorm in Patients With Dermatomyositis

A Phase 3 study testing Octanorm in people with Dermatomyositis, run by Octapharma at 1 site in Russia.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Dermatomyositis
What is being tested?
Octanorm
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
1 participants
Where
1 siteRussia
Study dates
2 Aug 2018to 29 Nov 2018
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 22Show fewer
  • 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.

Trial locations and contacts

1 site in 1 country.

Russia1 site
  • I.M. SECHENOV FIRST MOSCOW STATE MEDICAL UNIVERSITY Rheumatology Department Of, Clinici Of Nephrology

    Moscow, 119992

Other trials in Russia

About this study

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)

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

Conditions
Dermatomyositis
Being tested
Octanorm, Placebo
Medical subject headings
Dermatomyositis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • OctanormDrug

    Octanorm 0.5g/kg/week

  • PlaceboOther

    Placebo

Study groups in NCT03686969
Study groupRoleWhat they receive
OctanormExperimental0.5g/kg/week octanorm 16.5%
Receives: Drug: Octanorm
PlaceboPlacebo comparatorPlacebo
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. MMT-8

    MMT-8; a set of 8 designated muscles tested bilaterally [potential score 0 - 150]

    Measured: 32 weeks

  2. 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.

    Measured: 32 weeks

  3. 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).

    Measured: 32 weeks

Secondary outcomes (56)
  1. 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).

    Measured: 32 weeks

  2. Muscle Enzymes - Aldolase

    Measurement of aldolase in blood

    Measured: 32 weeks

  3. Muscle Enzymes - Creatine Kinase

    Measurement of creatine kinase in blood

    Measured: 32 weeks

  4. Muscle Enzymes - Alanine Aminotransferase

    Measurement of alanine aminotransferase in blood

    Measured: 32 weeks

  5. Muscle Enzymes - Aspartate Aminotransferase

    Measurement of aspartate aminotransferase in blood

    Measured: 32 weeks

  6. Muscle Enzymes - Lactate Dehydrogenase

    Measurement of lactate dehydrogenase in blood

    Measured: 32 weeks

  7. Health Assessment Questionnaire

    • 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).

    Measured: 32 weeks

  8. SF-36v2 Health Survey

    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.

    Measured: 32 weeks

  9. Mean Change in TIS

    Total Improvement Score

    Measured: 32 weeks

  10. Time to Clinically Important Deterioration

    Time to clinically important deterioration

    Measured: 32 weeks

  11. Adverse Events

    Occurrence of all adverse events

    Measured: 32 weeks

  12. TEEs

    Monitoring safety with occurrence of all thromboembolic events (TEEs)

    Measured: 32 weeks

  13. HTRs

    Monitoring safety with occurrence of all hemolytic transfusion reactions (HTRs)

    Measured: 32 weeks

  14. Injection Site Reactions

    Monitoring safety by assessing local injection site reactions

    Measured: 32 weeks

  15. Blood Pressure

    Monitoring safety through blood pressure values

    Measured: 32 weeks

  16. Heart Rate

    Monitoring safety through heart rate values

    Measured: 32 weeks

  17. Body Temperature

    Monitoring safety through body temperature values

    Measured: 32 weeks

  18. Respiratory Rate

    Monitoring safety through respiratory rate values

    Measured: 32 weeks

  19. Physical Examination

    The physical examination outcome will be analyzed based on changes from baseline as adverse events.

    Measured: 32 Weeks

  20. Sodium

    Monitoring safety through lab sodium levels

    Measured: 32 weeks

  21. Potassium

    Monitoring safety through lab potassium levels

    Measured: 32 weeks

  22. Glucose

    Monitoring safety through lab glucose levels

    Measured: 32 weeks

  23. ALAT

    Monitoring safety through lab ALAT levels

    Measured: 32 weeks

  24. ASAT

    Monitoring safety through lab ASAT levels

    Measured: 32 weeks

  25. LDH

    Monitoring safety through lab LDH levels

    Measured: 32 weeks

  26. Total Bilirubin

    Monitoring safety through lab total bilirubin levels

    Measured: 32 weeks

  27. Blood Urea Nitrogen

    Monitoring safety through lab blood urea nitrogen levels

    Measured: 32 weeks

  28. Urea

    Monitoring safety through lab urea levels

    Measured: 32 weeks

  29. Creatinine

    Monitoring safety through lab creatinine levels

    Measured: 32 weeks

  30. Albumin

    Monitoring safety through lab albumin levels

    Measured: 32 weeks

  31. Hematocrit

    Monitoring safety through lab hematocrit levels

    Measured: 32 weeks

  32. Hemoglobin

    Monitoring safety through lab hemoglobin levels

    Measured: 32 weeks

  33. Red Blood Cell Count

    Monitoring safety through lab red blood cell count levels

    Measured: 32 weeks

  34. White Blood Cell Count

    Monitoring safety through lab white blood cell count levels

    Measured: 32 weeks

  35. Platelets

    Monitoring safety through lab platelet levels

    Measured: 32 weeks

  36. Serum Haptoglobin

    Monitoring safety through lab serum haptoglobin levels

    Measured: 32 weeks

  37. Plasma-Free Hemoglobin

    Monitoring safety through lab plasma-free hemoglobin

    Measured: 32 weeks

  38. Direct Coombs' Test

    Monitoring safety through Direct Coombs' test

    Measured: 32 weeks

  39. D-dimers

    Monitoring safety through D-dimers test

    Measured: 32 weeks

  40. Serum IgG

    Monitoring safety through lab IgG levels

    Measured: 32 weeks

  41. Aldolase

    Monitoring safety through lab aldolase levels

    Measured: 32 weeks

  42. Creatine Kinase

    Monitoring safety through lab creatine kinase levels

    Measured: 32 weeks

  43. Pregnancy Test

    Monitoring safety through pregnancy test

    Measured: 32 weeks

  44. Urine Protein

    Monitoring safety through lab urine protein levels

    Measured: 32 weeks

  45. Urine Glucose

    Monitoring safety through lab urine glucose levels

    Measured: 32 weeks

  46. Urine pH

    Monitoring safety through lab urine pH levels

    Measured: 32 weeks

  47. Urine Nitrite

    Monitoring safety through lab urine nitrite levels

    Measured: 32 weeks

  48. Urine Ketones

    Monitoring safety through lab urine ketone levels

    Measured: 32 weeks

  49. Urine Leukocytes

    Monitoring safety through lab urine leukocyte levels

    Measured: 32 weeks

  50. Urine Hemoglobin

    Monitoring safety through lab urine hemoglobin levels

    Measured: 32 weeks

  51. Urine Bilirubin

    Monitoring safety through lab urine bilirubin levels

    Measured: 32 weeks

  52. Urine Urobilinogen

    Monitoring safety through lab urine urobilinogen levels

    Measured: 32 weeks

  53. Urine Hemosiderin

    Monitoring safety through lab urine hemosiderin levels

    Measured: 32 weeks

  54. HIV

    Monitoring safety through HIV testing

    Measured: 32 weeks

  55. Hepatitis B

    Monitoring safety through hepatitis B testing

    Measured: 32 weeks

  56. Hepatitis C

    Monitoring safety through hepatitis C testing

    Measured: 32 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Sep 2018
  2. Study start2 Aug 2018
  3. Primary completion22 Nov 2018
  4. Study completion29 Nov 2018

Why it stopped: Study complexity, low study recruitment

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
1 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03686969
Sponsor's protocol ID
SCGAM-02
Status last verified
Apr 2021
First submitted
31 Aug 2018
Last updated
23 Apr 2021
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Octanorm in people with Dermatomyositis, run by Octapharma at 1 site in Russia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 23 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

MMT-8 (time frame: 32 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Apr 2021.

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