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TerminatedNCT04025632Updated Jul 27, 2022Results posted

Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy

A Phase 2 interventional study of zilucoplan and Placebo in Immune Mediated Necrotizing Myopathy, sponsored by Ra Pharmaceuticals. Terminated at 18 sites in 4 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2022-07-27.

Sponsored by Ra Pharmaceuticals · Phase 2, Interventional, and Treatment

Why this study was terminated
The study was terminated based on the primary efficacy endpoint analysis after the first data lock.
Phase
Phase 2
Study type
Interventional
Enrollment
27
Allocation
Randomized
Ages
18 Years to 75 Years
Sex
All
01

Study summary

The purpose of the study is to evaluate the safety and efficacy of zilucoplan in patients with Immune-Mediated Necrotizing Myopathy (IMNM). Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or matching placebo for 8 weeks.

02

Conditions studied

  • Immune Mediated Necrotizing Myopathy

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03

In context

Muscular Diseases

280 studies on the registry are indexed under Muscular Diseases; 63 are open to participants now.

This study's enrollment of 27 is below the median of 34 across 157 interventional studies indexed under Muscular Diseases.

Browse Muscular Diseases studies →

Lead sponsor

Ra Pharmaceuticals is the lead sponsor of 4 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Clinical diagnosis of IMNM (Immune-Mediated Necrotizing Myopathy)
  • Positive serology for anti-3-hydroxy-3-methyl-glutaryl-coenzyme A reductase (HMGCR) or anti-signal recognition particle (SRP) autoantibodies
  • Clinical evidence of weakness (≤ grade 4 out of 5) on manual muscle testing in at least one proximal limb muscle group
  • Creatine kinase (CK) of >1000 U/L at Screening
  • No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study
  • No changes in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the first 8-weeks on study

Exclusion criteria

Exclusion Criteria:

  • History of meningococcal disease
  • Current or recent systemic infection within 2 weeks prior to Screening or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Screening
  • Recent initiation of intravenous immunoglobulin (IVIG) (i.e., first cycle administered less than 90 days prior to Baseline)
  • Rituximab use within 90 days prior to Baseline or anticipated to occur during study
  • Statin use within 30 days prior to Baseline or anticipated to occur during study
  • Plasma exchange within 4 weeks prior to Baseline or expected to occur during the 8-week Treatment Period
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
27 participants (actual)

Study arms

  • Experimental
    0.3 mg/kg zilucoplan

    Daily subcutaneous (SC) injection

    Drug: zilucoplan

  • Placebo comparator
    Placebo

    Daily subcutaneous (SC) injection

    Other: Placebo

Interventions

  • Drugzilucoplan

    Daily subcutaneous (SC) inection

  • OtherPlacebo

    Daily subcutaneous (SC) inection

06

What researchers measure

Primary outcomes

  1. Percentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels

    All laboratory samples were obtained prior to administration of study drug at applicable visits. CK levels were measured by a central laboratory.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

  2. Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)

    A TEAE was defined as: * An adverse event (AE) that occurred after study treatment start that was not present at the time of treatment start. * An AE that increased in severity after treatment start if the event was present at the time of treatment start.

    Time frame: Baseline (Day 1) to end of Main Portion (Week 8)

Secondary outcomes

  1. Number of Participants Who Achieve at Least Minimal Response Based on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria Scale

    The ACR/EULAR scale utilized a conjoint analysis-based continuous model using absolute percent change from Baseline in core set measures (physician, patient, and Myositis Disease Activity Assessment Tool (MDAAT); muscle strength; Health Assessment Questionnaire (HAQ); and muscle enzyme levels). A total improvement score (range 0-100) was determined by summing scores for each core set measure and comparing improvement in each respective core set measure. The threshold for minimal improvement was ≥20 in the total improvement score with higher scores indicating a better outcome.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

  2. Change From Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time

    The 3TUG test involved the ambulatory participant getting up from a seated position in a chair, walking at their normal pace for 3 meters, turning around, walking back to the chair, and sitting down. This sequence was repeated 3 times without rest, and the 3TUG test time is the average of the 3 lap times. A negative change from baseline indicated a better outcome.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

  3. Change From Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Score

    The proximal MMT assessed muscle strength using manual muscle testing in 7 muscle groups (left and right sides assessed separately). The total MMT score for this study, inclusive of both sides, could range from 0-140, where 0 means no strength in any muscles and 140 means full strength in all the muscles examined. A negative change from Baseline indicated a worse outcome.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

  4. Change From Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score

    The Physician Global Activity VAS Score measured the treating physician's global evaluation of the participant's overall disease activity using a 10 cm VAS labelled with "no activity" at the left end and "maximum activity" at the right end. The Physician Global Activity VAS Score ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

  5. Change From Baseline to Week 8 in Patient Global Activity VAS Score

    The Patient Global Activity VAS Score measured the treating participant's global evaluation of their overall disease activity using a 10 cm VAS labelled with "no activity" at the left end and "maximum activity" at the right end. The Patient Global Activity VAS score ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

  6. Change From Baseline to Week 8 in HAQ Score

    The HAQ had 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities with 2 to 3 questions for each section. Scoring within each section ranged from 0 (without any difficulty) to 3 (unable to do). The total HAQ score was then calculated by summing the scores and dividing by the number of categories answered. The total HAQ score for this study could range from 0-3, where 0 means no functional impairment and 3 means complete functional impairment. A negative change from Baseline indicated a better outcome.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

  7. Change From Baseline to Week 8 in MDAAT Extramuscular Disease Activity VAS Score

    The MDAAT extramuscular disease activity VAS score measured the degree of disease activity of extramuscular organ systems and muscle. The scoring was performed by the physician and ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

  8. Change From Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

    The FACIT-Fatigue Scale is a 13-item tool which measured an individual's level of fatigue during their usual daily activities over the past week. The level of fatigue was measured on a 4-point Likert scale. The total FACIT-Fatigue Scale score for this study could range from 0-52, where 0 means the participants were very much fatigued during their usual daily activities and 52 means the participants were not at all fatigued during their usual daily activities. A negative change from Baseline indicated a worse outcome.

    Time frame: Baseline (Day 1) and end of Main Portion (Week 8)

07

Results

Posted May 16, 2022
Limitations and caveats
The study was terminated based on the primary efficacy endpoint analysis after the first data lock.

Participant flow

This study was performed in 4 countries (France, the Netherlands, the United Kingdom, and the United States of America) between 07 November 2019 and 14 June 2021.

Participant flow — Overall Study
MilestonePlaceboZilucoplan 0.3 mg/kg
Started1512
Completed main portion1512
Started main portion safety follow-up20
Completed main portion safety follow-up00
Started extension portion1312
Completed00
Not completed1512
Withdrew: Adverse event11
Withdrew: Withdrawal by subject11
Withdrew: Study terminated by sponsor109
Withdrew: Physician decision31

Outcome measures

PrimaryPercentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels

All laboratory samples were obtained prior to administration of study drug at applicable visits. CK levels were measured by a central laboratory.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Mean · percentage change
Percentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels
percentage changePlaceboZilucoplan 0.3 mg/kg
Percentage Change From Baseline to Week 8 in Serum Creatine Kinase (CK) Levels-20.72 ± 31.22-9.86 ± 26.06
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · 2-sided Van Elteren test · p = 0.464 (Conducted using a 2-sided Van Elteren test, which represents an extension of the Wilcoxon rank sum test for comparing 2 treatments in a stratified experiment using within-stratum ranks assigning greater weight to rank sums from smaller strata.) · Wilcoxon-mann-whitney odds: 0.55 · 95% CI 0.19 to 1.57The magnitude of association between treatment groups was expressed as in Wilcoxon-Mann-Whitney odds followed by the 95% confidence intervals.
PrimaryNumber of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)

A TEAE was defined as: * An adverse event (AE) that occurred after study treatment start that was not present at the time of treatment start. * An AE that increased in severity after treatment start if the event was present at the time of treatment start.

Time frame:
Baseline (Day 1) to end of Main Portion (Week 8)
Reported as:
Count of participants · Participants
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)
ParticipantsMain Portion: PlaceboMain Portion: Zilucoplan 0.3 mg/kg
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)139
SecondaryNumber of Participants Who Achieve at Least Minimal Response Based on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria Scale

The ACR/EULAR scale utilized a conjoint analysis-based continuous model using absolute percent change from Baseline in core set measures (physician, patient, and Myositis Disease Activity Assessment Tool (MDAAT); muscle strength; Health Assessment Questionnaire (HAQ); and muscle enzyme levels). A total improvement score (range 0-100) was determined by summing scores for each core set measure and comparing improvement in each respective core set measure. The threshold for minimal improvement was ≥20 in the total improvement score with higher scores indicating a better outcome.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Count of participants · Participants
Number of Participants Who Achieve at Least Minimal Response Based on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria Scale
ParticipantsPlaceboZilucoplan 0.3 mg/kg
Number of Participants Who Achieve at Least Minimal Response Based on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Response Criteria Scale76
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Regression, Logistic · p = 0.919 (P-value for the comparison of treatment groups was calculated using logistic regression with investigational medicinal product and strata as fixed factors.) · Odds ratio (or): 1.088 · 95% CI 0.214 to 5.535
SecondaryChange From Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time

The 3TUG test involved the ambulatory participant getting up from a seated position in a chair, walking at their normal pace for 3 meters, turning around, walking back to the chair, and sitting down. This sequence was repeated 3 times without rest, and the 3TUG test time is the average of the 3 lap times. A negative change from baseline indicated a better outcome.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Least squares mean · seconds
Change From Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time
secondsPlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 8 in Triple Timed Up and Go Test (3TUG) Time-0.712 ± 0.789-1.401 ± 0.788
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Linear Mixed Effect Model · p = 0.496 (Based on a linear model with treatment and strata (anti-3-hydroxy-3-methyl-glutaryl-coenzyme A reductase \[HMGCR\]+/anti-signal recognition particle \[SRP\]+) as fixed factors with Baseline 3TUG as a covariate.) · Least squares (ls) mean difference: -0.688 · 95% CI -2.781 to 1.404The difference presented is zilucoplan 0.3 mg/kg minus placebo.
SecondaryChange From Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Score

The proximal MMT assessed muscle strength using manual muscle testing in 7 muscle groups (left and right sides assessed separately). The total MMT score for this study, inclusive of both sides, could range from 0-140, where 0 means no strength in any muscles and 140 means full strength in all the muscles examined. A negative change from Baseline indicated a worse outcome.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 8 in Proximal Manual Muscle Testing (MMT) Score-0.18 ± 3.443.71 ± 3.81
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Linear Mixed Effect Model · p = 0.431 (Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline proximal MMT as a covariate.) · Ls mean difference: 3.89 · 95% CI -6.18 to 13.95The difference presented is zilucoplan 0.3 mg/kg minus placebo.
SecondaryChange From Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score

The Physician Global Activity VAS Score measured the treating physician's global evaluation of the participant's overall disease activity using a 10 cm VAS labelled with "no activity" at the left end and "maximum activity" at the right end. The Physician Global Activity VAS Score ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 8 in Physician Global Activity Visual Analogue Scale (VAS) Score-0.626 ± 0.557-0.830 ± 0.671
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Linear Mixed Effect Model · p = 0.800 (Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline Physician Global Activity VAS as a covariate.) · Ls mean difference: -0.204 · 95% CI -1.855 to 1.448The difference presented is zilucoplan 0.3 mg/kg minus placebo.
SecondaryChange From Baseline to Week 8 in Patient Global Activity VAS Score

The Patient Global Activity VAS Score measured the treating participant's global evaluation of their overall disease activity using a 10 cm VAS labelled with "no activity" at the left end and "maximum activity" at the right end. The Patient Global Activity VAS score ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 8 in Patient Global Activity VAS Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 8 in Patient Global Activity VAS Score-0.685 ± 0.707-1.966 ± 0.854
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Linear Mixed Effect Model · p = 0.221 (Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline Patient Global Activity VAS as a covariate.) · Ls mean difference: -1.281 · 95% CI -3.390 to 0.829The difference presented is zilucoplan 0.3 mg/kg minus placebo.
SecondaryChange From Baseline to Week 8 in HAQ Score

The HAQ had 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities with 2 to 3 questions for each section. Scoring within each section ranged from 0 (without any difficulty) to 3 (unable to do). The total HAQ score was then calculated by summing the scores and dividing by the number of categories answered. The total HAQ score for this study could range from 0-3, where 0 means no functional impairment and 3 means complete functional impairment. A negative change from Baseline indicated a better outcome.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 8 in HAQ Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 8 in HAQ Score0.022 ± 0.151-0.125 ± 0.183
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Linear Mixed Effect Model · p = 0.508 (Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline HAQ as a covariate.) · Ls mean difference: -0.147 · 95% CI -0.601 to 0.307The difference presented is zilucoplan 0.3 mg/kg minus placebo.
SecondaryChange From Baseline to Week 8 in MDAAT Extramuscular Disease Activity VAS Score

The MDAAT extramuscular disease activity VAS score measured the degree of disease activity of extramuscular organ systems and muscle. The scoring was performed by the physician and ranged from 0 (absent extramuscular disease activity) to 10 (maximum extramuscular disease activity). A negative change from Baseline indicated a better outcome.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 8 in MDAAT Extramuscular Disease Activity VAS Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 8 in MDAAT Extramuscular Disease Activity VAS Score-0.144 ± 0.336-0.287 ± 0.398
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Linear Mixed Effect Model · p = 0.765 · Ls mean difference: -0.143 · 95% CI -1.123 to 0.837The difference presented is zilucoplan 0.3 mg/kg minus placebo.
SecondaryChange From Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

The FACIT-Fatigue Scale is a 13-item tool which measured an individual's level of fatigue during their usual daily activities over the past week. The level of fatigue was measured on a 4-point Likert scale. The total FACIT-Fatigue Scale score for this study could range from 0-52, where 0 means the participants were very much fatigued during their usual daily activities and 52 means the participants were not at all fatigued during their usual daily activities. A negative change from Baseline indicated a worse outcome.

Time frame:
Baseline (Day 1) and end of Main Portion (Week 8)
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score
score on a scalePlaceboZilucoplan 0.3 mg/kg
Change From Baseline to Week 8 in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score3.45 ± 3.418.98 ± 4.08
Statistical analysis
  • Placebo vs Zilucoplan 0.3 mg/kg · Linear Mixed Effect Model · p = 0.265 (Based on a linear model with treatment and strata (anti-HMGCR+/anti-SRP+) as fixed factors with Baseline FACIT-Fatigue Scale as a covariate.) · Ls mean difference: 5.53 · 95% CI -4.49 to 15.55The difference presented is zilucoplan 0.3 mg/kg minus placebo.

Adverse events

Collected over Baseline (Day 1) to End of Safety Follow-up (Week 83). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Main Portion: Placebo0/15 (0%)3/15 (20%)13/15 (86.7%)
Main Portion: Zilucoplan 0.3 mg/kg0/12 (0%)0/12 (0%)9/12 (75%)
Extension Portion: Zilucoplan 0.3 mg/kg1/25 (4%)8/25 (32%)15/25 (60%)
Most frequent serious events
Showing 10 of 11
Most frequent serious events
EventMain Portion: PlaceboMain Portion: Zilucoplan 0.3 mg/kgExtension Portion: Zilucoplan 0.3 mg/kg
COVID-19Infections and infestations0/150/123/25
Ventricular tachycardiaCardiac disorders1/150/120/25
Rhinovirus infectionInfections and infestations1/150/120/25
Urinary tract infectionInfections and infestations1/150/120/25
Liver function test increasedInvestigations1/150/120/25
HypoxiaRespiratory, thoracic and mediastinal disorders1/150/120/25
Rectal haemorrhageGastrointestinal disorders0/150/121/25
AstheniaGeneral disorders0/150/121/25
Sinusitis bacterialInfections and infestations0/150/121/25
Staphylococcal sepsisInfections and infestations0/150/121/25
Most frequent other events
Showing 10 of 49
Most frequent other events
EventMain Portion: PlaceboMain Portion: Zilucoplan 0.3 mg/kgExtension Portion: Zilucoplan 0.3 mg/kg
HeadacheNervous system disorders4/154/122/25
NauseaGastrointestinal disorders3/153/120/25
VertigoEar and labyrinth disorders2/150/121/25
FallInjury, poisoning and procedural complications0/150/123/25
AnaemiaBlood and lymphatic system disorders0/151/122/25
PalpitationsCardiac disorders0/151/120/25
Haemorrhoidal haemorrhageGastrointestinal disorders0/151/120/25
Injection site painGeneral disorders1/151/121/25
Injection site pruritusGeneral disorders0/151/121/25
Injection site erythemaGeneral disorders0/151/120/25

Baseline characteristics

Intent-to-Treat (ITT) Population included all randomized participants.

Age, Continuous
Age, Continuous(years)PlaceboZilucoplan 0.3 mg/kgTotal
Mean52.8 ± 13.656.9 ± 9.054.6 ± 11.8
Sex: Female, Male
Sex: Female, Male(Participants)PlaceboZilucoplan 0.3 mg/kgTotal
Female7613
Male8614
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)PlaceboZilucoplan 0.3 mg/kgTotal
Hispanic or Latino459
Not Hispanic or Latino8513
Unknown or Not Reported325
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)PlaceboZilucoplan 0.3 mg/kgTotal
American Indian/Alaska native000
Asian000
Black or African American134
Native Hawaiian or other Pacific Islander000
White10717
Other/Mixed000
Unknown or Not Reported101
Missing325
08

Study locations

18 sites
  • University of California Los Angeles
    Los Angeles, California 90095, United States
  • University of California Irvine
    Orange, California 92868, United States
  • University of Florida Health Jacksonville
    Jacksonville, Florida 32209, United States
  • University of South Florida
    Tampa, Florida 33612, United States
  • University of Kansas Medical Center
    Kansas City, Kansas 66160, United States
  • National Institute of Health
    Bethesda, Maryland 20892, United States
  • Brigham and Women's Hospital
    Boston, Massachusetts 02115, United States
  • Washington University School of Medicine in Saint Louis
    Saint Louis, Missouri 63110, United States
  • Northwell Health Neuroscience Institute
    Great Neck, New York 11021, United States
  • The Ohio State University
    Columbus, Ohio 43221, United States
  • University of Pennsylvania
    Philadelphia, Pennsylvania 19104, United States
  • Wesley Neurology Clinic
    Memphis, Tennessee 38018, United States
  • Austin Neuromuscular Center
    Austin, Texas 78756, United States
  • The University of Texas Health Science Center at Houston
    Houston, Texas 77030, United States
  • Hôpital Universitaire Pitié Salpêtrière
    Paris, 75013, France
  • Amsterdam UMC
    Amsterdam, 1105 AZ, Netherlands
  • University College London Hospitals NHS Foundation Trust
    London, WC1N3BG, United Kingdom
  • Salford Royal NHS Barnes Clinical Research Facility
    Manchester, M6 8HD, United Kingdom
09

References and documents

Study documents

  • Study protocol · Feb 16, 2021
  • Statistical analysis plan · Jun 9, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

Supporting information: Study protocol, Sap, Csr

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 27, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04025632
Lead sponsor
Ra Pharmaceuticals
Responsible party
Sponsor
First posted
Jul 19, 2019
Start date
Nov 7, 2019
Primary completion
Mar 4, 2021
Completion
Jun 14, 2021
Results posted
May 16, 2022
Last update
Jul 27, 2022

Study contacts

UCB Cares
study director · UCB Pharma

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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