Trial · NCT01753076

Study of Ozanezumab (GSK1223249) Versus Placebo in the Treatment of Amyotrophic Lateral Sclerosis

NCT01753076, Phase 2, Completed, GlaxoSmithKline; results first posted 2017-04-19

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Study of Ozanezumab (GSK1223249) Versus Placebo in the Treatment of Amyotrophic Lateral Sclerosis. Trials, record NCT01753076. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01753076

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Trial

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

Title: Study of Ozanezumab (GSK1223249) Versus Placebo in the Treatment of Amyotrophic Lateral Sclerosis
NCT number: NCT01753076
Status: Completed
Phases: Phase 2
Enrollment: 304 participants, actual
Lead sponsor: GlaxoSmithKline
Conditions: Amyotrophic Lateral Sclerosis
Interventions: Ozanezumab, Placebo
First posted: 2012-12-20
Last updated: 2017-12-21
Results first posted: 2017-04-19

Source ClinicalTrials.gov

Registered outcomes10

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

List	Outcome	Time frame	Description
Primary	Joint Rank Scores for Combined Analysis of Function (Amyotrophic Lateral Sclerosis Functional Rating Scale Revised [ALSFRS-R] Score) and 48 Week Overall Survival	Week 48	The joint rank score is a combined assessment of function and survival. Function is assessed using change from Baseline in the ALSFRS-R total score. To calculate joint rank scores, every participant was compared with all other participants in a pair wise manner and assigned a score of -1, 0 or 1 based on their relative outcomes. A subject's joint rank score is the sum of their scores across the pair wise comparisons. The . The ALSFRS-R was a quickly administered (5 min) ordinal rating scale used to determine a participant's assessment of their capability and independence in 12 functional activities. There were 12 questions, graded by the participant 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing. Lower scores of ALSFRS-R reflect greater impairment.
Secondary	Change From Baseline in the ALSFRS-R Total Score at Week 48	Baseline and Week 48	The rate of decline was estimated by the change from Baseline in ALSFRS-R. The monthly slope for the ALSFRS-R score (i.e., the monthly rate of decline) was calculated as change from Baseline in the ALSFRS-R score at the last visit for that treatment period divided by the study day at the last visit for that treatment period /30.4. The Week 0 (Visit 2) value was considered to be the Baseline value. Change from Baseline was calculated by subtracting the derived Baseline value from the post-Baseline value. The ALSFRS-R was a quickly administered (5 min) ordinal rating scale used to determine a participant's assessment of their capability and independence in 12 functional activities. There were 12 questions, graded by the participant 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing.
Secondary	Rate of Decline Over Week 48 in the ALSFRS-R Total Score	Baseline to Week 48	The rate of decline was estimated by the change from Baseline in ALSFRS-R. The monthly slope for the ALSFRS-R score (i.e., the monthly rate of decline) was calculated as change from Baseline in the ALSFRS-R score at the last visit for that treatment period divided by the study day at the last visit for that treatment period devided by 30.4. The Week 0 (Visit 2) value was considered to be the Baseline value. Change from Baseline was calculated by subtracting the derived Baseline value from the post-Baseline value. The ALSFRS-R was a quickly administered (5 min) ordinal rating scale used to determine a participant's assessment of their capability and independence in 12 functional activities. There were 12 questions, graded by the participant 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing.
Secondary	Change From Baseline in Slow Vital Capacity (SVC) at Week 48	Baseline and Week 48	SVC was measured by using a validated spirometer. Three SVC measurements were performed for each participant at each assessment provided the difference from the second trial (if arranged by the numerical value) was not greater than 10%. If the difference between the best and the next best (based on the largest numerical value) SVC value from the first three trials was greater than 10%, additional trials (up to 5 in total) could have been performed. The Week 0 (visit 2) value was considered to be the Baseline value. Change from Baseline was calculated by subtracting the derived Baseline value from the post-Baseline value. A mixed-model repeated measures (MMRM) adjusted for treatment, visit, treatment by visit, Baseline SVC, Baseline SVC by visit, riluzole use. and country group was used for the analysis.
Secondary	Change From Baseline in Muscle Strength as Measured by Hand Held Dynamometry (HHD) Score at Week 48	Baseline and Week 48	The HHD is a device placed between the hand of the practitioner and the tested body part and provides a quantified measurement of muscle strength. Each muscle was tested twice, and both values were recorded. Additionally, a third trial could have been performed if the variability between the first two trials was greater than 15 % or if the rater thought that one of the first two trials was not valid. The Week 0 (Visit 2) value was considered to be the Baseline value. Percent change from Baseline for each muscle group was calculated as 100\*(HHD score minus the Baseline score) divided by the Baseline score. The average percent change was the mean percent change across the muscle groups that were non-missing/non-zero at Baseline. MMRM adjusted for treatment, visit, treatment by visit, number of non-missing/non-zero muscle groups at Baseline, number of non-missing/non-zero muscle groups at Baseline by Visit, riluzole use, and country group was used for the analysis.
Secondary	Number of Clinical Global Impression-improvement Scale (CGI-I) Responders at Week 48	Week 48	The CGI-I scale is a single observer-rated item measuring global improvement relative to Baseline. The CGI-I score is rated on a 7-point scale, from 1 (very much improved) to 7 (very much worse). Participant status at Baseline was assessed using the Clinical Global Impression Severity scale (CGI-S), which is a 7-point scale (1: normal, not at all ill; 7: most extremely ill) used to rate the severity of the participant's illness. Participants achieving a score of 1-4 in the CGI-I at Week 48 were considered to be responders. A a logistic regression adjusted for CGI-S at Baseline, riluzole use, and world region was used for the analysis.
Secondary	Overall Survival at Week 48 and Week 60	Week 48 and Week 60	Overall survival is defined as the time from randomization to death or censoring at the time point of analysis, whichever comes first. Kaplan Meier estimates at Week 48 were evaluated at Day 344. A participant was considered to have completed if he/she was censored at Day 344. Kaplan Meier estimates at Week 60 were evaluated at Day 428. A participant was considered to have completed if he/she was censored at Day 428. Confidence intervals were estimated using the Brookmeyer Crowley method. Results are shown as the estimated percentage of participants alive at Weeks 48 and 60. Week 48: Only on-treatment data (data within 21 days of the last dose) were analyzed. Week 60: Including off treatment data (data after 21 days after the last dose) were analyzed.
Secondary	Progression-free Survival at Week 48	Week 48	Progression-free survival at Week 48 is defined as the time from randomization to progression (decline of at least six points on the ALSFRS-R from Baseline) or death or censored at Week 48, whichever comes first. Kaplan Meier estimates at Week 48 were evaluated at Day 344. A participant was considered to have completed if he/she was censored at Day 344. Confidence intervals were estimated using the Brookmeyer Crowley method. Results are shown as the estimated percentage of participants alive and without disease progression at Week 48.
Secondary	Change From Baseline in the EuroQol 5 Dimensions-5 Level Short Form (EQ-5D-5L) Utility Score at Week 48	Baseline and Week 48	A utility score for each participant was calculated based on the value set for England. The Week 0 (Visit 2) value was considered to be the Baseline value. Change from Baseline was calculated by subtracting the derived Baseline value from the post-Baseline value. EQ-5D-5L is a standardized, participant-rated instrument for use as a measure of health outcomes. The EQ 5D-5L includes 2 components: the EQ-5D-5L descriptive system and the EQ-Visual Analog Scale (EQ-VAS). The EQ-5D-5L descriptive system provides a profile of the participant's health state in 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). . For each of these dimensions, the participant self assigned a score: 1 (no problems); 2 (slight problems); 3 (moderate problems); 4 (severe problems); 5 (extreme problems).Minimum score on scale was 1 and maximum score was 5 for each dimension. A negative change from Baseline indicates improvement.
Secondary	Change From Baseline in the Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Total Score at Week 48	Baseline and Week 48	The ALSAQ-40 is a disease specific health status assessment for individuals with ALS/motor neuron disease. The ALSAQ-40 is comprised of 40 questions measuring 5discrete dimensions of health status that are affected by the disease: physical mobility (10 items); activities of daily living and independence (10 items); eating and drinking (3 items); communication (7 items); emotional reactions (10 items). Participants were asked to indicate the frequency of each event by selecting one of five options (Likert scale: 0-4): never/rarely/sometimes/often/ always or cannot do at all. The total score (minimum possible score=0, maximum possible score=160) was calculated by adding the five domain scores. A low score indicates a better health state. Change from Baseline was calculated by subtracting the derived Baseline value from the post-Baseline value. A mixed-model repeated measures adjusted for treatment, Visit, Treatment by Visit, and Baseline ALSAQ-40 total score was used for the analysis.

Source ClinicalTrials.gov

Outcomes10

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Joint Rank Scores for Combined Analysis of Function (Amyotrophic Lateral Sclerosis Functional Rating Scale Revised [ALSFRS-R] Score) and 48 Week Overall Survival	Week 48	ITT population. Only on-treatment data (data within 21 days of the last dose) were analyzed.	Scores on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in the ALSFRS-R Total Score at Week 48	Baseline and Week 48	ITT Population. Only on-treatment data (data within 21 days of the last dose) were analyzed.	Scores on a scale	LEAST_SQUARES_MEAN
SECONDARY	Rate of Decline Over Week 48 in the ALSFRS-R Total Score	Baseline to Week 48	ITT Population. Only on-treatment data (data within 21 days of the last dose) were analyzed..	change in scores on a scale per month	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Slow Vital Capacity (SVC) at Week 48	Baseline and Week 48	ITT population. Only those participants available at indicated time points were analyzed.	Liters (L)	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Muscle Strength as Measured by Hand Held Dynamometry (HHD) Score at Week 48	Baseline and Week 48	ITT Population. Only participants with data available at the specified time points were analyzed.	Percent change	LEAST_SQUARES_MEAN
SECONDARY	Number of Clinical Global Impression-improvement Scale (CGI-I) Responders at Week 48	Week 48	ITT Population. Only participants with data available at the specified time points were analyzed.	Participants	NUMBER
SECONDARY	Overall Survival at Week 48 and Week 60	Week 48 and Week 60	ITT Population	Percentage of participants	NUMBER
SECONDARY	Progression-free Survival at Week 48	Week 48	ITT Population. Only on-treatment data (data within 21 days of the last dose) were analyzed.	Percentage of participants surviving	NUMBER
SECONDARY	Change From Baseline in the EuroQol 5 Dimensions-5 Level Short Form (EQ-5D-5L) Utility Score at Week 48	Baseline and Week 48	ITT Population. Only participants with data available at the specified time points were analyzed.	Scores on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in the Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Total Score at Week 48	Baseline and Week 48	ITT Population. Only participants with data available at the specified time points were analyzed	Scores on a scale	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group22

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

Outcome	Group	Value	Spread	Units
Joint Rank Scores for Combined Analysis of Function (Amyotrophic Lateral Sclerosis Functional Rating Scale Revised [ALSFRS-R] Score) and 48 Week Overall Survival	Placebo	15.0	Standard Error 13.58	Scores on a scale
Joint Rank Scores for Combined Analysis of Function (Amyotrophic Lateral Sclerosis Functional Rating Scale Revised [ALSFRS-R] Score) and 48 Week Overall Survival	Ozanezumab 15 mg/kg	-14.9	Standard Error 13.54	Scores on a scale
Change From Baseline in the ALSFRS-R Total Score at Week 48	Placebo	-9.1	Standard Error 0.64	Scores on a scale
Change From Baseline in the ALSFRS-R Total Score at Week 48	Ozanezumab 15 mg/kg	-10.4	Standard Error 0.64	Scores on a scale
Rate of Decline Over Week 48 in the ALSFRS-R Total Score	Placebo	-0.84	Standard Error 0.063	change in scores on a scale per month
Rate of Decline Over Week 48 in the ALSFRS-R Total Score	Ozanezumab 15 mg/kg	-0.96	Standard Error 0.062	change in scores on a scale per month
Change From Baseline in Slow Vital Capacity (SVC) at Week 48	Placebo	-0.899	Standard Error 0.0804	Liters (L)
Change From Baseline in Slow Vital Capacity (SVC) at Week 48	Ozanezumab 15 mg/kg	-1.026	Standard Error 0.0804	Liters (L)
Change From Baseline in Muscle Strength as Measured by Hand Held Dynamometry (HHD) Score at Week 48	Placebo	-34.7	Standard Error 3.77	Percent change
Change From Baseline in Muscle Strength as Measured by Hand Held Dynamometry (HHD) Score at Week 48	Ozanezumab 15 mg/kg	-42.9	Standard Error 3.75	Percent change

1 to 10 of 22Source ClinicalTrials.gov

See all 22 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Placebo	151
Overall Study	STARTED	Ozanezumab 15 mg/kg	152
Overall Study	COMPLETED	Placebo	110
Overall Study	COMPLETED	Ozanezumab 15 mg/kg	106
Overall Study	NOT COMPLETED	Placebo	41
Overall Study	NOT COMPLETED	Ozanezumab 15 mg/kg	46

Source ClinicalTrials.gov

Adverse events226

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	Fall	Injury, poisoning and procedural complications	Placebo	68	151
other	Fall	Injury, poisoning and procedural complications	Ozanezumab IV	56	152
other	Contusion	Injury, poisoning and procedural complications	Placebo	18	151
other	Contusion	Injury, poisoning and procedural complications	Ozanezumab IV	11	152
other	Diarrhoea	Gastrointestinal disorders	Placebo	12	151
other	Diarrhoea	Gastrointestinal disorders	Ozanezumab IV	25	152
other	Constipation	Gastrointestinal disorders	Placebo	12	151
other	Constipation	Gastrointestinal disorders	Ozanezumab IV	23	152
other	Nausea	Gastrointestinal disorders	Placebo	11	151
other	Nausea	Gastrointestinal disorders	Ozanezumab IV	14	152

1 to 10 of 226Source ClinicalTrials.gov

See all 226 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo	13	151
Total, all-cause mortality	Ozanezumab IV	18	152
Total, other adverse events	Placebo	119	151
Total, other adverse events	Ozanezumab IV	120	152
Total, serious adverse events	Placebo	46	151
Total, serious adverse events	Ozanezumab IV	47	152

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
28139349	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 comparisons3

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

Comparison	Source
Ozanezumab 15 mg/kg vs Placebo: Number of Clinical Global Impression-improvement Scale (CGI-I) Responders at Week 48	Trial comparisons
Ozanezumab IV vs Placebo: deaths from any cause	Trial comparisons
Ozanezumab IV 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.

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Read this trial from software.

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