Generalized Myasthenia GravisPhase 2NCT03315130
Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis
A Phase 2 study testing zilucoplan (RA101495) in people with Generalized Myasthenia Gravis, run by Ra Pharmaceuticals at 30 sites in 2 countries.
Trial summary
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Generalized Myasthenia Gravis
- What is being tested?
- zilucoplan (RA101495)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 30 sitesin 2 countries
- Study dates
- 11 Oct 2017to 19 Nov 2020
- Phase
- Phase 2
- 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 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IVa] at Screening
- Positive serology for acetylcholine receptor (AChR) autoantibodies
- QMG score ≥ 12 at Screening and Randomization
- No change in corticosteroid dose for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
- No change in immunosuppressive therapy, including dose, for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
Who cannot join
- Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12 week Treatment Period
- History of meningococcal disease
- Current or recent systemic infection within 2 weeks prior to Randomization or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization
Trial locations and contacts
30 sites in 2 countries.
30 sites in 2 countries
United States26 sites
University of Maryland
Baltimore, Maryland, 21201
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of Buffalo
Buffalo, New York, 14260
Lahey Hospital and Medical Center
Burlington, Massachusetts, 01805
University of Vermont
Burlington, Vermont, 05405
The Research Center of Southern California
Carlsbad, California, 92011
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Rush University Medical Center
Chicago, Illinois, 60612
Ohio State University
Columbus, Ohio, 43210
Wesley Neurology Clinic
Cordova, Tennessee, 38018
University of Texas Southwestern
Dallas, Texas, 75390
Wayne State University
Detroit, Michigan, 48202
Michigan State University
East Lansing, Michigan, 48864
University of Florida
Jacksonville, Florida, 32209
University of Kansas Medical Center
Kansas City, Kansas, 66160
UCLA Medical Center
Los Angeles, California, 90095
Center for Neurological Disorders
Milwaukee, Wisconsin, 53215
Diagnostic and Medical Clinic - Mobile
Mobile, Alabama, 36604
Yale University
New Haven, Connecticut, 06520
Hospital for Special Surgery
New York, New York, 10021
Mount Sinai Hospital
New York, New York, 10029
University of California Irvine Health ALS and Neuromuscular Center
Orange, California, 92868
Allegheny Neurological Associates
Pittsburgh, Pennsylvania, 15212
University of Utah
Salt Lake City, Utah, 84132
University of South Florida
Tampa, Florida, 33620
George Washington University
Washington D.C., District of Columbia, 20052
Canada4 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7
London Health Sciences Centre University Hospital
London, Ontario, N6A 5A5
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
Toronto General Hospital
Toronto, Ontario, M5G 2C4
About this study
Official title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects With Generalized Myasthenia Gravis
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- zilucoplan (RA101495), Placebo
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
zilucoplan (RA101495)Drug
Daily subcutaneous (SC) injection
PlaceboDrug
Daily subcutaneous (SC) injection
| Study group | Role | What they receive |
|---|---|---|
| 0.1 mg/kg zilucoplan (RA101495) | Experimental | Not described Receives: Drug: zilucoplan (RA101495) |
| 0.3 mg/kg zilucoplan (RA101495) | Experimental | Not described Receives: Drug: zilucoplan (RA101495) |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for myasthenia gravis (MG). The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.
Measured: From Baseline to Week 12
Secondary outcomes (12)
Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale
The MG-ADL is a brief 8-item survey designed to evaluate MG symptom severity. The scale consists of 8 items each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the 8 individual scores with range of 0-24. Higher scores are associated with more severe symptoms of MG.
Measured: From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life 15r (MG-QOL15r) Survey
The MG-QOL15r is a 15-item survey that was designed to assess quality of life in participants with MG. The survey consisted of 15 questions and the corresponding responses were each scored on a 0-2 point scale (0=Not much at all, 1=Somewhat, 2=Very Much). The total score is the sum of the 15 individual item scores with a range of 0-30. Higher scores indicate more severe impact of the disease on aspects of the participant's life.
Measured: From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the MG Composite Scale Total Score
The MG Composite is a 10-item scale that has been used to measure the clinical status of participants with MG, in order to evaluate treatment response. It consists of 10 items which included ptosis (score range=0 to 3), double vision on lateral gaze left/right/both (score range=0 to 4), eye closure (score range=0 to 2), talking (score range=0 to 6), chewing (score range=0 to 6), swallowing (score range=0 to 6), breathing (score range=0 to 9), neck flexion or extension (score range=0 to 4), shoulder abduction (score range=0 to 5) and hip flexion (score range= 0 to 5). The total score is the sum of the 10 individual scores with a range of 0-50. Higher scores in the MG Composite indicate more severe impairment due to the disease.
Measured: From Baseline to Week 12
Main Portion: Percentage of Participants With >= 3-point Reduction in QMG Total Score at Week 12
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.
Measured: Week 12
Main Portion: Percentage of Participants Who Required Rescue Therapy Over the 12-week Treatment Period
Percentage of participants who used at least 1 dose of rescue medication were reported.
Measured: Up to Week 12
Main Portion: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A serious adverse event (SAE) is any untoward medical occurrence that at any dose resulted in death, life-threatening, significant or persistent disability/incapacity, congenital anomaly/birth defect, important medical event, initial inpatient hospitalization or prolongation of hospitalization.
Measured: From Baseline to Week 12
Main Portion: Change From Baseline in Sheep Red Blood Cell (sRBC) Lysis Assay at Week 12 (Pre-dose)
A BioTek ELx800 automated microplate reader is used to measure the optical density at 415 nanometer (nm) of human plasma samples to calculate the percent lysis of sheep erythrocytes that has occurred as a result of total complement activity. The measure of complement activity is determined by the degree of hemolysis of the erythrocytes.
Measured: Baseline and Week 12 (Pre-dose)
Main Portion: Change From Baseline in C5 Levels at Week 12 (Pre-dose)
Blood samples were collected from participants to assess Complement Component 5C levels.
Measured: Baseline and Week 12 (Pre-dose)
Main Portion: Plasma Concentration of RA101495 and Its Major Metabolites
RA102758 and RA103488 are the metabolites of RA101495.
Measured: 1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12
Main Portion: Maximum Plasma Concentration (Cmax) on Day 1
Cmax is defined as the maximum observed plasma concentration.
Measured: Pre-dose, 1, 3 and 6 hours postdose on Day 1
Main Portion: Time Corresponding to Cmax (Tmax) on Day 1
Tmax is defined as the time to observe maximum plasma concentration.
Measured: Pre-dose, 1, 3 and 6 hours postdose on Day 1
Main Portion: Metabolites (RA102758 and RA103488) to Parent Ratio
RA102758 and RA103488 are the metabolites of RA101495.
Measured: Pre-dose, 1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2017
- Study start11 Oct 2017
- Primary completion10 Dec 2018
- Study completion19 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Generalized Myasthenia GravisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ra Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273 (UCB)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03315130
- Sponsor's protocol ID
- RA101495-02.201
- Status last verified
- Jul 2022
- First submitted
- 16 Oct 2017
- Last updated
- 27 Jul 2022
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Howard JF Jr, Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, Benatar M, Duda PW, MacDougall JE, Farzaneh-Far R, Kaminski HJ; Zilucoplan MG Study Group; Barohn R, Dimachkie M, Pasnoor M, Farmakidis C, Liu T, Colgan S, Benatar MG, Bertorini T, Pillai R, Henegar R, Bromberg M, Gibson S, Janecki T, Freimer M, Elsheikh B, Matisak P, Genge A, Guidon A, David W, Habib AA, Mathew V, Mozaffar T, Hinton JL, Hewitt W, Barnett D, Sullivan P, Ho D, Howard JF Jr, Traub RE, Chopra M, Kaminski HJ, Aly R, Bayat E, Abu-Rub M, Khan S, Lange D, Holzberg S, Khatri B, Lindman E, Olapo T, Sershon LM, Lisak RP, Bernitsas E, Jia K, Malik R, Lewis-Collins TD, Nicolle M, Nowak RJ, Sharma A, Roy B, Nye J, Pulley M, Berger A, Shabbir Y, Sachdev A, Patterson K, Siddiqi Z, Sivak M, Bratton J, Small G, Kohli A, Fetter M, Vu T, Lam L, Harvey B, Wolfe GI, Silvestri N, Patrick K, Zakalik K, Duda PW, MacDougall J, Farzaneh-Far R, Pontius A, Hoarty M. Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial. JAMA Neurol. 2020 May 1;77(5):582-592. doi: 10.1001/jamaneurol.2019.5125.
PubMed 32065623
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects With Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT03315130
- Acronym / sponsor ID
- RA101495-02.201
- Conditions
- Generalized Myasthenia Gravis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Ra PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 11 Oct 2017 (actual)
- Primary completion
- 10 Dec 2018 (actual)
- Study completion
- 19 Nov 2020 (actual)
- First posted
- 19 Oct 2017 (actual)
- Results
- Posted 27 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 0.1 mg/kg zilucoplan (RA101495) | Experimental | Drug: zilucoplan (RA101495) |
| 0.3 mg/kg zilucoplan (RA101495) | Experimental | Drug: zilucoplan (RA101495) |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IVa] at Screening
- Positive serology for acetylcholine receptor (AChR) autoantibodies
- QMG score ≥ 12 at Screening and Randomization
- No change in corticosteroid dose for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
- No change in immunosuppressive therapy, including dose, for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
Key exclusion criteria (3 listed)
- Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12 week Treatment Period
- History of meningococcal disease
- Current or recent systemic infection within 2 weeks prior to Randomization or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| zilucoplan (RA101495)Daily subcutaneous (SC) injection | Drug | 0.1 mg/kg zilucoplan (RA101495), 0.3 mg/kg zilucoplan (RA101495) |
| PlaceboDaily subcutaneous (SC) injection | Drug | Placebo |
Primary outcome
Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score
Time frame: From Baseline to Week 12
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for myasthenia gravis (MG). The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.
12 secondary outcomes
Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale
Time frame: From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life 15r (MG-QOL15r) Survey
Time frame: From Baseline to Week 12
Main Portion: Change From Baseline to Week 12 in the MG Composite Scale Total Score
Time frame: From Baseline to Week 12
Main Portion: Percentage of Participants With >= 3-point Reduction in QMG Total Score at Week 12
Time frame: Week 12
Main Portion: Percentage of Participants Who Required Rescue Therapy Over the 12-week Treatment Period
Time frame: Up to Week 12
Main Portion: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Baseline to Week 12
Main Portion: Change From Baseline in Sheep Red Blood Cell (sRBC) Lysis Assay at Week 12 (Pre-dose)
Time frame: Baseline and Week 12 (Pre-dose)
Main Portion: Change From Baseline in C5 Levels at Week 12 (Pre-dose)
Time frame: Baseline and Week 12 (Pre-dose)
Main Portion: Plasma Concentration of RA101495 and Its Major Metabolites
Time frame: 1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12
Main Portion: Maximum Plasma Concentration (Cmax) on Day 1
Time frame: Pre-dose, 1, 3 and 6 hours postdose on Day 1
Main Portion: Time Corresponding to Cmax (Tmax) on Day 1
Time frame: Pre-dose, 1, 3 and 6 hours postdose on Day 1
Main Portion: Metabolites (RA102758 and RA103488) to Parent Ratio
Time frame: Pre-dose, 1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 32065623Howard JF Jr, Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, Benatar M, Duda PW, MacDougall JE, Farzaneh-Far R, Kaminski HJ; Zilucoplan MG Study Group; Barohn R, Dimachkie M, Pasnoor M, Farmakidis C, Liu T, Colgan S, Benatar MG, Bertorini T, Pillai R, Henegar R, Bromberg M, Gibson S, Janecki T, Freimer M, Elsheikh B, Matisak P, Genge A, Guidon A, David W, Habib AA, Mathew V, Mozaffar T, Hinton JL, Hewitt W, Barnett D, Sullivan P, Ho D, Howard JF Jr, Traub RE, Chopra M, Kaminski HJ, Aly R, Bayat E, Abu-Rub M, Khan S, Lange D, Holzberg S, Khatri B, Lindman E, Olapo T, Sershon LM, Lisak RP, Bernitsas E, Jia K, Malik R, Lewis-Collins TD, Nicolle M, Nowak RJ, Sharma A, Roy B, Nye J, Pulley M, Berger A, Shabbir Y, Sachdev A, Patterson K, Siddiqi Z, Sivak M, Bratton J, Small G, Kohli A, Fetter M, Vu T, Lam L, Harvey B, Wolfe GI, Silvestri N, Patrick K, Zakalik K, Duda PW, MacDougall J, Farzaneh-Far R, Pontius A, Hoarty M. Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial. JAMA Neurol. 2020 May 1;77(5):582-592. doi: 10.1001/jamaneurol.2019.5125.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
001 844 599 2273 (UCB)
30 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing zilucoplan (RA101495) in people with Generalized Myasthenia Gravis, run by Ra Pharmaceuticals at 30 sites in 2 countries.
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 5 inclusion and 3 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: finished, as of the sponsor's last update on 27 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 27 Jun 2022). 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?
Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score (time frame: From Baseline to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03315130, which also lists 1 linked publication.
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 27 Jul 2022.