Trial · NCT01281189

Phase 3 Study of Dexpramipexole in ALS

NCT01281189, Phase 3, Completed, Knopp Biosciences; results first posted 2021-06-07

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Phase 3 Study of Dexpramipexole in ALS. Trials, record NCT01281189. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01281189

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Phase 3 Study of Dexpramipexole in ALS
NCT number: NCT01281189
Status: Completed
Phases: Phase 3
Enrollment: 942 participants, actual
Lead sponsor: Knopp Biosciences
Conditions: Amyotrophic Lateral Sclerosis
Interventions: Dexpramipexole, Placebo
First posted: 2011-01-21
Last updated: 2021-06-07
Other registries' IDs: eudract:2010-022818-19
Results first posted: 2021-06-07

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Composite Assessment of Function and Survival (CAFS) at 12 Months	12 months	The Composite Assessment of Function and Survival (CAFS) is a between-group comparison of a single ranked clinical outcome based on (1) the change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) score and (2) time to death. Each subject is ranked according to time-to-death (earlier deaths ranked lower than later deaths). Subjects who survive are ranked more favorably than subjects who died. Among the survivors, subjects are ranked according to change in ALSFRS-R (greater worsening of ALSFRS-R is ranked lower than less worsening or an improvement in ALSFRS-R). The ranked scores range from 001 to 941 (the number of subjects in the Efficacy Population) with larger rank score numbers associated with a better outcome. The ranks were analyzed using an ANCOVA model, which includes treatment as a fixed effect and adjusts for baseline ALSFRS-R score, duration of symptoms, site of onset, and use of riluzole. The least square mean rank score is presented for each treatment group.
Primary	Death up to 12 Months (CAFs Individual Component)	12 months	The longest duration of follow-up for this time to the death analysis was 12 months. In the study, subjects were followed for 12-18 months.
Primary	Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component)	12 months	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 of 48, with higher scores representing better function.
Secondary	Death or Respiratory Insufficiency (DRI) up to Month 18	18 months	Time to Death or Respiratory Insufficiency (DRI) is defined as receipt of a tracheostomy or the use of non-invasive ventilation (NIV) for ≥22 hours per day for at least 10 consecutive days. If NIV is used to meet the criteria for respiratory insufficiency, no measured slow vital capacity (SVC) at any subsequent assessment may be \>50%. Time to DRI is calculated from the date of the first dose to the first date of one of the following events: death, tracheostomy, or the 10th day of consecutive NIV with no measured SVC \>50% at any subsequent assessment.
Secondary	Death up to 18 Months	18 months	Estimated time to death up to 18 months. This includes deaths reported greater than 30 days following discontinuation from the study (the time period for reporting all-cause mortality), regardless of subject disposition, up to 18 months from first dose.
Secondary	≤50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months	18 months	The date of reaching ≤50% of predicted upright slow vital capacity (SVC) is defined as the date of the first visit at which a predicted upright SVC is ≤50% and continues to remain ≤50% at the subsequent visit except for the last available observation. The time to reach ≤50% of predicted upright SVC is defined as the duration between the date of reaching ≤50% of predicted upright SVC and the date of the first dose of study medication. If the subject is alive and does not reach ≤50% of predicted upright SVC, the time to reach ≤50% of predicted upright SVC will be censored and equal to the number of days from the first dose of study medication until the visit date when the subject's last available SVC assessment is performed. The earliest time (Reaching ≤50% Predicted Upright SVC or death) is used in analysis.

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Composite Assessment of Function and Survival (CAFS) at 12 Months	12 months	The efficacy population is defined as subjects who were randomized and received at least 1 dose of study treatment and who either had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period. Subjects were analyzed in the treatment group to which they are randomized.	units on a scale	LEAST_SQUARES_MEAN
PRIMARY	Death up to 12 Months (CAFs Individual Component)	12 months	The efficacy population is defined as subjects who were randomized and received at least 1 dose of study treatment and who either had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period. Subjects were analyzed in the treatment group to which they are randomized.	percentage of participants	NUMBER
PRIMARY	Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component)	12 months	The efficacy population is defined as subjects who were randomized and received at least 1 dose of study treatment and who either had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period. Subjects will be analyzed in the treatment group to which they are randomized.	units on a scale	LEAST_SQUARES_MEAN
SECONDARY	Death or Respiratory Insufficiency (DRI) up to Month 18	18 months	The efficacy population is defined as subjects who were randomized and received at least 1 dose of study treatment and who either had at least 1 available post-dosing efficacy evaluation (ALSFRS-R) or died during the study period. Subjects were analyzed in the treatment group to which they are randomized.	percentage of participants	NUMBER
SECONDARY	Death up to 18 Months	18 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. Subjects were analyzed in the treatment group to which they are randomized. An attempt was made to collect living status for each subject 18 months after randomization, regardless of the subject's disposition in the study (active or discontinued) or primary reasons for discontinuation.	percentage of participants	NUMBER
SECONDARY	≤50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months	18 months	The intent-to-treat (ITT) population is defined as subjects who were randomized and received at least 1 dose of study treatment.	percentage of participants	NUMBER

Source ClinicalTrials.gov

Results by group12

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Composite Assessment of Function and Survival (CAFS) at 12 Months	Placebo	438.84	95% Confidence Interval 412.807 to 464.878	units on a scale
Composite Assessment of Function and Survival (CAFS) at 12 Months	Dexpramipexole	441.76	95% Confidence Interval 415.426 to 468.084	units on a scale
Death up to 12 Months (CAFs Individual Component)	Placebo	17.2		percentage of participants
Death up to 12 Months (CAFs Individual Component)	Dexpramipexole	16.0		percentage of participants
Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component)	Placebo	-13.415	95% Confidence Interval -14.275 to -12.555	units on a scale
Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component)	Dexpramipexole	-13.339	95% Confidence Interval -14.199 to -12.480	units on a scale
Death or Respiratory Insufficiency (DRI) up to Month 18	Placebo	27.2		percentage of participants
Death or Respiratory Insufficiency (DRI) up to Month 18	Dexpramipexole	22.3		percentage of participants
Death up to 18 Months	Placebo	23.1		percentage of participants
Death up to 18 Months	Dexpramipexole	20.5		percentage of participants

1 to 10 of 12Source ClinicalTrials.gov

See all 12 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Placebo	468
Overall Study	STARTED	Dexpramipexole	474
Overall Study	COMPLETED	Placebo	321
Overall Study	COMPLETED	Dexpramipexole	331
Overall Study	NOT COMPLETED	Placebo	147
Overall Study	NOT COMPLETED	Dexpramipexole	143

Source ClinicalTrials.gov

Adverse events498

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
other	NASOPHARYNGITIS	Infections and infestations	Placebo	76	468
other	NASOPHARYNGITIS	Infections and infestations	Dexpramipexole	69	474
other	URINARY TRACT INFECTION	Infections and infestations	Placebo	56	468
other	URINARY TRACT INFECTION	Infections and infestations	Dexpramipexole	56	474
other	UPPER RESPIRATORY TRACT INFECTION	Infections and infestations	Placebo	17	468
other	UPPER RESPIRATORY TRACT INFECTION	Infections and infestations	Dexpramipexole	30	474
other	PNEUMONIA	Infections and infestations	Placebo	32	468
other	PNEUMONIA	Infections and infestations	Dexpramipexole	24	474
other	BRONCHITIS	Infections and infestations	Placebo	28	468
other	BRONCHITIS	Infections and infestations	Dexpramipexole	21	474

1 to 10 of 498Source ClinicalTrials.gov

See all 498 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo	108	468
Total, all-cause mortality	Dexpramipexole	97	474
Total, other adverse events	Placebo	447	468
Total, other adverse events	Dexpramipexole	459	474
Total, serious adverse events	Placebo	233	468
Total, serious adverse events	Dexpramipexole	225	474

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
24067398	DERIVED

Source PubMed

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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Dexpramipexole vs Placebo: deaths from any cause	Trial comparisons
Dexpramipexole vs Placebo: serious adverse events	Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT01281189

GET https://conductscience.org/api/trials/v1/NCT01281189/events?page=2

API reference For AI assistants