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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-005375-16 | EudraCT Number |
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The study was terminated early due to slow recruitment.
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The aim of the study was to compare Dysport treatment results (as assessed by Modified Ashworth Scale (MAS) in the elbow joint 4 weeks post treatment) following two treatment techniques: the current clinical practice injection technique using high-concentration dilution (300 U/mL Dysport) versus the neuromuscular junction (NMJ)-targeted injection technique using low-concentration dilution (100 U/mL Dysport). The hypothesis was that one high-volume, low-concentration injection located centrally in the area/band of the NMJ zones would be as effective as the technique used in current medical practice.
This was a randomised, evaluator-blinded, comparative, parallel group, multicentre study, conducted in four countries (Denmark, Finland, Norway and Sweden). For each subject, the primary efficacy assessments using MAS were performed by the same blinded evaluator, and every effort was made at each site to ensure that the MAS evaluator was the same person throughout the study. Dysport doses were to follow clinical practice for subjects suffering from upper limb spasticity position pattern type 1, 3 or 4 post stroke or traumatic brain injury.
The hypothesis for this study was that one low-concentration botulinum neurotoxin type A (BoNT-A) injection per muscle, centrally located in the area/band of the NMJ zones, would spread to and block the surrounding NMJ zones and be as effective as the technique used today in clinical practice. The possibility to reduce the number of injection points would decrease the risk of injection discomfort, pain and injection site bleeding for the patient. A simplified injection technique with one injection per muscle and in a defined location would also benefit physicians.
At Baseline (Visit 1), subjects were randomised to one of two treatment groups:
Each subject visited the clinic on at least three occasions:
For subjects not receiving any post study BoNT-A injection at Week 12 due to remaining treatment effect, an extra visit (Visit 4) for study follow-up was to take place at the next routine visit planned for a new BoNT-A injection, at the latest 24 weeks post study injection.
The duration of each subject's study period was at a minimum 12 weeks and maximum 24 weeks. The overall duration of the study was planned to be approximately 36 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NMJ Targeted | Experimental | NMJ targeted technique and low-concentration dilution (Dysport 100 U/mL). The same number and sites of injections/deposits per muscle were given as per prestudy. With a Dysport dilution of 300 U/mL the volume to be injected varied between 0.1 mL and 0.7 mL per muscle. The dose and the choice of muscles involved in the elbow flexion were the same as for the last prestudy treatment. |
|
| Current Clinical Practice | Active Comparator | Current clinical practice technique and high-concentration dilution (Dysport 300 U/mL). A single injection per muscle was given in the midline of the band of NMJ zones. With a Dysport dilution of 100 U/mL the volume to be injected varied between 0.4 mL and 2.0 mL per muscle. The dose and the choice of muscles involved in the elbow flexion were the same as for the last prestudy treatment. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Botulinum toxin type A | Biological |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline for Elbow Flexors Muscle Tone as Measured by the Modified Ashworth Scale (MAS) at Week 4 | A clinically relevant change was one level decrease on the MAS scale. Subjects meeting the defined decrease at Week 4 were considered as responders in the primary efficacy analysis. | Baseline to Week 4 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline for Elbow Flexors Muscle Tone as Measured by the Modified Ashworth Scale (MAS) at Week 4 and Week 12 | Increased muscle tone in elbow flexors was assessed using the MAS. Scale ranges from 0 (no increase in muscle tone) to 4 (affected part rigid in flexion or extension). | Baseline to Week 12 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Aalborg Sygehus Nord | Aalborg | 9000 | Denmark | |||
| Glostrup Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31061021 | Derived | Rekand T, Biering-Sorensen B, He J, Vilholm OJ, Christensen PB, Ulfarsson T, Belusa R, Strom T, Myrenfors P, Maisonobe P, Dalager T. Botulinum toxin treatment of spasticity targeted to muscle endplates: an international, randomised, evaluator-blinded study comparing two different botulinum toxin injection strategies for the treatment of upper limb spasticity. BMJ Open. 2019 May 5;9(5):e024340. doi: 10.1136/bmjopen-2018-024340. |
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Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized, and study documents will be redacted to protect the privacy of study participants.
Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.
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Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.
Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).
272 subjects were planned to be randomised (136 subjects in each group). In total, 100 subjects were enrolled and 88 (44 subjects in each group) were randomised and received study medication; 12 subjects were screening failures.
From September 2012, subjects were recruited across 20 sites in four countries: Denmark (5 sites), Finland (2 sites), Norway (2 sites) and Sweden (11 sites). Due to slow recruitment, the study was terminated early.
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| ID | Title | Description |
|---|---|---|
| FG000 | NMJ Targeted | Neuromuscular junction (NMJ) targeted technique and low-concentration dilution (Dysport 100 U/mL): A single injection per muscle was given in the midline of the band of NMJ zones. With a Dysport dilution of 100 U/mL the volume to be injected varied between 0.4 mL and 2.0 mL per muscle. The dose and the choice of muscles involved in the elbow flexion were the same as for the last prestudy treatment. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Change From Baseline for Elbow Flexors Muscle Tone as Measured by the MAS at Week 12 |
A clinically relevant change was one level decrease on the MAS scale. Subjects meeting the defined decrease at Week 12 were considered as responders. |
| Baseline to Week 12 |
| Change From Baseline of Spasticity Related Pain Measured by Visual Analogue Scale (VAS), Assessed by the Subject | Pain assessment using the VAS. The VAS was a 10 cm straight horizontal line scoring scale. Score range on VAS was from 0 to 10 where 0 indicated no pain and 10 indicated worst pain imaginable. | Baseline, Week 4 and Week 12 |
| Injection Site Pain Measured by VAS at Day 1. | Pain assessment using the VAS. The VAS was a 100 mm straight horizontal line scoring scale. Score range on VAS was from 0 to 100 where 0 indicated no pain and 100 indicated worst pain imaginable. | Baseline |
| Achievement of the Primary Goal Measured by Goal Attainment Scale (GAS) | At Baseline, an agreed primary goal related to elbow flexion in one of the following categories was determined: active function, impairment, involuntary movement, mobility, pain passive function or other. Each goal was usually rated -1, unless the subject was as bad as he/she could be in that particular goal area, in which case the Baseline score was -2. The evaluator rated the outcome score at Week 4 or Week 12, depending on the time point defined at the baseline visit (Visit 1), using the GAS five-point scale (-2, -1, 0, +1 and +2). | Up to Week 12 |
| Subject Global Evaluation of Treatment Effect | Comparison of treatment effect between previous (prestudy) and study treatment cycles assessed by the subject at the end of study (Week 12 up to Week 24 (Visit 3 or Visit 4)). Categorised as follows: Much worse / Worse / Same / Better / Much better. | Up to Week 24 |
| Investigator Preference of Injection Technique | When all patients at the site had completed the study (last subject last visit) a global assessment of the injection technique was to be made by the Investigators. The following question was answered: "Based on your experience during this study, which injection technique do you prefer?" | Following last visit of the last subject at each site |
| Glostrup Municipality |
| 2600 |
| Denmark |
| Regionshospitalet Hammel | Hammel | 8450 | Denmark |
| Bispebjerg Hospital | København NV | 2400 | Denmark |
| Roskilde Hospital | Roskilde | 4000 | Denmark |
| Vejle Hospital | Vejle | 7100 | Denmark |
| Regionshopsitalet Viborg | Viborg | 8800 | Denmark |
| North Karelia Central Hospital | Joensuu | 80210 | Finland |
| Central Hospital of Central Finland | Jyväskylä | 40503 | Finland |
| Haukeland University Hospital | Bergen | 5021 | Norway |
| Sykehuset Telemark HF | Skien | , 3700 | Norway |
| Mälarsjukhuset MSE | Eskilstuna | 631-88 | Sweden |
| Sahlgrenska University Hospital | Gothenburg | Sweden |
| Hallands Sjukhus, Neurology Clinic | Halmstad | 30185 | Sweden |
| Sundsvall-Härnösand, Rehabilitation Medicine | Härnösand | 87182 | Sweden |
| Nyköpings Lasarett, | Nyköping | 61185 | Sweden |
| Örnsköldsviks Sjukhus, Neurology Clinic | Örnsköldsvik | 891 89 | Sweden |
| Östersunds Rehabilitation Center | Östersund | 83102 | Sweden |
| Neurorehab Sävar | Sävar | 91831 | Sweden |
| Neurology Clinic Stockholm | Stockholm | 114 33 | Sweden |
| Danderyds Hospital, | Stockholm | 18288 | Sweden |
| Rehabilitation Center Gotland | Visby | 62184 | Sweden |
| Ystad Lasarett | Ystad | 27133 | Sweden |
| FG001 | Current Clinical Practice | Current clinical practice technique and high-concentration dilution (Dysport 300 U/mL): The same number and sites of injections/deposits per muscle were given as per prestudy. With a Dysport dilution of 300 U/mL the volume to be injected varied between 0.1 mL and 0.7 mL per muscle. The dose and the choice of muscles involved in the elbow flexion were the same as for the last prestudy treatment. |
| COMPLETED |
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| NOT COMPLETED |
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The intention-to-treat (ITT) population was defined as all treated subjects having at least one Baseline assessment of the primary efficacy parameter. The ITT population was analysed using subjects as randomised.
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| ID | Title | Description |
|---|---|---|
| BG000 | NMJ Targeted | NMJ targeted technique and low-concentration dilution (Dysport 100 U/mL): A single injection per muscle was given in the midline of the band of NMJ zones. With a Dysport dilution of 100 U/mL the volume to be injected varied between 0.4 mL and 2.0 mL per muscle. The dose and the choice of muscles involved in the elbow flexion were the same as for the last prestudy treatment. |
| BG001 | Current Clinical Practice | Current clinical practice technique and high-concentration dilution (Dysport 300 U/mL): The same number and sites of injections/deposits per muscle were given as per prestudy. With a Dysport dilution of 300 U/mL the volume to be injected varied between 0.1 mL and 0.7 mL per muscle. The dose and the choice of muscles involved in the elbow flexion were the same as for the last prestudy treatment. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants | No |
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| Region of Enrollment | Count of Participants | Participants | No |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline for Elbow Flexors Muscle Tone as Measured by the Modified Ashworth Scale (MAS) at Week 4 | A clinically relevant change was one level decrease on the MAS scale. Subjects meeting the defined decrease at Week 4 were considered as responders in the primary efficacy analysis. | The primary analysis was based on both the ITT and Per Protocol (PP) populations. The ITT population was all treated subjects having at least one Baseline assessment of the primary efficacy parameter. The PP population was all subjects in the ITT population for whom no major protocol violations or deviations occurred until Week 4 (Visit 2). | Posted | Number | responders | Baseline to Week 4 |
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| Secondary | Change From Baseline for Elbow Flexors Muscle Tone as Measured by the Modified Ashworth Scale (MAS) at Week 4 and Week 12 | Increased muscle tone in elbow flexors was assessed using the MAS. Scale ranges from 0 (no increase in muscle tone) to 4 (affected part rigid in flexion or extension). | ITT population: all treated subjects having at least one Baseline assessment of the primary efficacy parameter. Only subjects with non-missing values were included in this analysis. CCP=Current Clinical Practice; N'=number of subjects with data at each time point for each treatment group; NMJ=neuromuscular junction. | Posted | Median | Full Range | units on a scale | Baseline to Week 12 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline for Elbow Flexors Muscle Tone as Measured by the MAS at Week 12 | A clinically relevant change was one level decrease on the MAS scale. Subjects meeting the defined decrease at Week 12 were considered as responders. | ITT population: all treated subjects having at least one Baseline assessment of the primary efficacy parameter. Only subjects with non-missing values were included in the analysis. | Posted | Number | responders | Baseline to Week 12 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline of Spasticity Related Pain Measured by Visual Analogue Scale (VAS), Assessed by the Subject | Pain assessment using the VAS. The VAS was a 10 cm straight horizontal line scoring scale. Score range on VAS was from 0 to 10 where 0 indicated no pain and 10 indicated worst pain imaginable. | ITT population: all treated subjects having at least one Baseline assessment of the primary efficacy parameter. CCP=Current Clinical Practice; N'=number of subjects with data at each time point for each treatment group; NMJ=neuromuscular junction. | Posted | Mean | Standard Deviation | Change in VAS from Baseline | Baseline, Week 4 and Week 12 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Injection Site Pain Measured by VAS at Day 1. | Pain assessment using the VAS. The VAS was a 100 mm straight horizontal line scoring scale. Score range on VAS was from 0 to 100 where 0 indicated no pain and 100 indicated worst pain imaginable. | ITT population: all treated subjects having at least one Baseline assessment of the primary efficacy parameter. Only subjects with non-missing values were included in the analysis. | Posted | Mean | Standard Deviation | mm | Baseline |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Achievement of the Primary Goal Measured by Goal Attainment Scale (GAS) | At Baseline, an agreed primary goal related to elbow flexion in one of the following categories was determined: active function, impairment, involuntary movement, mobility, pain passive function or other. Each goal was usually rated -1, unless the subject was as bad as he/she could be in that particular goal area, in which case the Baseline score was -2. The evaluator rated the outcome score at Week 4 or Week 12, depending on the time point defined at the baseline visit (Visit 1), using the GAS five-point scale (-2, -1, 0, +1 and +2). | ITT population: all treated subjects having at least one Baseline assessment of the primary efficacy parameter. Only subjects with non-missing values were included in the analysis. Only overall GAS score data are summarised below. | Posted | Count of Participants | Participants | Up to Week 12 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Subject Global Evaluation of Treatment Effect | Comparison of treatment effect between previous (prestudy) and study treatment cycles assessed by the subject at the end of study (Week 12 up to Week 24 (Visit 3 or Visit 4)). Categorised as follows: Much worse / Worse / Same / Better / Much better. | ITT population: all treated subjects having at least one Baseline assessment of the primary efficacy parameter. Only subjects with non-missing values were included in the analysis. If a subject had more than one evaluation entry, the last one was used. | Posted | Count of Participants | Participants | Up to Week 24 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Investigator Preference of Injection Technique | When all patients at the site had completed the study (last subject last visit) a global assessment of the injection technique was to be made by the Investigators. The following question was answered: "Based on your experience during this study, which injection technique do you prefer?" | The question on preferred injection technique was answered by 3 of the 20 Investigators. | Posted | Number | Investigators | Following last visit of the last subject at each site |
|
|
Adverse events (AEs) were monitored from informed consent to the time when the subject's participation in the study ended (Week 12 to Week 24 (Visit 3 or Visit 4)). AEs were elicited by direct, non-leading questioning at each clinic visit or by spontaneous reports.
The safety population was defined as all subjects who received at least one dose of study medication. The safety population was analysed using subjects as treated. If a subject experienced more than one event in a System Organ Class (SOC), the subject was counted only once in that SOC.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | NMJ Targeted | NMJ targeted technique and low-concentration dilution (Dysport 100 U/mL): A single injection per muscle was given in the midline of the band of NMJ zones. With a Dysport dilution of 100 U/mL the volume to be injected varied between 0.4 mL and 2.0 mL per muscle. The dose and the choice of muscles involved in the elbow flexion were the same as for the last prestudy treatment. | 2 | 44 | 13 | 44 | ||
| EG001 | Current Clinical Practice | Current clinical practice technique and high-concentration dilution (Dysport 300 U/mL): The same number and sites of injections/deposits per muscle were given as per prestudy. With a Dysport dilution of 300 U/mL the volume to be injected varied between 0.1 mL and 0.7 mL per muscle. The dose and the choice of muscles involved in the elbow flexion were the same as for the last prestudy treatment . | 2 | 44 | 9 | 44 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cystitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Eye infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Injection site hypersensitivity | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Epididymitis | Reproductive system and breast disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Acquired hydrocele | Reproductive system and breast disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pain of skin | Skin and subcutaneous tissue disorders | MedDRA 15.0 | Systematic Assessment |
|
Due to slow recruitment, the study was terminated early. Therefore, fewer subjects than planned were enrolled.
The Sponsor required reasonable opportunity to review any abstract, presentation or paper before the material was submitted for publication or communicated. This also applied to any amendments that were requested by referees or journal editors. The Sponsor committed to comment on the draft documents within the time period agreed in the contractual arrangements between the Sponsor and authors or their institution. Delays were also possible if publication would adversely affect patentability.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director, Neurology | Ipsen | clinicaltrials@ipsen.com | clinicaltrials@ipsen.com |
| ID | Term |
|---|---|
| D019274 | Botulinum Toxins, Type A |
| C542869 | abobotulinumtoxinA |
| ID | Term |
|---|---|
| D001905 | Botulinum Toxins |
| D008666 | Metalloendopeptidases |
| D010450 | Endopeptidases |
| D010447 | Peptide Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
| D045726 | Metalloproteases |
| D001426 | Bacterial Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D001427 | Bacterial Toxins |
| D014118 | Toxins, Biological |
| D001685 | Biological Factors |
Not provided
Not provided
| Male |
|
| Norway |
|
| Finland |
|
| Denmark |
|
| A responder was defined as a subject with at least one level decrease on the MAS scale. | Generalised linear model | 0.5682 | Based on a generalised linear model including factors for spasticity pattern. | Treatment difference | -0.1673 | 2-Sided | 95 | -0.3630 | 0.0284 | Difference of the two clinical success rates (NMJ Targeted minus Current Clinical Practice). | Non-Inferiority or Equivalence (legacy) | Based on a π of 63% in the reference group, a clinically relevant Δ of 17%, α=2.5% (one sided) and power=80%, the sample size estimate yielded 122 valid subjects per group. Including a 10% addition for premature withdrawals, 136 subjects per group were necessary to be randomised in the study (i.e., a total of 272 subjects). |
| A responder was defined as a subject with at least one level decrease on the MAS scale. | Generalised linear model | 0.8543 | Based on a generalised linear model including factors for country. | Treatment difference | -0.1673 | 2-Sided | 5 | -0.3630 | 0.0284 | Difference of the two clinical success rates (NMJ Targeted minus Current Clinical Practice). | Non-Inferiority or Equivalence (legacy) | Based on a π of 63% in the reference group, a clinically relevant Δ of 17%, α=2.5% (one sided) and power=80%, the sample size estimate yielded 122 valid subjects per group. Including a 10% addition for premature withdrawals, 136 subjects per group were necessary to be randomised in the study (i.e., a total of 272 subjects). |
| A responder was defined as a subject with at least one level decrease on the MAS scale. | Generalised linear model | 0.9167 | Based on a generalised linear model including factors for MAS at Baseline. | Treatment difference | -0.1673 | 2-Sided | 95 | -0.3630 | 0.0284 | Difference of the two clinical success rates (NMJ Targeted minus Current Clinical Practice). | Non-Inferiority or Equivalence (legacy) | Based on a π of 63% in the reference group, a clinically relevant Δ of 17%, α=2.5% (one sided) and power=80%, the sample size estimate yielded 122 valid subjects per group. Including a 10% addition for premature withdrawals, 136 subjects per group were necessary to be randomised in the study (i.e., a total of 272 subjects). |
| A responder was defined as a subject with at least one level decrease on the MAS scale. | Generalised linear model | 0.6052 | Based on a generalised linear model including factors for treatment. | Treatment difference | 0.0707 | 2-Sided | 95 | -0.1948 | 0.3362 | Difference of the two clinical success rates (NMJ Targeted minus Current Clinical Practice). | Non-Inferiority or Equivalence (legacy) | Based on a π of 63% in the reference group, a clinically relevant Δ of 17%, α=2.5% (one sided) and power=80%, the sample size estimate yielded 122 valid subjects per group. Including a 10% addition for premature withdrawals, 136 subjects per group were necessary to be randomised in the study (i.e., a total of 272 subjects). |
| A responder was defined as a subject with at least one level decrease on the MAS scale. | Generalised linear model | 0.5369 | Based on a generalised linear model including factors for spasticity pattern. | Treatment difference | 0.0707 | 2-Sided | 95 | -0.1948 | 0.3362 | Difference of the two clinical success rates (NMJ Targeted minus Current Clinical Practice). | Non-Inferiority or Equivalence (legacy) | Based on a π of 63% in the reference group, a clinically relevant Δ of 17%, α=2.5% (one sided) and power=80%, the sample size estimate yielded 122 valid subjects per group. Including a 10% addition for premature withdrawals, 136 subjects per group were necessary to be randomised in the study (i.e., a total of 272 subjects). |
| A responder was defined as a subject with at least one level decrease on the MAS scale. | Generalised linear model | 0.7732 | Based on a generalised linear model including factors for country. | Treatment difference | 0.0707 | 2-Sided | 95 | -0.1948 | 0.3362 | Difference of the two clinical success rates (NMJ Targeted minus Current Clinical Practice). | Non-Inferiority or Equivalence (legacy) | Based on a π of 63% in the reference group, a clinically relevant Δ of 17%, α=2.5% (one sided) and power=80%, the sample size estimate yielded 122 valid subjects per group. Including a 10% addition for premature withdrawals, 136 subjects per group were necessary to be randomised in the study (i.e., a total of 272 subjects). |
| A responder was defined as a subject with at least one level decrease on the MAS scale. | Generalised linear model | 0.1758 | Based on a generalised linear model including factors for MAS at Baseline. | Treatment difference | 0.0707 | 2-Sided | 95 | -0.1948 | 0.3362 | Difference of the two clinical success rates (NMJ Targeted minus Current Clinical Practice). | Non-Inferiority or Equivalence (legacy) | Based on a π of 63% in the reference group, a clinically relevant Δ of 17%, α=2.5% (one sided) and power=80%, the sample size estimate yielded 122 valid subjects per group. Including a 10% addition for premature withdrawals, 136 subjects per group were necessary to be randomised in the study (i.e., a total of 272 subjects). |
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