A Phase 3 interventional study of Amifampridine Phosphate and Placebo Oral Tablet in Myasthenia Gravis, Generalized, sponsored by Catalyst Pharmaceuticals, Inc.. Completed at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-03-12.
Sponsored by Catalyst Pharmaceuticals, Inc. (part of Angelini Pharma) · Phase 3, Interventional, and Treatment
Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.
Randomized, double-blind, placebo-controlled, parallel group study is designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in patients with MuSK-MG. In addition, a sample of AChR-MG patients will be assess for efficacy and safety of amifampridine phosphate. Planned duration of participation for each patient is at least 38 days, excluding the screening period. Eligible patients will be titrated to an efficacious dose of amifampridine phosphate and those who demonstrate improvement will be randomized to either placebo or amifampridine, in a double-blind fashion, for 10 days.
330 studies on the registry are indexed under Myasthenia Gravis; 144 are open to participants now.
This study's enrollment of 93 is above the median of 44 across 217 interventional studies indexed under Myasthenia Gravis.
Browse Myasthenia Gravis studies →Catalyst Pharmaceuticals, Inc. is the lead sponsor of 13 studies on the registry; 1 is open to participants now.
First submitted before Catalyst Pharmaceuticals, Inc. became part of Angelini Pharma.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Amifampridine Phosphate
Drug: Placebo Oral Tablet
tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day
tablets matching amifampridine phosphate, 3 to 4 times a day
Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results
Myasthenia Gravis-Activities of Daily Living (MG-ADL) is a self-report scale to assess the patient's MG symptoms and functional performance of activities of daily living. The eight items are scored on a scale of 0-3 with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. Each item was assessed by the patient at the last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total MG-ADL score was calculated as the sum of each item score, with a maximum score of 24 (most severe symptoms/impairment) and minimum score of 0 (least severe symptoms/impairment). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnos
Time frame: Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early).
Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results
Quantitative Myasthenia Gravis (QMG) assesses the patient's general body strength and fatigability. Each test item is scored on a scale of 0-3 with 3 representing the most severe symptom results and 0 representing no symptom results. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total QMG score was calculated as the sum of each item score, with a maximum score of 39 (most severe symptoms) and minimum score of 0 (least severe symptoms). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.
Time frame: Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time at which a patient discontinued treatment early).
The study was conducted from 18 April 2018 - 24 April 2020 at 26 sites in the United States and Europe.
| Milestone | Amifampridine Phosphate - MuSK | Amifampridine Phosphate - AChR-MG | Placebo - MuSk | Placebo - AChR-MG | Amifampridine Phosphate Only - MuSK | Amifampridine Phosphate Only- AChR-MG |
|---|---|---|---|---|---|---|
| Started | 27 | 7 | 28 | 8 | 17 | 6 |
| Completed | 27 | 7 | 27 | 8 | 0 | 0 |
| Not completed | 0 | 0 | 1 | 0 | 17 | 6 |
Myasthenia Gravis-Activities of Daily Living (MG-ADL) is a self-report scale to assess the patient's MG symptoms and functional performance of activities of daily living. The eight items are scored on a scale of 0-3 with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. Each item was assessed by the patient at the last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total MG-ADL score was calculated as the sum of each item score, with a maximum score of 24 (most severe symptoms/impairment) and minimum score of 0 (least severe symptoms/impairment). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnos
| Score on a Scale | Amifampridine Phosphate - MuSK | Placebo - MuSK | Amifampridine Phosphate - AChR | Placebo - AChR |
|---|---|---|---|---|
| Baseline (Day 0) MG-ADL Total Raw Score | 4.96 ± 2.915 | 3.86 ± 2.103 | 6.14 ± 3.388 | 7.00 ± 4.175 |
| Post-Baseline(Day 10 or time at which a patient discontinued treatment) MG-ADL Total Raw Score | 6.00 ± 3.658 | 6.11 ± 3.19 | 4.71 ± 3.147 | 10.38 ± 3.583 |
| CFB MG-ADL Total Score | 1.04 ± 2.980 | 2.25 ± 3.658 | -1.43 ± 2.225 | 3.38 ± 2.825 |
Quantitative Myasthenia Gravis (QMG) assesses the patient's general body strength and fatigability. Each test item is scored on a scale of 0-3 with 3 representing the most severe symptom results and 0 representing no symptom results. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total QMG score was calculated as the sum of each item score, with a maximum score of 39 (most severe symptoms) and minimum score of 0 (least severe symptoms). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.
| Score on a Scale | Amifampridine Phosphate - MuSK | Placebo - MuSK | Amifampridine Phosphate - AChR | Placebo - AChR |
|---|---|---|---|---|
| Baseline (Day 0) QMG Total Raw Score | 10.00 ± 3.873 | 8.64 ± 3.744 | 10.57 ± 3.359 | 14.13 ± 4.155 |
| Post-Baseline (day 10 or time at which a patient discontinued treatment early) QMG Total Raw Score | 11.19 ± 5.367 | 10.16 ± 3.648 | 10.86 ± 3.024 | 14.43 ± 3.952 |
| CFB QMG Total Score | 1.19 ± 3.99 | 1.80 ± 3.948 | 0.29 ± 3.039 | 0.86 ± 2.673 |
Collected over Serious adverse events (SAEs) were recorded for each patient from the time informed consent was obtained at Screening and continues through four weeks after the last dose or at the early discontinuation visit, for a total of approximately 52 days. Non-serious AEs were recorded for each patient after the first administration of study drug through the last visit or at the early discontinuation visit, for a total of approximately 10 days.. Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Amifampridine Phosphate | 0/86 (0%) | 2/86 (2.3%) | 80/86 (93%) |
| Placebo | 0/36 (0%) | 0/36 (0%) | 7/36 (19.4%) |
| Overall | 0/86 (0%) | 2/86 (2.3%) | 80/86 (93%) |
| Event | Amifampridine Phosphate | Placebo | Overall |
|---|---|---|---|
| Myasthenia gravisNervous system disorders | 1/86 | 0/36 | 1/86 |
| Myasthenia gravis crisisNervous system disorders | 1/86 | 0/36 | 1/86 |
| Event | Amifampridine Phosphate | Placebo | Overall |
|---|---|---|---|
| Paraesthesia oralGastrointestinal disorders | 37/86 | 0/36 | 37/86 |
| ParaesthesiaNervous system disorders | 34/86 | 0/36 | 34/86 |
| NauseaGastrointestinal disorders | 14/86 | 0/36 | 14/86 |
| DiarrhoeaGastrointestinal disorders | 12/86 | 0/36 | 12/86 |
| FatigueGeneral disorders | 10/86 | 2/36 | 12/86 |
| HeadacheNervous system disorders | 11/86 | 0/36 | 11/86 |
| Hypoaesthesia oralGastrointestinal disorders | 9/86 | 0/36 | 9/86 |
| DizzinessNervous system disorders | 8/86 | 0/36 | 8/86 |
| Abdominal pain upperGastrointestinal disorders | 7/86 | 0/36 | 7/86 |
| DyspepsiaGastrointestinal disorders | 7/86 | 0/36 | 7/86 |
The analysis of baseline characteristics included all patients in the Safety population, who are those patients who enrolled and received at least one dose of amifampridine. Patients who began the run-in period belong to the Safety population whether they were randomized to treatment or not.
| Age, Continuous(years) | Amifampridine Phosphate | Placebo | Amifampridine Phosphate Only | Total |
|---|---|---|---|---|
| Mean | 53.1 ± 14.92 | 53.5 ± 12.61 | 45.9 ± 11.31 | 51.9 ± 13.52 |
| Sex: Female, Male(Participants) | Amifampridine Phosphate | Placebo | Amifampridine Phosphate Only | Total |
|---|---|---|---|---|
| Female | 24 | 28 | 14 | 66 |
| Male | 10 | 8 | 2 | 20 |
| Ethnicity (NIH/OMB)(Participants) | Amifampridine Phosphate | Placebo | Amifampridine Phosphate Only | Total |
|---|---|---|---|---|
| Hispanic or Latino | 1 | 2 | 15 | 18 |
| Not Hispanic or Latino | 33 | 34 | 1 | 68 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Amifampridine Phosphate | Placebo | Amifampridine Phosphate Only | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 1 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 3 | 2 | 2 | 7 |
| White | 31 | 34 | 13 | 78 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Weight(kg) | Amifampridine Phosphate | Placebo | Amifampridine Phosphate Only | Total |
|---|---|---|---|---|
| Mean | 84.6 ± 21.11 | 84.3 ± 27.27 | 76.1 ± 32.28 | 82.9 ± 25.96 |
| Height(cm) | Amifampridine Phosphate | Placebo | Amifampridine Phosphate Only | Total |
|---|---|---|---|---|
| Mean | 164.7 ± 7.64 | 163.5 ± 10.75 | 163.6 ± 7.37 | 164.0 ± 8.99 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
This study is completed, as verified in Jul 2021. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Amifampridine
Catalyst Pharmaceuticals, Inc. →