A Phase 3 interventional study of Amifampridine Phosphate in Myasthenia Gravis, MuSK and AChR Myasthenia Gravis, sponsored by Catalyst Pharmaceuticals, Inc.. Terminated at 3 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-05-07.
Sponsored by Catalyst Pharmaceuticals, Inc. (part of Angelini Pharma) · Phase 3, Interventional, and Treatment
Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.
The Primary objective :
To characterize the long-term safety and tolerability of amifampridine phosphate in patients with MG
The Secondary Objective:
To assess the clinical efficacy of amifampridine phosphate over time in patients with MG based on change in Myasthenia Gravis Activities of Daily Living Score (MG-ADL)
This was a long-term extension study for subjects who participated in Protocol MSK-002 where the efficacy and safety of amifampridine was evaluated in subjects diagnosed with MuSK MG or AChR-MG. The optimal dose and schedule for amifampridine from the end of the Run-in Period from Study MSK-002 was initially used for each patient. The Investigator could adjust the dose of amifampridine during the course of the trial, in order to optimize neuromuscular benefit for the patient. Clinic visits for safety assessment and for evaluation of MG-ADL were made at Months 3, 6, 9, 12, 15, 21, 27, 33 and 39. Additional visits could occur at the discretion of the Investigator.
The Original protocol (12 Nov 2017) included analysis of MG-ADL change over time, however, Statistical Analysis Plan (SAP), dated 21 Feb 2023, removed the analysis of MG-ADL score change over time.
330 studies on the registry are indexed under Myasthenia Gravis; 144 are open to participants now.
This study's enrollment of 63 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.
Inclusion Criteria
Individuals who met any of the exclusion criteria in the original protocol or those listed below are not eligible to participate in the study:
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.
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.
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 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.
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.
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.
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.
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.
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.
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.
tablets equivalent to 10mg amifampridine, 3 to 4 times per day. Initially, the daily amifampridine doses were to be the doses that were determined at the end of the Run-in Period from the MSK-002 study. The usual range was from 30 to 80 mg total daily dose, given in three (3) or four (4) doses, with no single dose \> 20 mg
Drug: Amifampridine Phosphate
tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)
Evaluate the long-term safety and tolerability of amifampridine phosphate through the number of patients with treatment-emergent adverse events mapped to the Medical Dictionary for Regulatory Activities (MedDRA) SOCs and PTs. \[ Time Frame: over 39 months \] Descriptive statistics will be used to summarize study data.
Time frame: over 39 months
extension study to MSK-002
| Milestone | Amifampridine Phosphate |
|---|---|
| Started | 63 |
| Completed | 3 |
| Not completed | 60 |
| Withdrew: Adverse event | 6 |
| Withdrew: Lost to follow-up | 4 |
| Withdrew: Physician decision | 10 |
| Withdrew: Pregnancy | 1 |
| Withdrew: Withdrawal by subject | 16 |
| Withdrew: Study terminated by sponsor | 22 |
| Withdrew: Protocol violation | 1 |
Evaluate the long-term safety and tolerability of amifampridine phosphate through the number of patients with treatment-emergent adverse events mapped to the Medical Dictionary for Regulatory Activities (MedDRA) SOCs and PTs. \[ Time Frame: over 39 months \] Descriptive statistics will be used to summarize study data.
| Participants | Treatment Emergent Adverse Events |
|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) | 58 |
Collected over Study Duration 39 months. Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Serious Adverse Event | 0/63 (0%) | 14/63 (22.2%) | 58/63 (92.1%) |
| Event | Serious Adverse Event |
|---|---|
| Myasthenia GravisNervous system disorders | 4/63 |
| corona virus infectionInfections and infestations | 3/63 |
| PneumoniaInfections and infestations | 3/63 |
| Aortic Valve StenosisCardiac disorders | 1/63 |
| DysphagiaGastrointestinal disorders | 1/63 |
| small intestinal obstructionGastrointestinal disorders | 1/63 |
| vomitingGastrointestinal disorders | 1/63 |
| Staphylococcal infectionInfections and infestations | 1/63 |
| cervical spine stenosisMusculoskeletal and connective tissue disorders | 1/63 |
| Bone NeoplasmNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/63 |
| Event | Serious Adverse Event |
|---|---|
| Paresthesia OralGastrointestinal disorders | 15/63 |
| ParaesthesiaNervous system disorders | 14/63 |
| HeadacheNervous system disorders | 7/63 |
| FatigueGeneral disorders | 6/63 |
| Urinary Tract InfectionInfections and infestations | 6/63 |
| Myasthenia GravisNervous system disorders | 6/63 |
| Abdominal Pain UpperGastrointestinal disorders | 5/63 |
| CataractEar and labyrinth disorders | 4/63 |
| Hypoasthesia OralGastrointestinal disorders | 4/63 |
| NauseaGastrointestinal disorders | 4/63 |
| Age, Categorical(Participants) | Amifampridine Phosphate |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 53 |
| >=65 years | 10 |
| Age, Continuous(years) | Amifampridine Phosphate |
|---|---|
| Mean | 53 ± 14.08 |
| Sex: Female, Male(Participants) | Amifampridine Phosphate |
|---|---|
| Female | 47 |
| Male | 16 |
| Race (NIH/OMB)(Participants) | Amifampridine Phosphate |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 4 |
| White | 59 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Amifampridine Phosphate |
|---|---|
| United States | 36 |
| Italy | 27 |
| Weight(Kg) | Amifampridine Phosphate |
|---|---|
| Mean | 85.9 ± 24.6 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
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Studies of the drug tested here: Amifampridine
Catalyst Pharmaceuticals, Inc. →