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NCT01416181: ASCEND in SPMS

A Clinical Study of the Efficacy of Natalizumab on Reducing Disability Progression in Participants With Secondary Progressive Multiple Sclerosis

Terminated Phase 3 Results posted Last updated 11 September 2017
What this trial tests

Phase 3 trial testing natalizumab in Secondary Progressive Multiple Sclerosis in 889 participants. Terminated before completion.

Timeline
13 September 2011
Primary endpoint
28 July 2015
13 April 2016

Quick facts

Lead sponsorBiogen
PhasePhase 3
StatusTerminated
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingtriple
Primary purposetreatment
Enrollment889
Start date13 September 2011
Primary completion28 July 2015
Estimated completion13 April 2016
Sites161 locations across Denmark, France, Finland, Italy, Netherlands, Russia, Belgium, Sweden

Drugs / interventions tested

Conditions studied

Sponsor

Biogen — full company profile →

Who can join

Adults 18 to 58, any sex, with Secondary Progressive Multiple Sclerosis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Part 1: Percentage of Participants With Confirmed Progression of Disability in One or More of the Expanded Disability Status Scale (EDSS), Timed 25-Foot Walk (T25FW), or 9-Hole Peg Test (9HPT) Primary · Up to 96 weeks (2 years)

Confirmed disability progression, defined as ≥1 of the following criteria (confirmed at a second visit ≥6 months later and at Week 96): * Confirmed progression in EDSS (EDSS score increased from baseline \[BL\] by ≥1 point if BL EDSS ≤5.5 or by ≥0.5 points if BL EDSS ≥6); * Confirmed progression in T25FW (T25FW increased by ≥20% of the BL walk); * Confirmed progression in 9HPT (9HPT increased by ≥20% of the time taken at BL on either hand and confirmed on the same hand). The EDSS measures disability status on a scale ranging from 0 to 10, with higher scores indicating more disability. The T2

Confirmed on ≥1 of EDSS, T25FW, or 9HPT at 2 years
GroupValue95% CI
Placebo4843.1 – 52.4
Natalizumab 300 mg4439.8 – 49.1
Confirmed on EDSS at 2 years
GroupValue95% CI
Placebo1511.7 – 18.3
Natalizumab 300 mg1612.3 – 19.1
Confirmed on T25FW at 2 years
GroupValue95% CI
Placebo3530.8 – 39.7
Natalizumab 300 mg3530.4 – 39.3
Confirmed on 9HPT (either hand) at 2 years
GroupValue95% CI
Placebo2319.3 – 27.1
Natalizumab 300 mg1511.3 – 17.9
Confirmed on 9HPT (dominant hand) at 2 years
GroupValue95% CI
Placebo1310.2 – 16.5
Natalizumab 300 mg107.2 – 12.8
Confirmed on 9HPT (non-dominant hand) at 2 years
GroupValue95% CI
Placebo1612.5 – 19.2
Natalizumab 300 mg107.2 – 12.8
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Primary · 218 weeks

AE: any untoward medical occurrence that did not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: resulted in death; in the view of the Investigator, placed the participant at immediate risk of death (a life-threatening event); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in a congenital anomaly/birth defect. An SAE may have also been any other medically important event in the opinion of the Investigator.

Any event
GroupValue95% CI
Placebo250
Natalizumab 300 mg245
Moderate or severe event
GroupValue95% CI
Placebo157
Natalizumab 300 mg158
Severe event
GroupValue95% CI
Placebo28
Natalizumab 300 mg27
Related event
GroupValue95% CI
Placebo63
Natalizumab 300 mg56
Serious event
GroupValue95% CI
Placebo24
Natalizumab 300 mg39
Discontinuation of treatment due to event
GroupValue95% CI
Placebo12
Natalizumab 300 mg5
Withdrawal from study due to an event
GroupValue95% CI
Placebo11
Natalizumab 300 mg3
Part 1: Percentage of Participants With a T25FW Response Secondary · Up to 96 weeks

T25FW response is defined as any improvement from the best pre-dose T25FW in at least 75% of the scheduled on-treatment visits through Week 96. The T25FW is a quantitative mobility and leg function performance test based on a timed walk over 25 feet. The participant is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly as possible, but safely. The time is calculated from the initiation of the instruction to start and ends when the participant has reached the 25-foot mark. The task is immediately administered again by having the patient walk back

GroupValue95% CI
Placebo1712.7 – 20.3
Natalizumab 300 mg1914.6 – 22.4
Part 1: Change From Baseline in the 12-Item MS Walking Scale (MSWS-12) Secondary · Baseline and Week 96

MSWS-12 is a participant self-assessment of the walking limitations due to MS during the past 2 weeks. It contains 12 items that measure the impact of MS on walking. Items are summed to generate a total score and transformed to a scale with a range of 0 to 100, where higher scores indicate greater impact on walking. A negative number on change from BL value indicates an improvement in MSWS-12.

GroupValue95% CI
Placebo4.04± 21.061
Natalizumab 300 mg2.70± 22.110
Part 1: Change From Baseline in Manual Ability Score Based on the ABILHAND Questionnaire Secondary · Baseline and Week 96

The ABILHAND Questionnaire measures the participant's perceived difficulty in performing everyday manual activities in the last 3 months. The participant completes a 56-item questionnaire by estimating their own difficulty or ease in performing each of 56 activities. Items are summed to generate a total score and transformed to a scale with a range of 0 to 100, where high scores indicate greater impact on manual ability. A positive number on change from baseline value indicates an improvement in ABILHAND.

GroupValue95% CI
Placebo-3.45± 14.739
Natalizumab 300 mg-2.44± 13.023
Part 1: Change From Baseline in the Multiple Sclerosis Impact Scale-29 Physical (MSIS-29 Physical) Score Secondary · Baseline and Week 96

The 29-item MSIS-29 is a participant-reported outcome measure to assess the impact of MS on day-to-day life during the past 2 weeks from a participant's perspective; it measures 20 physical items and 9 psychological items. The physical score is generated by summing individual items and then transforming to a scale with a range of 0 to 100, where high scores indicate worse health. A negative number on change from baseline value indicates an improvement in MSIS-29.

GroupValue95% CI
Placebo3.34± 20.947
Natalizumab 300 mg0.61± 19.885
Part 1: Percentage Change From Week 24 in Whole Brain Volume at Week 96 Secondary · Week 24 and Week 96

Whole brain volume as measured by MRI.

GroupValue95% CI
Placebo-0.72± 0.656
Natalizumab 300 mg-0.66± 0.596
Part 1: Percentage of Participants Defined as Confirmed Progressors on EDSS Functional System Scores Secondary · Up to 96 weeks

The EDSS measures disability status on a scale ranging from 0 to 10, with higher scores indicating more disability. Scoring is based on measures of impairment in eight functional systems on examination by a neurologist. Participants with confirmed progression of disability in EDSS physical functional system scores will be defined as those who met one of the following criteria: * an increase of ≥ 1 point from baseline system score of ≥ 1 or an increase of ≥ 2 points from baseline system score of 0 in at least 2 physical functional systems, or * an increase of ≥ 2 points from baseline system sc

GroupValue95% CI
Placebo2925.2 – 33.7
Natalizumab 300 mg2520.6 – 28.6
Part 2: Percentage of Participants With Disability Worsening at 156 Weeks Secondary · Week 156

Percentage of participants with disability worsening at each scheduled efficacy visit in Part 2, defined as one or more of the following: • ≥ 20% worsening from Part 1 baseline in T25FW; • ≥ 20% worsening from Part 1 baseline in 9HPT; • Worsening from Part 1 baseline in EDSS (≥ 1 point increase if Part 1 baseline EDSS ≤ 5.5 or ≥ 0.5 point increase if Part 1 baseline EDSS \> 5.5). The EDSS measures disability status on a scale ranging from 0 to 10, with higher scores indicating more disability. The T25FW is a quantitative mobility and leg function performance test where the participant is timed

Confirmed on ≥1 of EDSS, T25FW, 9HPT at 156 weeks
GroupValue95% CI
Placebo6155.2 – 66.7
Natalizumab 300 mg5245.8 – 57.3
Confirmed on EDSS at 156 weeks
GroupValue95% CI
Placebo2318.3 – 28.4
Natalizumab 300 mg1813.8 – 22.6
Confirmed on T25FW at 156 weeks
GroupValue95% CI
Placebo4640.1 – 51.9
Natalizumab 300 mg4135.2 – 46.5
Confirmed on 9HPT (either hand) at 156 weeks
GroupValue95% CI
Placebo2823.1 – 33.8
Natalizumab 300 mg1914.4 – 23.4
Confirmed on 9HPT (dominant hand) at 156 weeks
GroupValue95% CI
Placebo1813.0 – 22.0
Natalizumab 300 mg128.0 – 15.4
Confirmed on 9HPT (non-dominant hand) at 156 weeks
GroupValue95% CI
Placebo1813.0 – 22.0
Natalizumab 300 mg139.2 – 16.9
Part 2: Absolute Change From Baseline (Part 1) in T25FW Secondary · Baseline (Part 1) and Weeks 156, 204

The T25FW is a quantitative mobility and leg function performance test based on a timed 25-foot walk. The participant is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly as possible, but safely. The time is calculated from the initiation of the instruction to start and ends when the participant has reached the 25-foot mark. The task is immediately repeated; the score for the T25FW is the average of the two completed trials. Lower scores on time taken to reach 25 foot mark reflect a better outcome. Values are presented for the overall group, as

Overall: Change from BL to Week 156; n=255, 264
GroupValue95% CI
Placebo12.80± 35.111
Natalizumab 300 mg7.78± 21.251
Overall: Change from BL to Week 204; n=39, 38
GroupValue95% CI
Placebo25.01± 56.582
Natalizumab 300 mg9.01± 22.507
CP Group: Change from BL to Week 156; n=156, 135
GroupValue95% CI
Placebo21.67± 42.510
Natalizumab 300 mg16.26± 26.943
CP Group: Change from BL to Week 204; n=25, 18
GroupValue95% CI
Placebo40.06± 66.275
Natalizumab 300 mg20.89± 28.200
NP Group: Change from BL to Week 156; n=99, 129
GroupValue95% CI
Placebo-1.17± 3.804
Natalizumab 300 mg-1.09± 3.593
NP Group: Change from BL to Week 204; n=14, 20
GroupValue95% CI
Placebo-1.88± 5.917
Natalizumab 300 mg-1.67± 4.613
Part 2: Percentage Change From Baseline (Part 1) in T25FW Secondary · Baseline (Part 1) and Weeks 156, 204

The T25FW is a quantitative mobility and leg function performance test based on a timed 25-foot walk. The participant is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly as possible, but safely. The time is calculated from the initiation of the instruction to start and ends when the participant has reached the 25-foot mark. The task is immediately repeated; the score for the T25FW is the average of the two completed trials. Values are presented for the overall group, as well as the CP (defined in the primary outcome measure description above)

Overall: Change from BL at Week 156; n=255, 264
GroupValue95% CI
Placebo75.52± 166.191
Natalizumab 300 mg55.91± 130.286
Overall: Change from BL at Week 204; n=39, 38
GroupValue95% CI
Placebo130.72± 272.117
Natalizumab 300 mg71.09± 177.668
CP Group: Change from BL at Week 156; n=156, 135
GroupValue95% CI
Placebo127.05± 195.138
Natalizumab 300 mg113.51± 160.857
CP Group: Change from BL at Week 204; n=25, 18
GroupValue95% CI
Placebo206.78± 316.344
Natalizumab 300 mg158.91± 228.458
NP Group: Change from BL at Week 156; n=99, 129
GroupValue95% CI
Placebo-5.68± 21.708
Natalizumab 300 mg-4.37± 25.050
NP Group: Change from BL at Week 204; n=14, 20
GroupValue95% CI
Placebo-5.09± 26.591
Natalizumab 300 mg-7.94± 29.856
Part 2: Absolute Change From Baseline (Part 1) in 9HPT (Dominant Hand) Secondary · Baseline (Part 1) and Weeks 156, 204

The 9HPT is a brief, standardized, quantitative test of upper extremity function. The participant picks up 9 pegs puts them in a block containing nine empty holes, and, once they are in the holes, removes them again as quickly as possible one at a time. The total time to complete the task is recorded. Two consecutive trials with the dominant hand are immediately followed by two consecutive trials with the non-dominant hand. The two trials for each hand are averaged. Values are presented for the overall group, as well as the CP (defined in the primary outcome measure description above) and NP s

Overall: Change from BL to Week 156; n= 257, 271
GroupValue95% CI
Placebo5.22± 27.728
Natalizumab 300 mg2.12± 16.719
Overall: Change from BL to Week 204; n=39, 38
GroupValue95% CI
Placebo0.56± 7.923
Natalizumab 300 mg2.65± 16.001
CP Group: Change from BL to Week 156; n=158, 138
GroupValue95% CI
Placebo10.12± 33.675
Natalizumab 300 mg5.01± 20.625
CP Group: Change from BL to Week 204; n=24, 19
GroupValue95% CI
Placebo2.36± 9.187
Natalizumab 300 mg6.03± 21.994
NP Group: Change from BL to Week 156; n=99, 133
GroupValue95% CI
Placebo-2.60± 9.543
Natalizumab 300 mg-0.89± 10.602
NP Group: Change from BL to Week 204; n=15, 19
GroupValue95% CI
Placebo-2.32± 4.153
Natalizumab 300 mg-0.73± 4.292

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events are captured through the last study visit; participants were followed through Week 228, or 24 weeks following last dose of study treatment, or premature withdrawal.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Placebo
Serious: 100/449 (22%)
Deaths:
Natalizumab 300 mg
Serious: 90/439 (21%)
Deaths:

Serious adverse events (150 terms)

ReactionSystemPlaceboNatalizumab 300 mg
Multiple sclerosis relapseNervous system disorders
Urinary tract infectionInfections and infestations
FallInjury, poisoning and procedural complications
PneumoniaInfections and infestations
Multiple sclerosisNervous system disorders
Anaphylactic reactionImmune system disorders
CellulitisInfections and infestations
GastroenteritisInfections and infestations
UrosepsisInfections and infestations
Uhthoff's phenomenonNervous system disorders
Acute myocardial infarctionCardiac disorders
Atrial fibrillationCardiac disorders
AstheniaGeneral disorders
Gait disturbanceGeneral disorders
Anaphylactic shockImmune system disorders
Lower respiratory tract infectionInfections and infestations
Ankle fractureInjury, poisoning and procedural complications
Fibula fractureInjury, poisoning and procedural complications
Muscle spasticityNervous system disorders
Optic neuritisNervous system disorders
Deep vein thrombosisVascular disorders
AnaemiaBlood and lymphatic system disorders
Coronary artery diseaseCardiac disorders
TachycardiaCardiac disorders
Pelvic kidneyCongenital, familial and genetic disorders
Other adverse events (22 terms — click to expand)

ReactionSystemPlaceboNatalizumab 300 mg
Multiple sclerosis relapseNervous system disorders
Urinary tract infectionInfections and infestations
NasopharyngitisInfections and infestations
FallInjury, poisoning and procedural complications
HeadacheNervous system disorders
FatigueGeneral disorders
Back painMusculoskeletal and connective tissue disorders
Upper respiratory tract infectionInfections and infestations
ArthralgiaMusculoskeletal and connective tissue disorders
Pain in extremityMusculoskeletal and connective tissue disorders
Muscular weaknessMusculoskeletal and connective tissue disorders
DizzinessNervous system disorders
NauseaGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
InfluenzaInfections and infestations
Muscle spasticityNervous system disorders
ConstipationGastrointestinal disorders
ContusionInjury, poisoning and procedural complications
CoughRespiratory, thoracic and mediastinal disorders
Muscle spasmsMusculoskeletal and connective tissue disorders
CystitisInfections and infestations
ParaesthesiaNervous system disorders

Most-reported serious reactions: Multiple sclerosis relapse, Urinary tract infection, Fall, Pneumonia, Multiple sclerosis, Anaphylactic reaction, Cellulitis, Gastroenteritis.

Data from ClinicalTrials.gov NCT01416181 adverse events section.

Sponsor's own description

This is a Phase 3b, multicenter, international study conducted in 2 parts. Upon completion of the placebo-controlled period (Part 1), participants will have the option of enrolling in a 2-year open-label extension (Part 2). Part 1: The primary objective of the study is to investigate whether treatment with natalizumab slows the accumulation of disability not related to relapses in participants with secondary progressive multiple sclerosis (SPMS). The secondary objectives of Part 1 of this study are to determine the proportion of participants with consistent improvement in Timed 25-Foot Walk (T25FW), the change in participant-reported ambulatory status as measured by the 12-item MS Walking Scale (MSWS-12), the change in manual ability based on the ABILHAND Questionnaire, the impact of natalizumab on participant-reported quality of life using the Multiple Sclerosis Impact Scale-29 Physical (MSIS-29 Physical), the change in whole brain volume between the end of study and Week 24 using magnetic resonance imaging (MRI) and the proportion of participants experiencing progression of disability as measured by individual physical Expanded Disability Status Scale (EDSS) system scores. Part 2: The primary objective of Part 2 of the study is to evaluate the safety profile of natalizumab in participants with SPMS. The secondary objectives of Part 2 of the study are to investigate long-term disability (based on clinical or participant-reported assessments) in participants with SPMS receiving natalizumab treatment for approximately 4 years and to assess change in brain volume and T2 lesion volume.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Effect of natalizumab on disease progression in secondary progressive multiple sclerosis (ASCEND): a phase 3, randomised, double-blind, placebo-controlled trial with an open-label extension.
    Kapoor R, Ho PR, Campbell N, Chang I, et al · · 2018 · cited 245× · PMID 29545067 · DOI 10.1016/s1474-4422(18)30069-3
  2. Clinical trials in progressive multiple sclerosis: lessons learned and future perspectives.
    Ontaneda D, Fox RJ, Chataway J. · · 2015 · cited 161× · PMID 25772899 · DOI 10.1016/s1474-4422(14)70264-9
  3. The role of immune cells, glia and neurons in white and gray matter pathology in multiple sclerosis.
    Mallucci G, Peruzzotti-Jametti L, Bernstock JD, Pluchino S. · · 2015 · cited 100× · PMID 25802011 · DOI 10.1016/j.pneurobio.2015.02.003
  4. Therapeutic Advances and Future Prospects in Progressive Forms of Multiple Sclerosis.
    Shirani A, Okuda DT, Stüve O. · · 2016 · cited 69× · PMID 26729332 · DOI 10.1007/s13311-015-0409-z
  5. Promoting remyelination in multiple sclerosis-recent advances.
    Münzel EJ, Williams A. · · 2013 · cited 66× · PMID 24242317 · DOI 10.1007/s40265-013-0146-8
  6. Development of a Sensitive Outcome for Economical Drug Screening for Progressive Multiple Sclerosis Treatment.
    Kosa P, Ghazali D, Tanigawa M, Barbour C, et al · · 2016 · cited 44× · PMID 27574516 · DOI 10.3389/fneur.2016.00131
  7. Efficacy and side effects of natalizumab therapy in patients with multiple sclerosis.
    Hoepner R, Faissner S, Salmen A, Gold R, et al · · 2014 · cited 43× · PMID 24855407 · DOI 10.4137/jcnsd.s14049
  8. Anti-inflammatory disease-modifying treatment and short-term disability progression in SPMS.
    Lorscheider J, Jokubaitis VG, Spelman T, Izquierdo G, et al · · 2017 · cited 37× · PMID 28794248 · DOI 10.1212/wnl.0000000000004330

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01416181.

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