A Phase 2 interventional study of Amifampridine Phosphate and Placebo Oral Tablet in Muscular Atrophy, Spinal, sponsored by Catalyst Pharmaceuticals, Inc.. Completed at 1 site in Italy. Open to participants aged 6 Years to 50 Years. Per ClinicalTrials.gov, last updated 2021-06-01.
Sponsored by Catalyst Pharmaceuticals, Inc. (part of Angelini Pharma) · Phase 2, Interventional, and Treatment
A two-period, two-treatment, crossover study to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory patients diagnosed with spinal muscular atrophy (SMA) Type 3.
This randomized (1:1), double-blind, placebo-controlled, 2-period, 2-treatment, crossover, outpatient study is designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory patients diagnosed with SMA Type 3. The study is planned to include approximately 12 male and female SMA Type 3 patients. The planned duration of participation for each patient is approximately 2 months, based upon length of dose titration and excluding the screening period, which can last up to 14 days. Patients should only be taking the assigned investigational product (amifampridine phosphate 10 mg tablets or matching placebo tablets), no new therapies are permitted during the study.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.
This study's enrollment of 13 is below the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.
Browse Muscular Atrophy, Spinal 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.
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.
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.
Oral tablets, 30 to 80 mg per day in divided doses 3 to 4 times a day for 4 weeks
Drug: Amifampridine Phosphate · Drug: Placebo Oral Tablet
Oral tablets, 30 to 80 mg per day in divided doses 3 to 4 times a day for 4 weeks
Drug: Amifampridine Phosphate · Drug: Placebo Oral Tablet
Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.
Also known as: 3,4 diaminopyridine phosphate
Placebo Oral Tablet
Hammersmith Functional Motor Scale Expanded (HFMSE) Summary Statistics and Mixed Model Analysis
Hammersmith Functional Motor Scale Expanded (HFMSE) assesses motor function by functional item in order of progressive difficulty, with higher values showing higher function abilities. Each item is scored on a scale of 0-2 with 2 representing item achieved unaided and 0 representing inability to achieve item. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period, during Period 1 at Day 7 and Day 14, and during Period 2 at Day 21 and Day 28. The total HFMSE score was calculated as the sum of each item score, with a maximum score of 66 (all items achieved unaided) and minimum score of 0 (all items failed). Change from baseline (CFB) will be assessed from Day 0 to Day 28. A mixed effects liner model was fit with the HFMSE change from baseline (CFB) scores at Day 28 as a response and treatment, sequence, and treatment by sequence as fixed effect terms and patient as a random effect.
Time frame: Screening, the first (Day 1) and last day (Day 0) of the Run-in period, during Period 1 at Day 7 and Day 14, and during Period 2 at Day 21 and Day 28
The study was conducted from 14 January 2019 - 17 September 2020 at two sites in Europe.
| Milestone | Amifampridine Phosphate - Placebo | Placebo - Amifampridine Phosphate | Not Randomized |
|---|---|---|---|
| Started | 6 | 6 | 1 |
| Completed | 6 | 6 | 0 |
| Not completed | 0 | 0 | 1 |
Hammersmith Functional Motor Scale Expanded (HFMSE) assesses motor function by functional item in order of progressive difficulty, with higher values showing higher function abilities. Each item is scored on a scale of 0-2 with 2 representing item achieved unaided and 0 representing inability to achieve item. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period, during Period 1 at Day 7 and Day 14, and during Period 2 at Day 21 and Day 28. The total HFMSE score was calculated as the sum of each item score, with a maximum score of 66 (all items achieved unaided) and minimum score of 0 (all items failed). Change from baseline (CFB) will be assessed from Day 0 to Day 28. A mixed effects liner model was fit with the HFMSE change from baseline (CFB) scores at Day 28 as a response and treatment, sequence, and treatment by sequence as fixed effect terms and patient as a random effect.
| Overall Score | Amifampridine Phosphate | Placebo |
|---|---|---|
| Hammersmith Functional Motor Scale Expanded (HFMSE) Summary Statistics and Mixed Model Analysis | 0.208 ± 0.326 | -0.583 ± 0.326 |
Collected over Serious adverse events (SAEs) were recorded for each patient from the time informed consent was obtained at Screening and continued through four weeks after the last visit or at the early discontinuation visit, for a total of approximately 14 weeks. Non-serious AEs were recorded for each patient after the first administration of study drug through the termination visit or at the early termination visit, for a total of approximately 10 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Amifampridine | 0/12 (0%) | 0/12 (0%) | 5/12 (41.7%) |
| Placebo | 0/12 (0%) | 0/12 (0%) | 3/12 (25%) |
| Event | Amifampridine | Placebo |
|---|---|---|
| Paraesthesia oralGastrointestinal disorders | 3/12 | 0/12 |
| Muscular weaknessMusculoskeletal and connective tissue disorders | 0/12 | 2/12 |
| Hypoaesthesia oralGastrointestinal disorders | 1/12 | 0/12 |
| FatigueGeneral disorders | 0/12 | 1/12 |
| Gait disturbanceGeneral disorders | 0/12 | 1/12 |
| Influenza like illnessGeneral disorders | 1/12 | 0/12 |
| InfluenzaInfections and infestations | 1/12 | 0/12 |
| FallInjury, poisoning and procedural complications | 1/12 | 1/12 |
| Transaminases increasedInvestigations | 1/12 | 0/12 |
| HeadacheNervous system disorders | 1/12 | 0/12 |
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 regardless of whether they were randomized to double blind medication on Day 0 belong to the Safety population.
| Age, Continuous(years) | Amifampridine Phosphate - Placebo | Placebo - Amifampridine Phosphate | Not Randomized | Total |
|---|---|---|---|---|
| Mean | 37.2 ± 8.5 | 30.2 ± 13.5 | 45.0 | 34.5 ± 11.3 |
| Sex: Female, Male(Participants) | Amifampridine Phosphate - Placebo | Placebo - Amifampridine Phosphate | Not Randomized | Total |
|---|---|---|---|---|
| Female | 4 | 1 | 0 | 5 |
| Male | 2 | 5 | 1 | 8 |
| Race (NIH/OMB)(Participants) | Amifampridine Phosphate - Placebo | Placebo - Amifampridine Phosphate | Not Randomized | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 0 | 1 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 | 0 |
| White | 6 | 5 | 1 | 12 |
| More than one race | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Weight(kg) | Amifampridine Phosphate - Placebo | Placebo - Amifampridine Phosphate | Not Randomized | Total |
|---|---|---|---|---|
| Mean | 64.7 ± 9.3 | 67.8 ± 11.8 | 76.9 | 67.0 ± 10.2 |
| Height(cm) | Amifampridine Phosphate - Placebo | Placebo - Amifampridine Phosphate | Not Randomized | Total |
|---|---|---|---|---|
| Mean | 169.3 ± 9.0 | 174.8 ± 6.1 | 170.0 | 171.9 ± 7.6 |
| BMI(kg/m^2) | Amifampridine Phosphate - Placebo | Placebo - Amifampridine Phosphate | Not Randomized | Total |
|---|---|---|---|---|
| Mean | 22.5 ± 1.4 | 22.3 ± 4.3 | 26.6 | 22.7 ± 3.1 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Amifampridine
Catalyst Pharmaceuticals, Inc. →