|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
11 December 2023 |
|
Main ID: |
NCT03819660 |
|
Date of registration:
|
23/01/2019 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
SMA3 |
|
Scientific title:
|
Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 |
|
Date of first enrolment:
|
March 7, 2019 |
|
Target sample size:
|
13 |
|
Recruitment status: |
Terminated |
|
URL:
|
https://clinicaltrials.gov/ct2/show/NCT03819660 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
Italy
| | | | | | | |
|
Contacts
|
|
Name:
|
Lorenzo Maggi, MD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Carlo Besta Institute, Milan, Italy |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
Individuals eligible to participate in this study must meet all the following inclusion
criteria:
1. Participated in the SMA-001 study
2. 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.
3. 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.
4. 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.
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:
1. Epilepsy and currently on medication.
2. Uncontrolled asthma.
3. Concomitant use with sultopride.
4. Concomitant use with medicinal products with a narrow therapeutic window.
5. Concomitant use with medicinal products with a known to cause QTc prolongation.
6. Clinically significant abnormalities in 12 lead ECG, in the opinion of the
Investigator.
7. Subjects with congenital QT syndromes.
8. Breastfeeding or pregnant at Screening or planning to become pregnant at any time
during the study.
9. Intolerable amifampridine-related side effects
10. Treatment with an investigational drug (other than amifampridine) or device while
participating in this study.
11. 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.
12. History of drug allergy to any pyridine-containing substances or any amifampridine
excipient(s).
Age minimum:
6 Years
Age maximum:
50 Years
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Spinal Muscular Atrophy Type 3
|
|
Intervention(s)
|
|
Drug: Amifampridine Phosphate 10 MG Oral Tablet
|
|
Primary Outcome(s)
|
|
Long-term Safety and Tolerability of Amifampridine
[Time Frame: 18 months]
|
|
Secondary Outcome(s)
|
|
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.]
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|