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NCT02728752: IIM

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

Completed Phase 3 Results posted Last updated 23 December 2021
What this trial tests

Phase 3 trial testing Octagam 10% in Dermatomyositis in 95 participants. Completed in 5 November 2019.

Timeline
27 February 2017
Primary endpoint
5 November 2019
5 November 2019

Quick facts

Lead sponsorOctapharma
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingtriple
Primary purposetreatment
Enrollment95
Start date27 February 2017
Primary completion5 November 2019
Estimated completion5 November 2019
Sites37 locations across Netherlands, Russia, Ukraine, Germany, Hungary, Poland, Romania, Canada

Drugs / interventions tested

Conditions studied

Sponsor

Octapharma — full company profile →

Who can join

Adults 18 to 79, any sex, with Dermatomyositis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Measure the Number of Patients Who Had an Increase of ≥20 Points on the Total Improvement Score (TIS) Primary · 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

GroupValue95% CI
Placebo21
Octagam10%37
Proportion of TIS Responders by Improvement Category at Week 16 Secondary · 16 weeks

The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement. Primary: Total number of all patients who had at least minimal, moderate, or major response. At Least Moderate: Number of patients who had moderate or major response. At Least Major: Number of patients who had a major response.

Primary
GroupValue95% CI
Placebo21
Octagam10%37
At least moderate improvement
GroupValue95% CI
Placebo11
Octagam10%32
At least major improvement
GroupValue95% CI
Placebo4
Octagam10%15
Proportion of TIS Responders by Improvement Category at Week 40 Secondary · 40 weeks

The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement. At Least Minimal: Total number of all patients who had minimal, moderate, or major response. At Least Moderate: Number of patients who had moderate or major response. At Least Major: Number of patients who had a major response.

At least minimal improvement
GroupValue95% CI
Placebo32
Octagam10%32
At least moderate improvement
GroupValue95% CI
Placebo28
Octagam10%26
At least major improvement
GroupValue95% CI
Placebo14
Octagam10%17
Mean Change From Baseline (Week 0) to End of First Period (Week 16) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Secondary · First 16 weeks

The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed. Absence of skin lesions as described above is scored with "0" for each of the above described areas. Skin lesion will be scored with at least "1" (present), some lesions will be graded from "1" (less severe) to "2" (severe); others with "1", "2" or "3". For the tota

Total Activity Score
GroupValue95% CI
Placebo-1.16± 7.000
Octagam10%-9.36± 10.542
Total Damage Score
GroupValue95% CI
Placebo-0.02± 0.771
Octagam10%-0.67± 1.871
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) Secondary · From week 16 to Week 40

The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed Absence of skin lesions as described above is scored with "0" for each of the above described areas. Skin lesion will be scored with at least "1" (present), some lesions will be graded from "1" (less severe) to "2" (severe); others with "1", "2" or "3". For the total

Total Activity Score
GroupValue95% CI
Placebo-8.52± 11.344
Octagam10%-1.79± 4.169
Total Damage Score
GroupValue95% CI
Placebo-0.24± 0.969
Octagam10%0.26± 2.220
Mean Change From Baseline (Week 0) to End of Extension Period (Week 40) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Secondary · 40 weeks

The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed. Absence of skin lesions as described above is scored with "0" for each of the above described areas. Skin lesion will be scored with at least "1" (present), some lesions will be graded from "1" (less severe) to "2" (severe); others with "1", "2" or "3". For the tota

Total Activity Score
GroupValue95% CI
Placebo-10.44± 10.034
Octagam10%-10.03± 8.995
Total Damage Score
GroupValue95% CI
Placebo-0.26± 1.197
Octagam10%-0.38± 2.523
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: SF-36v2 Health Survey Secondary · From start of the trial till Week 40

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. SF-36 scores ranging from 0 to 100. 0 represented the lowest possible score (worst health state) and 100 represented the highest possible score (best health state), with scores in between representing the percentages of the total possible score achieved by respondents on a given scale.

Baseline to Week 16
GroupValue95% CI
Placebo2.27± 4.598
Octagam10%7.06± 8.220
Baseline to Week 40
GroupValue95% CI
Placebo6.65± 8.258
Octagam10%8.77± 10.930
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Physician's Global Disease Activity Secondary · From start of the trial till Week 40

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). Assessment completed by physician. 0 is lowest score and 10 is highest score. Higher score associated with worse outcome.

Baseline to Week 16
GroupValue95% CI
Placebo-0.60± 1.815
Octagam10%-2.39± 1.987
Baseline to Week 40
GroupValue95% CI
Placebo-2.93± 1.888
Octagam10%-3.06± 1.817
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Patient Global Disease Activity Secondary · From start of the trial till Week 40

Patient's Global Disease Activity (10cm VAS assessing the overall activity of the patient's disease today from "No evidence of disease activity" to "Extremely active or severe disease activity", Disease Activity being active inflammation in the patient's muscles, skin, joints, intestines, heart, lungs or other parts of the body, which can improve when treated with medicines). 0 is lowest score and 10 is highest score. Higher score associated with worse outcome.

Baseline to Week 16
GroupValue95% CI
Placebo-1.11± 2.094
Octagam10%-2.19± 2.276
Baseline to Week 40
GroupValue95% CI
Placebo-2.77± 2.133
Octagam10%-2.71± 2.651
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: MMT-8 Secondary · From start of the trial till Week 40

Manual Muscle Testing - MMT-8; a set of 8 designated muscles tested bilaterally \[potential score 0 - 150\]. Higher score associated with better outcome.

Baseline to Week 16
GroupValue95% CI
Placebo3.21± 9.390
Octagam10%14.38± 14.581
Baseline to Week 40
GroupValue95% CI
Placebo12.00± 7.491
Octagam10%20.09± 14.486
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Health Assessment Questionnaire Secondary · From start of the trial till Week 40

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). Assessment completed by patients. Lowest score 0 highest score 24. Higher score associated with worse outcome.

Baseline to Week 16
GroupValue95% CI
Placebo-0.16± 0.366
Octagam10%-0.56± 0.590
Baseline to Week 40
GroupValue95% CI
Placebo-0.54± 0.524
Octagam10%-0.66± 0.805
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Alanine Aminotransferase) Secondary · From start of the trial till Week 40
Baseline to Week 16
GroupValue95% CI
Placebo-4.47± 29.796
Octagam10%-8.52± 18.474
Baseline to Week 40
GroupValue95% CI
Placebo-4.06± 14.787
Octagam10%-7.15± 18.480

Adverse events — posted to ClinicalTrials.gov

Time frame: 40 weeks. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

First Period Placebo
Serious: 2/48 (4%)
Deaths: 0/48
Overall Period Octagam
Serious: 14/95 (15%)
Deaths: 0/95

Serious adverse events (21 terms)

ReactionSystemFirst Period PlaceboOverall Period Octagam
Pulmonary embolismRespiratory, thoracic and mediastinal disorders
SepsisInfections and infestations
Condition aggravatedGeneral disorders
Sinus tachycardiaCardiac disorders
Ventricular extrasystolesCardiac disorders
Tropical spastic paresisInfections and infestations
Muscle spasmsMusculoskeletal and connective tissue disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
HypertensionVascular disorders
Cardiac failure congestiveCardiac disorders
Atypical pneumoniaInfections and infestations
Escherichia bacteraemiaInfections and infestations
PneumoniaInfections and infestations
Squamous cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Cerebral infarctionNervous system disorders
Cerebrovascular accidentNervous system disorders
HypoaesthesiaNervous system disorders
Loss of consciousnessNervous system disorders
Acute respiratory failureRespiratory, thoracic and mediastinal disorders
Deep vein thrombosisVascular disorders
Acute kidney injuryRenal and urinary disorders
Other adverse events (197 terms — click to expand)

ReactionSystemFirst Period PlaceboOverall Period Octagam
HeadacheNervous system disorders
NauseaGastrointestinal disorders
PyrexiaGeneral disorders
VomitingGastrointestinal disorders
Condition aggravatedGeneral disorders
MyalgiaMusculoskeletal and connective tissue disorders
ChillsGeneral disorders
ArthralgiaMusculoskeletal and connective tissue disorders
NasopharyngitisInfections and infestations
RashSkin and subcutaneous tissue disorders
Pain in extremityMusculoskeletal and connective tissue disorders
TachycardiaCardiac disorders
ConstipationGastrointestinal disorders
FatigueGeneral disorders
Body temperature increasedInvestigations
Coombs test positiveInvestigations
DizzinessNervous system disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
HypertensionVascular disorders
DiarrhoeaGastrointestinal disorders
Blood pressure increasedInvestigations
Haemoglobin decreasedInvestigations
Back painMusculoskeletal and connective tissue disorders
CoughRespiratory, thoracic and mediastinal disorders
AstheniaGeneral disorders
Chest painGeneral disorders
Oedema peripheralGeneral disorders
Peripheral swellingGeneral disorders
BronchitisInfections and infestations
Upper respiratory tract infectionInfections and infestations
Blood pressure systolic increasedInvestigations
Muscular weaknessMusculoskeletal and connective tissue disorders
AnaemiaBlood and lymphatic system disorders
LymphopeniaBlood and lymphatic system disorders
BradycardiaCardiac disorders
PalpitationsCardiac disorders
VertigoEar and labyrinth disorders
Vision blurredEye disorders
Abdominal painGastrointestinal disorders
Abdominal pain upperGastrointestinal disorders

Most-reported serious reactions: Pulmonary embolism, Sepsis, Condition aggravated, Sinus tachycardia, Ventricular extrasystoles, Tropical spastic paresis, Muscle spasms, Dyspnoea.

Data from ClinicalTrials.gov NCT02728752 adverse events section.

Sponsor's own description

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

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Current Classification and Management of Inflammatory Myopathies.
    Schmidt J. · · 2018 · cited 167× · PMID 29865091 · DOI 10.3233/jnd-180308
  2. Trial of Intravenous Immune Globulin in Dermatomyositis.
    Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csörgő Z, et al · · 2022 · cited 119× · PMID 36198179 · DOI 10.1056/nejmoa2117912
  3. Autoimmune Myopathies: Updates on Evaluation and Treatment.
    McGrath ER, Doughty CT, Amato AA. · · 2018 · cited 50× · PMID 30341597 · DOI 10.1007/s13311-018-00676-2
  4. Prospective, double-blind, randomized, placebo-controlled phase III study evaluating efficacy and safety of octagam 10% in patients with dermatomyositis ("ProDERM Study").
    Aggarwal R, Charles-Schoeman C, Schessl J, Dimachkie MM, et al · · 2021 · cited 36× · PMID 33429735 · DOI 10.1097/md.0000000000023677
  5. New insights into the treatment of myositis.
    Glaubitz S, Zeng R, Schmidt J. · · 2020 · cited 30× · PMID 31949477 · DOI 10.1177/1759720x19886494
  6. Intravenous immunoglobulins as first-line treatment in idiopathic inflammatory myopathies: a pilot study.
    Lim J, Eftimov F, Verhamme C, Brusse E, et al · · 2021 · cited 28× · PMID 33099648 · DOI 10.1093/rheumatology/keaa459
  7. Antibody Therapies in Autoimmune Inflammatory Myopathies: Promising Treatment Options.
    Zeng R, Glaubitz S, Schmidt J. · · 2022 · cited 15× · PMID 35394612 · DOI 10.1007/s13311-022-01220-z
  8. Safety and tolerability of intravenous immunoglobulin in patients with active dermatomyositis: results from the randomised, placebo-controlled ProDERM study.
    Aggarwal R, Schessl J, Charles-Schoeman C, Bata-Csörgő Z, et al · · 2024 · cited 12× · PMID 38233885 · DOI 10.1186/s13075-023-03232-2

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