Study Summary
This trial will test if ravulizumab is an effective treatment for dermatomyositis, a disease that causes muscle weakness and skin rashes.
Treatment Effectiveness
Phase-Based Effectiveness
Phase 2 & 3
Study Objectives
6 Primary · 45 Secondary · Reporting Duration: Week 2 through Week 50
Baseline through Week 26
Part A and Part B: CDASI Response (7-point improvement)
Part A and Part B: CDASI Response (7-point improvement) at Week 26
Part A and Part B: Clinical Worsening During RCP At Two Consecutive Visits
Part A and Part B: Clinical Worsening During Randomized Controlled Period At Two Consecutive Visits
Part A and Part B: Receipt of Rescue Therapy
Part A and Part B: Time To First Response Of TIS20, TIS40, Or TIS60
Part A: Proportion Of Participants With A ≥ 40-point Improvement Response On IMACS-TIS (TIS40) At Each Visit
Part A: Proportion Of Participants With A ≥ 60-point Improvement Response On IMACS-TIS (TIS60) At Each Visit
Part A: Proportion Of Participants With TIS20 At Each Visit
Part A: Time To First CDASI Activity Score Improvement
Part A: Time To First IMACS Myositis Core Set Measure Improvements
Part A: Time To First Response Of TIS20, TIS40, Or TIS60
Baseline through Week 50
Part B: Proportion Of Participants With TIS20 At Each Visit
Part B: Proportion Of Participants With TIS40 At Each Visit
Part B: Proportion Of Participants With TIS60 At Each Visit
Part B: Time To First CDASI Activity Score Improvement
Part B: Time To First IMACS Myositis Core Set Measure Improvements
Part B: Time To First Response Of TIS20, TIS40, Or TIS60
Baseline, Week 26
Part A and Part B: Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score At Week 26
Part A and Part B: Change From Baseline In Five IMACS Core Set Measures (extra-muscular disease activity based on MDAAT, physician global activity assessment, patient global activity assessment, MMT-8, HAQ) At Week 26
Part A and Part B: Change From Baseline In Five IMACS Core Set Measures At Week 26
Part A and Part B: Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response At Week 26
Part A: Change From Baseline Of Each Of The IMACS Core Set Measures At Week 26
Part A: Change In Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) At Week 26
Part A: Change In Investigator Global Assessment Scale For Skin Activity (IGA-SA) At Week 26
Part A: Mean Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score At Week 26
Baseline, Week 50
Part B: Mean Change From Baseline In CDASI Activity Score At Week 50
Part B: Mean Change From Baseline In Extra-muscular Disease Activity Based On Myositis Disease Activity Assessment Tool (MDAAT) At Week 50
Part B: Mean Change From Baseline In Health Assessment Questionnaire (HAQ) At Week 50
Part B: Mean Change From Baseline In Manual Muscle Testing Subset Of 8 Muscles (MMT-8) At Week 50
Part B: Mean Change From Baseline In Muscle Enzyme Values At Week 50
Part B: Mean Change From Baseline In Patient Global Activity Assessment At Week 50
Part B: Mean Change From Baseline In Physician Global Activity Assessment At Week 50
Week 50
Part B: Mean TIS At Each Visit From Week 2 Through Week 50
Week 26
Part A and B: TIS At Week 26
Part A and Part B: Response Related to Muscle Enzymes
Part A and Part B: Response Related to Muscle Enzymes: Normalization Of Most Abnormal Baseline Enzyme At Week 26
Part A and Part B: TIS20 Response at Week 26
Part A and Part B: TIS60 Response at Week 26
Part A: IMACS-TIS (TIS40) response at Week 26 of the Randomized Controlled Period as per defined composite estimand
Part A: IMACS-TISa (TIS40) response at Week 26 of the Randomized Controlled Period as per defined composite estimand
Part A: Mean TIS At Week 26
Myositis
Part A: Proportion Of Participants With CDASI MCID Improvement At Week 26
Part B: IMACS-TIS (TIS40) response at Week 26 of the Randomized Controlled Period as per defined composite estimand
Part B: IMACS-TISa (TIS40) response at Week 26 of the Randomized Controlled Period as per defined composite estimand
Week 50
Part B: Change In CDA-IGA At Week 50
Part B: Change In IGA-SA At Week 50
Part B: Mean TIS At Week 50
Part B: Proportion Of Participants With A ≥ 20-point Improvement Response On TIS20 At Week 50
Part B: Proportion Of Participants With CDASI MCID Improvement At Week 50
Trial Safety
Phase-Based Safety
This is further along than 68% of similar trials
Side Effects for
Ravulizumab
31%Headache
30%Nasopharyngitis
28%Upper respiratory tract infection
21%Fatigue
19%Pyrexia
19%Diarrhoea
18%Nausea
17%Cough
15%Abdominal pain
14%Back pain
14%Dizziness
13%Pain in extremity
11%Arthralgia
11%Influenza like illness
10%Rhinitis
10%Oropharyngeal pain
8%Abdominal pain upper
8%Urinary tract infection
8%Vomiting
8%Anaemia
8%Dyspnoea
7%Constipation
6%Chest pain
6%Dysphagia
5%Influenza
5%Palpitations
5%Gastroenteritis
5%Myalgia
5%Pruritus
3%Haemolysis
2%Haemolytic anaemia
2%Lower respiratory tract infection
1%Liver disorder
1%Ligament injury
1%Tibia fracture
1%Bile duct stone
1%Cholecystitis
1%Infection
1%Pneumonia
1%Bacteraemia
1%Escherichia urinary tract infection
1%Rhinovirus infection
1%Septic shock
1%Cerebrospinal fluid retention
1%Osteonecrosis
1%Basal cell carcinoma
1%COVID-19
1%Post procedural infection
1%Postoperative wound infection
1%Dupuytren's contracture
1%Urinary retention
1%Toothache
1%Biliary colic
1%Escherichia sepsis
1%Pharyngitis
1%Pneumococcal infection
1%Road traffic accident
1%Loss of consciousness
1%Intervertebral disc degeneration
1%Ureterolithiasis
1%Liver function test increased
1%Dermal cyst
1%Lung cancer metastatic
1%Renal cancer metastatic
1%Invasive papillary breast carcinoma
1%Pneumoperitoneum
1%Foot deformity
1%Endometrial cancer
1%Depression
1%Major depression
1%Suicide attempt
1%Aplastic anaemia
1%Breakthrough haemolysis
1%Seborrhoeic keratosis
1%Lower limb fracture
1%Pneumonia bacterial
1%Transfusion reaction
1%Deep vein thrombosis
1%Bone marrow failure
1%Ankle fracture
1%Enteritis
1%Suspected COVID-19
1%Colitis
1%Respiratory failure
1%Cholelithiasis
1%Hyperthermia
1%Epilepsy
Awards & Highlights
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Trial Design
2 Treatment Groups
Ravulizumab
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group
150 Total Participants · 2 Treatment Groups
Primary Treatment: Ravulizumab · Has Placebo Group · Phase 2 & 3
Ravulizumab
Drug
Experimental Group · 1 Intervention: Ravulizumab · Intervention Types: DrugPlacebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ravulizumab
FDA approved
Trial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: week 2 through week 50
Who is running the clinical trial?
AlexionLead Sponsor
239 Previous Clinical Trials
39,763 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
38,273 Total Patients Enrolled
Eligibility Criteria
Age 18+ · All Participants · 2 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:You have already tried one or more treatments for your condition, including medications like corticosteroids or immunosuppressive therapies, but they did not work well enough or you could not tolerate them.
Both males and females are eligible.
Who else is applying?
What state do they live in?
| Ohio | 100.0% |
How old are they?
| 18 - 65 | 100.0% |
What site did they apply to?
| Clinical Trial Site | 100.0% |
What portion of applicants met pre-screening criteria?
| Met criteria | 100.0% |
Frequently Asked Questions
What other data exists for Ravulizumab?
"Ravulizumab was first studied in 2016 at a clinical trial site. Since then, there have been 18245 completed trials. There are presently 16 actively recruiting trials, with many of these studies being performed in New York, New York." - Anonymous Online Contributor
Unverified Answer
Could you please list the various places where this research is conducted?
"There are 8 sites for this clinical trial, which are recruiting patients. The sites are located in New York, Orange and Baltimore, as well as 8 other locations. If you are considering participating, it would be helpful to choose the location nearest to you to reduce travel requirements." - Anonymous Online Contributor
Unverified Answer
What are some conditions that ravulizumab is used to treat?
"While Ravulizumab is most commonly used to treat disease activity, it can also help patients suffering from hemolysis, thrombotic microangiopathies, and atypical hemolytic uremic syndrome." - Anonymous Online Contributor
Unverified Answer
How many study participants are being sought for this research?
"That is correct. The clinical trial is currently recruiting participants, as indicated on clinicaltrials.gov. The listing was first posted on December 2nd, 2021 and was last updated on September 27th, 2022. The trial needs to enroll 150 patients at 8 different sites." - Anonymous Online Contributor
Unverified Answer
Are people still being recruited for this trial or has it been completed?
"Yes, this study is still looking for participants. According to the information on clinicaltrials.gov, the trial was first posted on December 2nd, 2021 and was last updated on September 27th, 2022." - Anonymous Online Contributor
Unverified Answer