Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01709149 | Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS) | PHASE2 | COMPLETED | 711 | — | — | Oct 1, 2012 | Mar 1, 2014 | Mar 31, 2020 | 75 | United States, Canada +6 |
| NCT01486849 | Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) | PHASE2 | COMPLETED | 27 | — | — | Nov 1, 2011 | Mar 1, 2012 | May 3, 2019 | 11 | United States |
| NCT01378676 | A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) | PHASE2 | COMPLETED | 49 | — | — | Jun 1, 2011 | Mar 1, 2012 | May 7, 2019 | 9 | United States |
| NCT01089010 | A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) | PHASE2 | COMPLETED | 67 | — | — | Mar 1, 2010 | Nov 1, 2010 | May 10, 2019 | 15 | United States |
The ALSFRS-R is used to measure the progression and severity of disease; it consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function.
Safety and tolerability of CK-2017357 after multiple oral doses to steady state in patients with ALS
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.
Measured using the DynEx Electronic Hand Dynamometer. Patients asked to squeeze the device with the maximum possible force to establish the maximum voluntary contraction.
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.
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.
SVC is measured using the Puritan Bennett Renaissance II Spirometry System and accessories.
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.
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
MVC is measured using the MicroFET 2 HHD.
Sub-Maximum Grip Strength Fatigability is measured using the DynEx Electronic Hand. Dynamometer
| Arm | Type | Description |
|---|---|---|
| CK-2017357 | EXPERIMENTAL | 125 mg tablets |
| Placebo | PLACEBO_COMPARATOR | Placebo tablets |
| Dose Titration of CK-2017357 (Group 1) | EXPERIMENTAL | Dose titration of active drug as add-on therapy to riluzole |
| Matching Placebo (Group 2) | PLACEBO_COMPARATOR | Placebo as add-on therapy to riluzole |
| Matching Placebo | PLACEBO_COMPARATOR | - |
| Active Drug Low Dose (CK-2017357 125 mg) | EXPERIMENTAL | - |
| Active Drug Mid Dose (CK-2017357 250 mg) | EXPERIMENTAL | - |
| Active Drug High Dose (CK-2017357 375 mg) | EXPERIMENTAL | - |
| Treatment Sequence 1 | EXPERIMENTAL | 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. |
| Treatment Sequence 2 | EXPERIMENTAL | 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. |
| Treatment Sequence 3 | EXPERIMENTAL | 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. |
| Treatment Sequence 4 | EXPERIMENTAL | 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. |
| Treatment Sequence 5 | EXPERIMENTAL | 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. |
| Treatment Sequence 6 | EXPERIMENTAL | 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. |
| Name | Type | Description |
|---|---|---|
| CK-2017357 | DRUG | CK-2017357 125 mg tablets twice daily |
| Placebo tablets | OTHER | Tablets |
| Riluzole | DRUG | Tablets |
| Placebo | DRUG | Matching placebo tablets BID for 21 days |
| Riluzole 50 MG | DRUG | - |
| Placebo (Part A) | DRUG | Placebo tablets once daily for 14 days (Part A) |
| CK-2017357 (Part A) | DRUG | One 125 mg CK-2017357 tablet once daily for 14 days (Part A) |
| Riluzole 50 MG (Part B) | DRUG | One 50 mg tablet once daily for 14 days (Part B) |
| Placebo (Part B) | DRUG | Placebo tablets once daily for 14 days (Part B) |
| CK-2017357 (Part B) | DRUG | One 125 mg tablet once daily for 14 days (Part B) |
| 250 mg CK-2017357 | DRUG | 250 mg CK-2017357 in capsules administered as a single oral dose. |
| 500 mg CK-2017357 | DRUG | 500 mg CK-2017357 in capsules administered as a single oral dose. |
Inclusion Criteria: 1. Able to comprehend and willing to sign an Informed Consent Form (ICF) 2. Male or female 18 years of age or older 3. A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS a...