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L'essai clinique NCT01377922 pour Lambert Eaton Myasthenic Syndrome est terminé. Consultez la vue en carte du Radar des Essais Cliniques et les outils de découverte par IA pour tous les détails, ou posez vos questions ici. | ||
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A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS) Phase III 38
Les détails de l'essai clinique sont principalement disponibles en anglais. Cependant, l'IA Trial Radar peut vous aider ! Cliquez simplement sur 'Expliquer l'étude' pour voir et discuter des informations sur l'étude dans la langue sélectionnée.
L'essai clinique NCT01377922 était conçu pour étudier le traitement de Lambert Eaton Myasthenic Syndrome. Il s'agissait d'une étude interventionnel en Phase III. Son statut actuel est : terminé. L'étude a commencé le 1 juin 2011, avec un objectif de recruter 38 participants. Dirigée par Catalyst Pharmaceuticals, l'étude s'est terminée le 1 juillet 2016. Les données du site ClinicalTrials.gov ont été mises à jour pour la dernière fois le 4 janvier 2018.
Résumé succinct
A Phase 3 study to evaluate the efficacy and safety of Amifampridine Phosphate in patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
Description détaillée
This multicenter, double-blind, placebo-controlled, randomized (1:1) discontinuation study is a 4-part study designed to evaluate the efficacy and safety of multiple dose administration of amifampridine phosphate in patients with LEMS. Data from parts 2 and 3 (the double-blind parts of the study) are presented in this record.
Titre officiel
A Phase 3, Double-blind, Placebo-controlled, Randomized Discontinuation Study Followed by Open-label Extension Evaluating Efficacy and Safety of Amifampridine Phosphate in Patients With Lambert-Eaton Myasthenic Syndrome (LEMS)
Pathologies
Lambert Eaton Myasthenic SyndromeAutres identifiants de l'étude
- LMS-002
Numéro NCT
Date de début (réel)
2011-06
Dernière mise à jour publiée
2018-01-04
Date de fin (estimée)
2016-07
Inscription (estimée)
38
Type d'étude
Interventionnel
PHASE
Phase III
Statut
Terminé
Objectif principal
Traitement
Méthode d'allocation
Randomisé
Modèle d'intervention
Parallèle
Masquage
Double aveugle
Bras / Interventions
| Groupe de participants/Bras | Intervention/Traitement |
|---|---|
Comparateur placeboPlacebo Matching placebo tablets administered 3-4 times a day (to the individual patient's tablet count of active at baseline) over 2 weeks. | PLACEBO Matching placebo tablets administered 3-4 times a day (to the individual patient's tablet count of active at baseline) over 2 weeks. |
ExpérimentalAmifampridine Phosphate Amifampridine, 30-80 mg given 3-4 times per day with a maximum single dose of 20 mg (2 x 10 mg tablets), for 2 weeks. | Amifampridine Phosphate Amifampridine, 30-80 mg given 3-4 times per day with a maximum single dose of 20 mg (2 x 10 mg tablets), for 2 weeks. |
Critère principal d'évaluation
Critère secondaire d'évaluation
| Critères d'évaluation | Description de la mesure | Période |
|---|---|---|
Change From Baseline Quantitative Myasthenia Gravis (QMG) at 14 Days | The QMG is a physician-rated test including 13 assessments, including facial strength, swallowing, grip strength, and duration of time that limbs can be maintained in outstretched positions. Each of the 13 items is scored from 0 (none) to 3 (severe). The total score can range from 0 to 39. Increased QMG total score correlates to worsening symptoms of LEMS. | Assessment at Baseline and Day 14 |
Change in SGI Score | Subject Global Impression (SGI) is a measure of changes in subject's perception of change in overall wellbeing.
The patient is asked to use the 7-point scale below to rate their impression of the effects of the study medication during the preceding 3 days on their physical well being.
1. Terrible
2. Mostly dissatisfied
3. Mixed
4. Partially satisfied
5. Mostly satisfied
6. Pleased
7. Delighted | Assessment at Baseline and Day 14 |
| Critères d'évaluation | Description de la mesure | Période |
|---|---|---|
Change From Baseline Timed 25 Foot Walking Test (T25FW) at 14 Days | The T25FW test, a component of the Multiple Sclerosis Functional Composite, was a quantitative mobility and leg function performance test based on a timed 25-foot walk (National Multiple Sclerosis Society). The patient was directed to walk a clearly marked 25-foot course as quickly and safely as possible. Following a rest of at least 5 minutes, the timed 25-foot walk was repeated. Patients could use assistive devices, such as canes, crutches, or walkers.
All data were normalized to the number of feet per minute, so if the patient walked 25 feet in less than a minute, the result was a speed greater than 25 feet/minute.
The measurement for the T25FW test was the average speed, expressed in feet/minute, of the 2 completed walks. | Assessment at Baseline and Day 14 |
Change in CGI-I Score | The Investigator completed the 7-point CGI I, based on changes in symptoms, behavior, and functional abilities, at the protocol-specified time points compared to the patient's condition at Day 0.
1. = Very much improved
2. = Much improved
3. = Minimally improved
4. = No change
5. = Minimally worse
6. = Much worse
7. = Very much worse | Baseline and Day 14 |
Critères d'éligibilité
Âges éligibles
Adulte, Adulte âgé
Âge minimum
18 Years
Sexes éligibles
Tous
Individuals eligible to participate in this study must meet all of the following inclusion criteria:
- ≥18 years of age
- Confirmed diagnosis of LEMS
- Normal respiratory function
- Normal swallowing function
- If receiving peripherally acting cholinesterase inhibitors a stable dose is required for at least 7 days prior to Screening.
- If receiving oral immunosuppressants a stable dose is required for at least 90 days prior to Screening.
- Negative pregnancy test for females of childbearing potential
- If sexually active, willing to use 2 acceptable methods of contraception
- Willing to perform all study procedures as physically possible.
- Willing and able to provide written informed consent after the nature of the study has been explained and prior to the start of any research-related procedures.
Individuals who meet any of the following exclusion criteria are not eligible to participate in the study:
- History of epilepsy or seizure.
- Known active brain metastasis.
- Use of Fampridine (4-aminopyridine), and any form of 3,4-diaminopyridine other than the IP provided, such as amifampridine base or Firdapse, during the study.
- Use of medications known to lower the epileptic threshold within 7 days or 5 half-lives.
- Use of medications which inhibit neuromuscular junction function within 7 days or 5 half-lives.
- Use of IVIG, plasmapheresis (plasma exchange), or immunoadsorption within 90 days
- Use of guanidine hydrochloride within 7 days
- Use of rituximab within 12 months
- History of drug allergy to any pyridine-containing substances or any amifampridine phosphate excipient(s).
- Use of any other investigational productwithin 30 days
- Treatment with a concomitant medication that prolongs the QT/QTc interval within 7 days or 5 half-lives.
- Treatment with sultopride (4-amino-N-\[(1-ethylpyrrolidin-2-yl)methyl\]-5-ethylsulfonyl-2-methoxybenzamide) within 7 days.
- An abnormal electrocardiogram (ECG).
- Documented history of arrhythmias.
- History of additional risk factors for torsade de pointes.
- Breastfeeding or pregnant or planning to become pregnant (self or partner) at any time during the study.
- Likely or expected to require treatment for cancer within 3 months (90 days) after entering.
- History of severe renal impairment or evidence of severe renal impairment
- Any condition that places the patient at high risk of poor treatment compliance or of not completing the study.
- History of uncontrolled asthma.
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14 Centres de l'étude dans 8 pays
Lyon, 69677, France
Alabama
Birmingham, Alabama, 35233, United States
Arizona
Scottsdale, Arizona, 85258, United States
California
Los Angeles, California, 90095, United States
Palo Alto, California, 94305, United States
Kansas
Kansas City, Kansas, 66160, United States
New York
New York, New York, 10032, United States
Bavaria
Munich, Bavaria, D-80336, Germany
Berlin, D-10117, Germany
Pécs, H-7623, Hungary
Warsaw, 02 097, Poland
Moscow, 125367, Russia
Belgrade, 11000, Serbia
Madrid, 28007, Spain
Catalyst Pharmaceuticals