- ICH GCP
- Registr klinických studií v USA
- Klinická studie NCT01089010
A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
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)
Přehled studie
Postavení
Podmínky
Intervence / Léčba
Detailní popis
Typ studie
Zápis (Aktuální)
Fáze
- Fáze 2
Kontakty a umístění
Studijní místa
-
-
Arizona
-
Phoenix, Arizona, Spojené státy, 85018
- Phoenix Neurological Associates, Ltd.
-
-
California
-
Fresno, California, Spojené státy, 93701
- University Neurology Associates
-
San Francisco, California, Spojené státy, 94115
- California Pacific Medical Center
-
-
Florida
-
Jacksonville, Florida, Spojené státy, 32224
- Mayo Clinic Florida
-
-
Kentucky
-
Lexington, Kentucky, Spojené státy, 40536
- University of Kentucky
-
-
Maryland
-
Baltimore, Maryland, Spojené státy, 21287
- Johns Hopkins Hospital
-
-
Massachusetts
-
Boston, Massachusetts, Spojené státy, 02114
- Massachusetts General Hospital
-
-
Missouri
-
Saint Louis, Missouri, Spojené státy, 63110
- Washington University
-
-
New York
-
Syracuse, New York, Spojené státy, 13210
- SUNY Upstate Medical Center
-
-
North Carolina
-
Durham, North Carolina, Spojené státy, 27705
- Duke University
-
-
Oregon
-
Portland, Oregon, Spojené státy, 97213
- Providence ALS Center
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, Spojené státy, 19102
- Drexel University College of Medicine, Dept of Neurology
-
University Park, Pennsylvania, Spojené státy, 17033
- Penn State
-
-
Texas
-
San Antonio, Texas, Spojené státy, 78229
- The University of Texas Health Science Center at San Antonio
-
-
Vermont
-
Burlington, Vermont, Spojené státy, 05401
- University of Vermont
-
-
Kritéria účasti
Kritéria způsobilosti
Věk způsobilý ke studiu
Přijímá zdravé dobrovolníky
Pohlaví způsobilá ke studiu
Popis
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)
- 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)
- Males or females 18 years of age or older
- Body Mass Index (BMI) of 18.0 to 30.0 kg/m2, inclusive
- Maximum voluntary grip strength in at least one hand between 10 and 40 pounds (females) or 10 and 60 pounds (males)
- Able to swallow capsules with water
- Upright Slow Vital Capacity (SVC) > 40% of predicted for age, height, and sex [See Appendix 16.6.1]
- Able to perform pulmonary function tests
- 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
- 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:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 times the upper limit of normal (ULN)
- Life expectancy < 3 months
- Participation in any trial in which receipt of investigational study drug occurred within 30 days prior to dosing
- Any prior treatment with CK-2017357
- In the opinion of the Investigator, the patient is not suitable to participate in the study
Studijní plán
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: Randomizované
- Intervenční model: Crossover Assignment
- Maskování: Čtyřnásobek
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
---|---|
Experimentální: 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.
|
Odpovídající placebo v kapslích podávaných jako jedna perorální dávka.
250 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
500 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
|
Experimentální: 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.
|
Odpovídající placebo v kapslích podávaných jako jedna perorální dávka.
250 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
500 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
|
Experimentální: 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.
|
Odpovídající placebo v kapslích podávaných jako jedna perorální dávka.
250 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
500 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
|
Experimentální: 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.
|
Odpovídající placebo v kapslích podávaných jako jedna perorální dávka.
250 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
500 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
|
Experimentální: 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.
|
Odpovídající placebo v kapslích podávaných jako jedna perorální dávka.
250 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
500 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
|
Experimentální: 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.
|
Odpovídající placebo v kapslích podávaných jako jedna perorální dávka.
250 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
500 mg CK-2017357 v tobolkách podávaných jako jedna perorální dávka.
Ostatní jména:
|
Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
---|---|---|
ALSFRS-R
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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)
Časové okno: 2 days
|
SVC is measured using the Puritan Bennett Renaissance II Spirometry System and accessories.
|
2 days
|
Maximum Voluntary Ventilation (MVV)
Časové okno: 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)
Časové okno: 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)
Časové okno: 2 days
|
MVC is measured using the MicroFET 2 HHD.
|
2 days
|
Repeated Sub-Maximum Grip Strength Fatigability
Časové okno: 2 days
|
Sub-Maximum Grip Strength Fatigability is measured using the DynEx Electronic Hand.
Dynamometer
|
2 days
|
Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
---|---|---|
Počet pacientů s nežádoucími účinky
Časové okno: 4 týdny
|
4 týdny
|
|
Characterize the relationship, if any, between the plasma concentration of CK-2017357 and ALSFRS-R.
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
|
Spolupracovníci a vyšetřovatelé
Sponzor
Vyšetřovatelé
- Studijní židle: Jeremy M Shefner, MD, PhD, State University of New York - Upstate Medical University
Publikace a užitečné odkazy
Termíny studijních záznamů
Hlavní termíny studia
Začátek studia
Primární dokončení (Aktuální)
Dokončení studie (Aktuální)
Termíny zápisu do studia
První předloženo
První předloženo, které splnilo kritéria kontroly kvality
První zveřejněno (Odhad)
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Naposledy ověřeno
Více informací
Termíny související s touto studií
Další relevantní podmínky MeSH
Další identifikační čísla studie
- CY 4021
Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .