Trial · NCT01622088
Phase 3 Extension Study of Dexpramipexole in ALS
NCT01622088, Phase 3, Terminated, Knopp Biosciences; results first posted 2022-04-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase 3 Extension Study of Dexpramipexole in ALS. Trials, record NCT01622088. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01622088
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Extension Study of Dexpramipexole in ALS
- NCT number
- NCT01622088
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 616 participants, actual
- Lead sponsor
- Knopp Biosciences
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- Dexpramipexole
- First posted
- 2012-06-18
- Last updated
- 2022-05-03
- Other registries' IDs
- eudract:2011-006119-70
- Results first posted
- 2022-04-07
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Subjects Who Reported an Adverse Event | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The number of subjects who reported an adverse event during the study |
| Primary | Number of Subjects Who Experienced a Serious Adverse Event | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The number of subjects enrolled who reported a serious adverse event during the study |
| Primary | Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study |
| Primary | Number of Participants With Potentially Clinically Significant Vital Sign Results | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test. |
| Primary | Number of Participants With Potentially Clinically Significant Hematology Results | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | Number of Participants with Potentially Clinically Significant Hematology Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test. |
| Primary | Number of Participants With Potentially Clinically Significant Blood Chemistry Results | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | Number of Participants with Potentially Clinically Significant Blood Chemistry Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test. |
| Primary | Number of Participants With Potentially Clinically Significant ECG Results | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | Number of Participants with Potentially Clinically Significant ECG Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test. |
| Secondary | Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study | Up to maximum 226 days: approximately 32.2 weeks | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score between 0 to 48, with higher scores representing better function. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis ALSFRS-R score. Units for slope are change per month in units on the ALSFRS-R scale. |
| Secondary | Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study | Up to maximum 226 days: approximately 32.2 weeks | SNIP is a test of inspiratory force (sternocleidomastoid and diaphragm) measured via a nasal cannula and is used to assess respiratory muscle weakness and to monitor changes in respiratory muscle strength over time. During the SNIP maneuver, the patient is asked to perform a strong, sharp, maximal sniff, whereby nasal pressure is measured via nasal cannula. The maximum recorded value after several attempts, with rest in between attempts, was use in the analysis. |
| Secondary | Death up to 6 Months | 6 Months | Kaplan-Meier estimate of percentage of subjects who died up to 6 months |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Subjects Who Reported an Adverse Event | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The safety population is defined as all subjects who received at least 1 dose of study treatment | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Subjects Who Experienced a Serious Adverse Event | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The safety population is defined as all subjects who received at least 1 dose of study treatment | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The safety population is defined as all subjects who received at least 1 dose of study treatment | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Vital Sign Results | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline vital sign measurement during the study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Hematology Results | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline hematology assessment during the study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Blood Chemistry Results | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline blood chemistry assessment during the study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant ECG Results | Baseline through end of study (maximum 226 days: approximately 32.2 weeks) | The analysis population includes all subjects who received at least one dose of study drug and who had an evaluable post-baseline ECG assessment during the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study | Up to maximum 226 days: approximately 32.2 weeks | Randomized subjects who had at least 1 post-baseline clinical evaluation | Change per month in ALSFRS-R | LEAST_SQUARES_MEAN |
| SECONDARY | Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study | Up to maximum 226 days: approximately 32.2 weeks | Randomized subjects who had at least 1 post-baseline clinical evaluation | Change per month in SNIP (in cm H20) | LEAST_SQUARES_MEAN |
| SECONDARY | Death up to 6 Months | 6 Months | Subjects who were randomized, received at least 1 dose of study treatment, had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period. | percentage of participants | NUMBER |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group88
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Subjects Who Reported an Adverse Event | Dexpramipexole 150 mg BID | 454 | Participants | ||
| Number of Subjects Who Experienced a Serious Adverse Event | Dexpramipexole 150 mg BID | 152 | Participants | ||
| Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event | Dexpramipexole 150 mg BID | 30 | Participants | ||
| Number of Participants With Potentially Clinically Significant Vital Sign Results | Dexpramipexole 150 mg BID | Systolic BP >180 mmHg | 2 | Participants | |
| Number of Participants With Potentially Clinically Significant Vital Sign Results | Dexpramipexole 150 mg BID | Systolic BP Increase from Baseline >40 mmHg | 6 | Participants | |
| Number of Participants With Potentially Clinically Significant Vital Sign Results | Dexpramipexole 150 mg BID | Systolic BP <90 mmHg | 4 | Participants | |
| Number of Participants With Potentially Clinically Significant Vital Sign Results | Dexpramipexole 150 mg BID | Systolic BP Decrease from Baseline >30 mmHg | 10 | Participants | |
| Number of Participants With Potentially Clinically Significant Vital Sign Results | Dexpramipexole 150 mg BID | Diastolic BP >105 mmHg | 20 | Participants | |
| Number of Participants With Potentially Clinically Significant Vital Sign Results | Dexpramipexole 150 mg BID | Diastolic BP increase from Baseline >30 mmHg | 5 | Participants | |
| Number of Participants With Potentially Clinically Significant Vital Sign Results | Dexpramipexole 150 mg BID | Diastolic BP <50 mmHg | 2 | Participants |
1 to 10 of 88Source ClinicalTrials.gov
See all 88 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Dexpramipexole 300 mg/Day | 616 | |
| Overall Study | COMPLETED | Dexpramipexole 300 mg/Day | 0 | |
| Overall Study | NOT COMPLETED | Dexpramipexole 300 mg/Day | 616 |
Source ClinicalTrials.gov
Adverse events86
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | URINARY TRACT INFECTION | Infections and infestations | Dexpramipexole 300 mg/Day | 31 | 616 | |
| other | RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | Dexpramipexole 300 mg/Day | 52 | 616 | |
| other | CONSTIPATION | Gastrointestinal disorders | Dexpramipexole 300 mg/Day | 49 | 616 | |
| other | NAUSEA | Gastrointestinal disorders | Dexpramipexole 300 mg/Day | 33 | 616 | |
| other | OEDEMA PERIPHERAL | General disorders | Dexpramipexole 300 mg/Day | 31 | 616 | |
| other | FALL | Injury, poisoning and procedural complications | Dexpramipexole 300 mg/Day | 68 | 616 | |
| serious | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | Dexpramipexole 300 mg/Day | 1 | 616 | |
| serious | LEUKOPENIA | Blood and lymphatic system disorders | Dexpramipexole 300 mg/Day | 1 | 616 | |
| serious | NEUTROPENIA | Blood and lymphatic system disorders | Dexpramipexole 300 mg/Day | 2 | 616 | |
| serious | CARDIAC ARREST | Cardiac disorders | Dexpramipexole 300 mg/Day | 2 | 616 |
1 to 10 of 86Source ClinicalTrials.gov
See all 86 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Dexpramipexole 300 mg/Day | 73 | 616 |
| Total, other adverse events | Dexpramipexole 300 mg/Day | 215 | 616 |
| Total, serious adverse events | Dexpramipexole 300 mg/Day | 152 | 616 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01622088
GET https://conductscience.org/api/trials/v1/NCT01622088/results?page=2