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Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 (SMA3)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03819660
Recruitment Status : Enrolling by invitation
First Posted : January 28, 2019
Last Update Posted : March 29, 2019
Sponsor:
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

Brief Summary:
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.

Condition or disease Intervention/treatment Phase
Muscle Atrophy Drug: Amifampridine Phosphate 10 MG Oral Tablet Phase 2

Detailed Description:
This open-label, long term safety study is designed to evaluate the safety of amifampridine phosphate in ambulatory patients diagnosed SMA Type 3 over an extended period. The study is planned to include approximately 12 male and female SMA Type 3 patients. The planned duration of participation for each patient may be up to 1 year. Patients must have demonstrated benefit during the initial SMA-001 trial.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Open-label, long term safety treatment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
Actual Study Start Date : March 7, 2019
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : April 1, 2021


Arm Intervention/treatment
Experimental: amifampridine phosphate
Oral tablets, 15 to 80 mg per day in divided doses 3 to 4 times a day for up to 18 months.
Drug: Amifampridine Phosphate 10 MG Oral Tablet
Oral tablets




Primary Outcome Measures :
  1. The number and severity of treatment related adverse events and serious adverse events as assessed by CTCAE v4.0 [ Time Frame: 18 months ]

    Safety analyses will be conducted on the safety population (i.e. all patients who receive at least 1 dose of amifampridine. The safety analysis will be descriptive and will be presented on observed data only.

    All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of TEAEs will be summarized by system organ class, preferred term, relationship to treatment, and severity.




Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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:

CRITERIA FOR INCLUSION AND EXCLUSION:

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03819660


Locations
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Italy
Neurological Institute Carlo Besta
Milano, Lombardy, Italy, 20133
Sponsors and Collaborators
Catalyst Pharmaceuticals, Inc.
Investigators
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Principal Investigator: Lorenzo Maggi, MD Carlo Besta Institute, Milan, Italy

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Responsible Party: Catalyst Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03819660     History of Changes
Other Study ID Numbers: SMA-002
First Posted: January 28, 2019    Key Record Dates
Last Update Posted: March 29, 2019
Last Verified: March 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Catalyst Pharmaceuticals, Inc.:
Type 3

Additional relevant MeSH terms:
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Atrophy
Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Amifampridine
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action