Spinal Muscular Atrophy Type 3Phase 2SMA3NCT03819660
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
A Phase 2 study testing Amifampridine Phosphate 10 MG Oral Tablet in people with Spinal Muscular Atrophy Type 3, run by Catalyst Pharmaceuticals, Inc. at 1 site in Italy.
Trial summary
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3. Read more about this study
- Who is it for?
- Ages 6 to 50 years
- Conditions the study is for
- Spinal Muscular Atrophy Type 3
- What is being tested?
- Amifampridine Phosphate 10 MG Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteItaly
- Study dates
- 7 Mar 2019to 13 Sep 2021
- Phase
- Phase 2
- 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 6 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Individuals eligible to participate in this study must meet all the following inclusion criteria:
- Participated in the SMA-001 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 SMA-001 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.
Who cannot join
- 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.
- Uncontrolled asthma.
- Concomitant use with sultopride.
- Concomitant use with medicinal products with a narrow therapeutic window.
- Concomitant use with medicinal products with a known to cause QTc prolongation.
Show all 13Show fewer
- Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
- Subjects with congenital QT syndromes.
- 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.
- History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
Read the detailed description
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.
- Conditions
- Spinal Muscular Atrophy Type 3
- Being tested
- Amifampridine Phosphate 10 MG Oral Tablet
- Keywords
- Type 3
- Medical subject headings
- Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Amifampridine Phosphate 10 MG Oral TabletDrug
Oral tablets
| Study group | Role | What they receive |
|---|---|---|
| amifampridine phosphate | Experimental | 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. Receives: Drug: Amifampridine Phosphate 10 MG Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long-term Safety and Tolerability of Amifampridine
Number of subjects with treatment emergent adverse events (TEAE).
Measured: 18 months
Secondary outcomes (1)
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.
Measured: Screening to end of study.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2019
- Study start7 Mar 2019
- Primary completion13 Sep 2021
- Study completion13 Sep 2021
Why it stopped: Development of indication not being pursued
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 Spinal Muscular Atrophy Type 3Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label, extension study , long term safety and tolerability
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lorenzo Maggi, MD, Principal investigator, Carlo Besta Institute, Milan, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03819660
- Sponsor's protocol ID
- SMA-002
- Acronym
- SMA3
- Status last verified
- Nov 2023
- First submitted
- 23 Jan 2019
- Last updated
- 30 Nov 2023
- 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 30 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
- ClinicalTrials.gov ID
- NCT03819660
- Acronym / sponsor ID
- SMA3 / SMA-002
- Conditions
- Spinal Muscular Atrophy Type 3
- Keywords
- Type 3
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Development of indication not being pursued
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Mar 2019 (actual)
- Primary completion
- 13 Sep 2021 (actual)
- Study completion
- 13 Sep 2021 (actual)
- First posted
- 28 Jan 2019 (actual)
- Results
- Posted 30 Nov 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label, extension study , long term safety and tolerability
| Arm | Type | Receives |
|---|---|---|
| amifampridine phosphate | Experimental | Drug: Amifampridine Phosphate 10 MG Oral TabletThe 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. |
Who may qualify
- Ages
- 6 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Individuals eligible to participate in this study must meet all the following inclusion criteria:
- Participated in the SMA-001 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 SMA-001 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.
Key exclusion criteria (13 listed)
- 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.
- Uncontrolled asthma.
- Concomitant use with sultopride.
- Concomitant use with medicinal products with a narrow therapeutic window.
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 Phosphate 10 MG Oral TabletOral tablets | Drug | amifampridine phosphate |
Primary outcome
Long-term Safety and Tolerability of Amifampridine
Time frame: 18 months
Number of subjects with treatment emergent adverse events (TEAE).
1 secondary outcome
To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL).
Time frame: Screening to end of study.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Spinal Muscular Atrophy Type 3
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 2
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Spinal Muscular Atrophy, SMA
Active, not recruitingPhase 3
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Active, not recruitingPhase 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
RecruitingObservational study
UK SMA Patient Registry
Spinal Muscular Atrophy, SMA
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.
Lorenzo Maggi, MD principal investigator
Carlo Besta Institute, Milan, Italy
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Amifampridine Phosphate 10 MG Oral Tablet in people with Spinal Muscular Atrophy Type 3, run by Catalyst Pharmaceuticals, Inc. at 1 site in Italy.
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 5 inclusion and 13 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 30 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 30 Nov 2023). 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?
Long-term Safety and Tolerability of Amifampridine (time frame: 18 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 30 Nov 2023.