A Phase 2 interventional study of Amifampridine Phosphate 10 MG Oral Tablet in Spinal Muscular Atrophy Type 3, sponsored by Catalyst Pharmaceuticals, Inc.. Terminated at 1 site in Italy. Open to participants aged 6 Years to 50 Years. Per ClinicalTrials.gov, last updated 2023-11-30.
Sponsored by Catalyst Pharmaceuticals, Inc. (part of Angelini Pharma) · Phase 2, Interventional, and Treatment
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
This open-label outpatient extension study was designed to evaluate the long-term safety and tolerability of amifampridine in patients diagnosed with SMA Type 3. In addition, the evaluation of the effects of amifampridine on the QoL was performed. The study was planned to enroll those patients who had completed the SMA-001 study and after all final evaluations for that study had been completed, or those who demonstrated benefit after completing the dose titration period but failed to meet the randomization criteria on Day 0 of SMA-001.
The duration of participation for each patient was expected to be at least 12 months as patients could continue in the study until amifampridine was approved by Regulatory Agencies or the clinical development of amifampridine was terminated for this indication. In addition to amifampridine, patients continued to receive previous concomitant medications, as needed.
61 studies on the registry are indexed under Spinal Muscular Atrophies of Childhood; 18 are open to participants now.
This study's enrollment of 13 is below the median of 15 across 41 interventional studies indexed under Spinal Muscular Atrophies of Childhood.
Browse Spinal Muscular Atrophies of Childhood 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.
Individuals eligible to participate in this study must meet all the following inclusion criteria:
Exclusion 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.
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.
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.
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.
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.
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.
The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily.
Drug: Amifampridine Phosphate 10 MG Oral Tablet
Oral tablets
Long-term Safety and Tolerability of Amifampridine
Number of subjects with treatment emergent adverse events (TEAE).
Time frame: 18 months
To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL).
Quality of life (QoL): the Individualized Quality of Life for neuromuscular disease (INQoL) or the Pediatric Quality of Life (PEDSQLTM) was used for adult or pediatric patients, respectively. Since there were no pediatric patients, none of the patients completed the PEDSQL. The INQol evaluated weakness, pain, fatigue, double vision, muscle locking, droopy eyelids, swallowing difficulties, activities, social relationships, emotions and body image. Each of these areas were measured in four categories as follows: 1- Incidence (0= No, 1 =Yes), 2-Severity (0= None to 7 = extreme), 3- Impact - (0= None to 6 = extreme) and 4-Importance (0= None to 6 = extreme). The numbers were summative and are input to the QOL calculation, which is a percentage of severity on a scale of 0-100. The mean value was taken across this population. The higher scores were a worse outcome.
Time frame: Screening to end of study.
| Milestone | Amifampridine Phosphate |
|---|---|
| Started | 13 |
| Completed | 13 |
| Not completed | 0 |
Number of subjects with treatment emergent adverse events (TEAE).
| Participants | Amifampridine Phosphate |
|---|---|
| Long-term Safety and Tolerability of Amifampridine | 5 |
Quality of life (QoL): the Individualized Quality of Life for neuromuscular disease (INQoL) or the Pediatric Quality of Life (PEDSQLTM) was used for adult or pediatric patients, respectively. Since there were no pediatric patients, none of the patients completed the PEDSQL. The INQol evaluated weakness, pain, fatigue, double vision, muscle locking, droopy eyelids, swallowing difficulties, activities, social relationships, emotions and body image. Each of these areas were measured in four categories as follows: 1- Incidence (0= No, 1 =Yes), 2-Severity (0= None to 7 = extreme), 3- Impact - (0= None to 6 = extreme) and 4-Importance (0= None to 6 = extreme). The numbers were summative and are input to the QOL calculation, which is a percentage of severity on a scale of 0-100. The mean value was taken across this population. The higher scores were a worse outcome.
| score on a scale | Amifampridine Phosphate |
|---|---|
| screening | 33.737 ± 17.677 |
| End of Study | 39.012 ± 14.337 |
Collected over 27 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Amifampridine Phosphate | 0/13 (0%) | 1/13 (7.7%) | 5/13 (38.5%) |
| Event | Amifampridine Phosphate |
|---|---|
| Intellectual DisabilityNervous system disorders | 1/13 |
| Event | Amifampridine Phosphate |
|---|---|
| Paraesthesia oralGastrointestinal disorders | 3/13 |
| ABDOMINAL PAIN UPPERGastrointestinal disorders | 2/13 |
| HYPERPYREXIAGeneral disorders | 2/13 |
| FEELING HOTGeneral disorders | 1/13 |
| INFLUENZA LIKE ILLNESSGeneral disorders | 1/13 |
| MALAISEGeneral disorders | 1/13 |
| FALLInjury, poisoning and procedural complications | 1/13 |
| POST LUMBAR PUNCTURE SYNDROMEGeneral disorders | 1/13 |
| HYPOKALAEMIAMetabolism and nutrition disorders | 1/13 |
| ParaesthesiaNervous system disorders | 1/13 |
| Age, Categorical(Participants) | Amifampridine Phosphate |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 13 |
| >=65 years | 0 |
| Age, Continuous(years) | Amifampridine Phosphate |
|---|---|
| Mean | 34.8 ± 11.3 |
| Sex: Female, Male(Participants) | Amifampridine Phosphate |
|---|---|
| Female | 5 |
| Male | 8 |
| Race (NIH/OMB)(Participants) | Amifampridine Phosphate |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 12 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | Amifampridine Phosphate |
|---|---|
| Italy | 6 |
| Serbia | 7 |
| Height(CM) | Amifampridine Phosphate |
|---|---|
| Mean | 171.9 (163 to 187) |
| Weight(Kg) | Amifampridine Phosphate |
|---|---|
| Mean | 67.1 (53 to 86) |
| BMI(kg/m^2) | Amifampridine Phosphate |
|---|---|
| Mean | 22.7 (16.7 to 28.4) |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Amifampridine
Spinal Muscular Atrophies of Childhood →
Catalyst Pharmaceuticals, Inc. →