Phase 2
Completed N=74
Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)
Source: ClinicalTrials.gov NCT00931944 ↗Enrolled (actual)
74
Serious AEs
64.9%
Results posted
Aug 2021
Primary outcomePrimary: Number of Participants With Potentially Clinically Significant Hematology Results — 5; 2; 3; 4 Participants
Summary
This is an open-label, multi-center study designed to extend the evaluation of the safety, tolerability, and clinical effects of oral administration of KNS-760704 in patients with ALS.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Potentially Clinically Significant Hematology Results |
5; 2; 3; 4 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities |
1; 5; 2; 3; 0; 0 | — |
| PRIMARY Number of Participants With Potentially Clinically ECG Abnormalities |
5; 1; 2; 3; 3; 2 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities |
4; 26; 1; 9; 0; 3 | — |
| SECONDARY Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12 |
-2.48 | — |
| SECONDARY Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24 |
-4.29 | — |
| SECONDARY Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48 |
-7.49 | — |
| SECONDARY Change in Upright Vital Capacity From Baseline to Week 12 |
-7.7 | — |
| SECONDARY Change in Upright Vital Capacity From Baseline to Week 24 |
-12.5 | — |
| SECONDARY Change in Upright Vital Capacity From Baseline to Week 48 |
-15.8 | — |
| SECONDARY Change in McGill Single-Item Scale (SIS) From Baseline to Week 12 |
-0.1 | — |
| SECONDARY Change in McGill Single-Item Scale (SIS) From Baseline to Week 24 |
-0.5 | — |
| SECONDARY Change in McGill Single-Item Scale (SIS) From Baseline to Week 48 |
-0.8 | — |
| SECONDARY Number of Subjects With Feeding Tube Placed During the Study. |
24 | — |
Eligibility Criteria
Inclusion Criteria
- Patient has provided signed informed consent for this trial before the commencement of any study-related procedure
- Patient is actively participating in Knopp Protocol KNS-760704-CL201 and has completed the Part 2 Week 28 visit in that study or patient is actively receiving RTPB under Research IND #60,948
Exclusion Criteria
- Patient did not participate in Knopp Protocol KNS-760704-CL201 or patient did not receive RTPB under Research IND #60,948.
- Patient discontinued from KNS-760704-CL201 for any reason other than enrollment into this study (applies to patients enrolled in KNS-760704-CL201 only)
- Patient was not taking RTPB under Research IND #60,948 prior to April 30, 2009 (applies to patients enrolled under Research IND #60,948 only)
Data sourced from ClinicalTrials.gov (NCT00931944). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.