Myasthenia Gravis, MuSKPhase 3NCT03579966
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
A Phase 3 study testing Amifampridine Phosphate in people with Myasthenia Gravis, MuSK and AChR Myasthenia Gravis, run by Catalyst Pharmaceuticals, Inc. at 3 sites in 2 countries.
Trial summary
Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis, MuSK, AChR Myasthenia Gravis
- What is being tested?
- Amifampridine Phosphate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 63 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 11 Jun 2018to 5 Aug 2022
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participated in the MSK-002 study
- Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures.
- Female patients of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin [HCG] at the end of MSK-002 study); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment.
- Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires.
- Individuals who met any of the exclusion criteria in the original protocol or those listed below are not eligible to participate in the study:
- Epilepsy and currently on medication.
Show all 11Show fewer
- Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
- Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
- Intolerable amifampridine-related side effects
- Treatment with an investigational drug (other than amifampridine) or device while participating in this study.
- Any medical condition that, in the opinion of the Investigator, might interfere with the patient's participation in the study, poses an added risk for the patient, or confound the assessment of the patient.
Who cannot join
None listed.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
Cleveland Clinic
Cleveland, Ohio, 44195
Univerity of Pennsylvania
Philadelphia, Pennsylvania, 19104
Italy1 site
Department of Neuroimmunology and Neuromuscular Diseases Carlo Besta Neurological Institute Via Celoria
Milan, 11-201332
About this study
Official title: Long Term Safety Study of Amifampridine Phosphate in Patients With MuSK Antibody Positive and AChR (Acetylcholine Receptor) Antibody Positive Myasthenia Gravis
Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.
Read the detailed description
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.
- Conditions
- Myasthenia Gravis, MuSK, AChR Myasthenia Gravis
- Being tested
- Amifampridine Phosphate
- Keywords
- Amifampridine Phosphate Amifampridine, 3,4-Diaminopyridine Phosphate, 3,4-Diaminopyridine, 3,4-DAP, Firdapse®, Myasthenia Gravis, MuSK, AChR
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Amifampridine PhosphateDrug
tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day
| Study group | Role | What they receive |
|---|---|---|
| amifampridine phosphate | Experimental | 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 Receives: Drug: Amifampridine Phosphate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: over 39 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2018
- Study start11 Jun 2018
- Primary completion5 Aug 2022
- Study completion5 Aug 2022
Why it stopped: Lack of measure appropriate data (MG-ADL).
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.
- Other trials for Myasthenia Gravis, MuSKStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Renato Mantegazza, MD, Principal investigator, Carlo Besta Neurological Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03579966
- Sponsor's protocol ID
- MSK-003
- Status last verified
- Apr 2024
- First submitted
- 5 Jun 2018
- Last updated
- 7 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Safety Study of Amifampridine Phosphate in Patients With MuSK Antibody Positive and AChR (Acetylcholine Receptor) Antibody Positive Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT03579966
- Acronym / sponsor ID
- MSK-003
- Conditions
- Myasthenia Gravis, MuSK, AChR Myasthenia Gravis
- Keywords
- Amifampridine Phosphate Amifampridine, 3,4-Diaminopyridine Phosphate, 3,4-Diaminopyridine, 3,4-DAP, Firdapse®, Myasthenia Gravis, MuSK, AChR
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of measure appropriate data (MG-ADL).
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Jun 2018 (actual)
- Primary completion
- 5 Aug 2022 (actual)
- Study completion
- 5 Aug 2022 (actual)
- First posted
- 9 Jul 2018 (actual)
- Results
- Posted 7 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| amifampridine phosphate | Experimental | Drug: Amifampridine Phosphatetablets 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Participated in the MSK-002 study
- Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures.
- Female patients of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin [HCG] at the end of MSK-002 study); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment.
- Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires.
- Individuals who met any of the exclusion criteria in the original protocol or those listed below are not eligible to participate in the study:
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Amifampridine Phosphatetablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day | Drug | amifampridine phosphate |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)
Time frame: over 39 months
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Renato Mantegazza, MD principal investigator
Carlo Besta Neurological Institute
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Amifampridine Phosphate in people with Myasthenia Gravis, MuSK and AChR Myasthenia Gravis, run by Catalyst Pharmaceuticals, Inc. at 3 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 11 inclusion and 0 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: stopped early, as of the sponsor's last update on 7 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Italy. 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 7 May 2024). 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?
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability) (time frame: over 39 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 May 2024.