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A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)

7 maggio 2019 aggiornato da: Cytokinetics

A Phase II, Double-Blind, Randomized, Placebo-Controlled, Three-Way Crossover, Pharmacokinetic and Pharmacodynamic Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)

The primary objective of this study is to demonstrate a pharmacodynamic effect of CK 2017357 on measures of skeletal muscle function or fatigability in patients with ALS.

Panoramica dello studio

Stato

Completato

Condizioni

Intervento / Trattamento

Descrizione dettagliata

This study is a Phase II, double-blind, randomized, placebo-controlled, three-way crossover study of CK-2017357 in patients with ALS. 36 to 72 patients will be randomized to one of six different treatment sequences. Each treatment sequence consists of three dosing periods; in each dosing period¸ patients receive a single oral dose of placebo, 250 mg of CK-2017357, or 500 mg of CK-2017357. All six treatment sequences will enroll approximately the same number of patients. A washout period of at least 6 days (to a maximum of 10 days) will be employed between the doses for each patient. This study is designed to assess the effect of CK-2017357 on maximal voluntary muscle strength, on the development of fatigue at maximal and sub-maximal voluntary muscle contraction, and on selected pulmonary function parameters. The plasma concentration of CK-2017357 will be measured at selected time points after each of two single doses of CK-2017357 in men and women. The plasma concentration versus time data obtained in this study may be used to develop a population PK model and estimate inter-subject variability of PK parameters in this target patient population, in particular between male and female study patients.

Tipo di studio

Interventistico

Iscrizione (Effettivo)

67

Fase

  • Fase 2

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

  • Stati Uniti
    • Arizona
      • Phoenix, Arizona, Stati Uniti, 85018
        • Phoenix Neurological Associates, Ltd.
    • California
      • Fresno, California, Stati Uniti, 93701
        • University Neurology Associates
      • San Francisco, California, Stati Uniti, 94115
        • California Pacific Medical Center
    • Florida
      • Jacksonville, Florida, Stati Uniti, 32224
        • Mayo Clinic Florida
    • Kentucky
      • Lexington, Kentucky, Stati Uniti, 40536
        • University Of Kentucky
    • Maryland
      • Baltimore, Maryland, Stati Uniti, 21287
        • Johns Hopkins Hospital
    • Massachusetts
      • Boston, Massachusetts, Stati Uniti, 02114
        • Massachusetts General Hospital
    • Missouri
      • Saint Louis, Missouri, Stati Uniti, 63110
        • Washington University
    • New York
      • Syracuse, New York, Stati Uniti, 13210
        • SUNY Upstate Medical Center
    • North Carolina
      • Durham, North Carolina, Stati Uniti, 27705
        • Duke University
    • Oregon
      • Portland, Oregon, Stati Uniti, 97213
        • Providence ALS Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, Stati Uniti, 19102
        • Drexel University College of Medicine, Dept of Neurology
      • University Park, Pennsylvania, Stati Uniti, 17033
        • Penn State
    • Texas
      • San Antonio, Texas, Stati Uniti, 78229
        • The University of Texas Health Science Center at San Antonio
    • Vermont
      • Burlington, Vermont, Stati Uniti, 05401
        • University of Vermont

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

18 anni e precedenti (Adulto, Adulto più anziano)

Accetta volontari sani

No

Sessi ammissibili allo studio

Tutto

Descrizione

Inclusion Criteria

For enrollment, patients were required to satisfy all of the following criteria at baseline:

1. Able to comprehend and willing to sign an Informed Consent Form (ICF)

  1. A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria) (Brooks, Miller et al. 2000)
  2. Males or females 18 years of age or older
  3. Body Mass Index (BMI) of 18.0 to 30.0 kg/m2, inclusive
  4. Maximum voluntary grip strength in at least one hand between 10 and 40 pounds (females) or 10 and 60 pounds (males)
  5. Able to swallow capsules with water
  6. Upright Slow Vital Capacity (SVC) > 40% of predicted for age, height, and sex [See Appendix 16.6.1]
  7. Able to perform pulmonary function tests
  8. Pre-study clinical laboratory findings (including troponin I [TnI] and creatine phosphokinase [CPK]) within normal range, or, if outside of the normal range, deemed not clinically significant by the Investigator
  9. For female patients only: The patient is post-menopausal (≥ 1 year) or sterilized, or if she is of childbearing potential, she is not breastfeeding, her pregnancy test is negative, she has no intention to become pregnant during the course of the study, and she is using contraceptive drugs or devices for the duration of the study and for 10 weeks after the end of the study.

For male patients only: Male patients agree for the duration of the study and 10 weeks after the end of the study to use a condom during sexual intercourse with female partners who are of reproductive potential and to have female partners use an additional effective means of contraception (e.g., diaphragm plus spermicide or oral contraceptives) or the male patient must agree to abstain from sexual intercourse for 10 weeks after the end of the study.

Exclusion Criteria

Patients satisfying any of the following criteria at baseline were excluded from enrollment:

  1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 times the upper limit of normal (ULN)
  2. Life expectancy < 3 months
  3. Participation in any trial in which receipt of investigational study drug occurred within 30 days prior to dosing
  4. Any prior treatment with CK-2017357
  5. In the opinion of the Investigator, the patient is not suitable to participate in the study

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Randomizzato
  • Modello interventistico: Assegnazione incrociata
  • Mascheramento: Quadruplicare

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: Treatment Sequence 1
Treatment sequence 1 consisted of three dosing periods in which patients received single oral doses of placebo, 250 mg, and 500 mg of CK-2017357, in that order, with approximately one week between each dose. Each patient acted as their own control, as all doses were represented in each treatment sequence.
Droga: Placebo
Placebo corrispondente in capsule somministrate come singola dose orale.
Droga: 250mg CK-2017357
250 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Droga: 500mg CK-2017357
500 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Sperimentale: Treatment Sequence 2
Treatment sequence 2 consisted of three dosing periods in which patients received single oral doses of placebo, 500 mg, and 250 mg of CK-2017357, in that order, with approximately one week between each dose. Each patient acted as their own control, as all doses were represented in each treatment sequence.
Droga: Placebo
Placebo corrispondente in capsule somministrate come singola dose orale.
Droga: 250mg CK-2017357
250 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Droga: 500mg CK-2017357
500 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Sperimentale: Treatment Sequence 3
Treatment sequence 3 consisted of three dosing periods in which patients received single oral doses of 250 mg, placebo and 500 mg of CK-2017357, in that order, with approximately one week between each dose. Each patient acted as their own control, as all doses were represented in each treatment sequence.
Droga: Placebo
Placebo corrispondente in capsule somministrate come singola dose orale.
Droga: 250mg CK-2017357
250 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Droga: 500mg CK-2017357
500 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Sperimentale: Treatment Sequence 4
Treatment sequence 4 consisted of three dosing periods in which patients received single oral doses of 250 mg, 500 mg and placebo of CK-2017357, in that order, with approximately one week between each dose. Each patient acted as their own control, as all doses were represented in each treatment sequence.
Droga: Placebo
Placebo corrispondente in capsule somministrate come singola dose orale.
Droga: 250mg CK-2017357
250 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Droga: 500mg CK-2017357
500 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Sperimentale: Treatment Sequence 5
Treatment sequence 5 consisted of three dosing periods in which patients received single oral doses of 500 mg, placebo, and 250 mg of CK-2017357, in that order, with approximately one week between each dose. Each patient acted as their own control, as all doses were represented in each treatment sequence.
Droga: Placebo
Placebo corrispondente in capsule somministrate come singola dose orale.
Droga: 250mg CK-2017357
250 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Droga: 500mg CK-2017357
500 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Sperimentale: Treatment Sequence 6
Treatment sequence6 consisted of three dosing periods in which patients received single oral doses of 500 mg, 250 mg, and placebo of CK-2017357, in that order, with approximately one week between each dose. Each patient acted as their own control, as all doses were represented in each treatment sequence.
Droga: Placebo
Placebo corrispondente in capsule somministrate come singola dose orale.
Droga: 250mg CK-2017357
250 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv
Droga: 500mg CK-2017357
500 mg di CK-2017357 in capsule somministrate come singola dose orale.
Altri nomi:
  • tirasemtiv

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
ALSFRS-R
Lasso di tempo: 2 days
An instrument for evaluating the functional status of patients with ALS. Minimum score is 0 and maximum score is 40. The higher the score the more function is retained.
2 days
Maximum grip strength
Lasso di tempo: 2 days
Measured using the DynEx Electronic Hand Dynamometer. Patients asked to squeeze the device with the maximum possible force to establish the maximum voluntary contraction.
2 days
Maximum grip strength fatigability
Lasso di tempo: 2 days
Handgrip fatigue is measured using the DynEx Electronic Hand Dynamometer. Patient is asked to squeeze the device until they can no longer stay above 60% of target or 120 seconds.
2 days
Shoulder extension fatigue
Lasso di tempo: 2 days
Patient is asked to hold one arm outstretched in front of them at a 90 degree angle. The time the arm falls below 90 degrees for > 2 seconds will be recorded, up to a total evaluation time of 2 minutes. This is then repeated with the other arm.
2 days
Slow Vital Capacity (SVC)
Lasso di tempo: 2 days
SVC is measured using the Puritan Bennett Renaissance II Spirometry System and accessories.
2 days
Maximum Voluntary Ventilation (MVV)
Lasso di tempo: 2 days
MVV is the volume of air that can be exhaled during 12 seconds of rapid deep breathing. The actual volume is extrapolated to one minute. the Puritan Bennett Renaissance II Spirometry System and accessories is used for this measurement.
2 days
Sniff Inspiratory Pressure (SNIP)
Lasso di tempo: 2 days
SNIP is measured at Functional Residual Capacity, the bottom of the tidal breathing cycle, through one plugged nostril while the other remains open using the Micro Medical MicroRPM Respiratory Pressure Meter
2 days
Maximum Voluntary Muscle Contraction (MVC)
Lasso di tempo: 2 days
MVC is measured using the MicroFET 2 HHD.
2 days
Repeated Sub-Maximum Grip Strength Fatigability
Lasso di tempo: 2 days
Sub-Maximum Grip Strength Fatigability is measured using the DynEx Electronic Hand. Dynamometer
2 days

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Numero di pazienti con eventi avversi
Lasso di tempo: 4 settimane
4 settimane
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and ALSFRS-R.
Lasso di tempo: 2 days
ALSFRS-R assessments will be paired with PK concentrations obtained at or near the same time as the ALSFRS-R assessments and analyzed for concentration related effects.
2 days
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum grip strength
Lasso di tempo: 2 days
Maximum grip strength assessments will be paired with PK concentrations obtained at or near the same time as the maximum grip strength assessments and analyzed for concentration related effects.
2 days
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum grip strength fatigability
Lasso di tempo: 2 days
Maximum grip strength fatigability assessments will be paired with PK concentrations obtained at or near the same time as the maximum grip strength fatigability assessments and analyzed for concentration related effects.
2 days
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and shoulder extension fatigue
Lasso di tempo: 2 days
Shoulder extension fatigue assessments will be paired with PK concentrations obtained at or near the same time as the shoulder extension fatigue assessments and analyzed for concentration related effects.
2 days
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and slow vital capacity
Lasso di tempo: 2 days
Slow vital capacity assessments will be paired with PK concentrations obtained at or near the same time as the slow vital capacity assessments and analyzed for concentration related effects.
2 days
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum voluntary ventilation
Lasso di tempo: 2 days
Maximum voluntary ventilation assessments will be paired with PK concentrations obtained at or near the same time as the maximum voluntary ventilation assessments and analyzed for concentration related effects.
2 days
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and sniff inspiratory pressure
Lasso di tempo: 2 days
Sniff inspiratory pressure assessments will be paired with PK concentrations obtained at or near the same time as the sniff inspiratory pressure assessments and analyzed for concentration related effects.
2 days
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum voluntary muscle contraction
Lasso di tempo: 2 days
Maximum voluntary muscle contraction assessments will be paired with PK concentrations obtained at or near the same time as the maximum voluntary muscle contraction assessments and analyzed for concentration related effects.
2 days
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and repeated sub-maximum grip strength fatigability
Lasso di tempo: 2 days
Repeated sub-maximum grip strength fatigability assessments will be paired with PK concentrations obtained at or near the same time as the repeated sub-maximum grip strength fatigability assessments and analyzed for concentration related effects.
2 days
Effect of CK-2017357 on patient determined global functional assessment
Lasso di tempo: 2 days
Patients will be asked to assess whether they feel the same, better or worse as compared to how they felt pre-dose
2 days
Effect of CK-2017357 on investigator determined global functional assessment
Lasso di tempo: 2 days
Investigator will assess whether they the patient appears the same, better or worse as compared to the patient's status at pre-dose
2 days

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Cytokinetics

Investigatori

  • Cattedra di studio: Jeremy M Shefner, MD, PhD, State University of New York - Upstate Medical University

Pubblicazioni e link utili

La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.

Pubblicazioni generali

Collegamenti utili

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio

1 marzo 2010

Completamento primario (Effettivo)

1 novembre 2010

Completamento dello studio (Effettivo)

1 novembre 2010

Date di iscrizione allo studio

Primo inviato

16 marzo 2010

Primo inviato che soddisfa i criteri di controllo qualità

17 marzo 2010

Primo Inserito (Stima)

18 marzo 2010

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

10 maggio 2019

Ultimo aggiornamento inviato che soddisfa i criteri QC

7 maggio 2019

Ultimo verificato

1 maggio 2019

Maggiori informazioni

Termini relativi a questo studio

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • CY 4021

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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