Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2021-001988-24 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of this study is to determine whether a single treatment with NT 201 (botulinum toxin) is superior to placebo (no medicine) for one-sided treatment of essential tremor in the motor-dominant arm (Unilateral Period). Participants will be assigned to the treatment groups by chance and neither the participants nor the research staff who interact with them will know the allocation.
The following treatment cycle will investigate the safety and tolerability of two-sided treatment with NT 201 (botulinum toxin) (Open Label Bilateral Period). All participants will receive NT 201 treatment.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NT 201 (IncobotulinumtoxinA, Xeomin) | Experimental | Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular injection of NT 201 (130-165 units) into muscles of the motor-dominant upper limb. Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units) into muscles of both upper limbs. |
|
| Placebo | Placebo Comparator | Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular placebo injection into muscles of the motor-dominant upper limb. Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units per arm) into muscles of both upper limbs. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NT 201 | Drug | Active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins. Solution for injection prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). |
| Measure | Description | Time Frame |
|---|---|---|
| Unilateral Treatment Period: Change From Baseline to Week 6 in Maximum Tremor Amplitude of the Wrist | TremorTek kinematic analytic investigational device is combination of computer software and wearable movement sensors that allows to collect motion data when placed on individual's arm to quantify tremor in various muscle groups that impact shoulder, elbow, wrist. This assessment system was used to measure maximum angular tremor amplitude at wrist of injected limb (unit: degrees). Angular tremor amplitude was measure of tremor severity. Reduction of maximum angular tremor amplitude at wrist of injected limb represented tremor improvement. | Baseline up to Week 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Unilateral Treatment Period: Change From Baseline to Week 6 in the Essential Tremor Rating Assessment Scale (TETRAS) Performance Dominant Upper Limb (UL) Score as Assessed by Investigator | The TETRAS was a validated clinical scale for the assessment of essential tremor severity. The TETRAS performance subscale was utilized by the qualified investigators to assess the tremor severity. The TETRAS Performance dominant UL score included TETRAS Performance items 4 (including subitems a, b, c for 3 manoeuvres), 6 to 8 of dominant UL. Each individual item score ranged from 0 to 4. The performance dominant UL score ranged from 0 to 24. Higher scores indicated more severe tremor. |
Not provided
Inclusion Criteria:
- Score of ≥ 2 (at least 1 cm tremor amplitude) in at least two out of three maneuvers of test item 4 (upper limb tremor) of the TETRAS performances scale confirmed by an independent TETRAS expert by means of video assessment.
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Merz Medical Expert | Merz Pharmaceuticals GmbH | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| USF, Department of Neurology, Merz Investigational Site #0010020 | Tampa | Florida | 33612 | United States | ||
Not provided
Not provided
Not provided
Not provided
Not provided
A total of 114 subjects were screened, out of which 78 subjects were randomized, and 77 subjects were treated in this study.
Subjects were recruited at 14 investigational sites in the United States, Poland and Canada.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Unilateral Treatment Period: NT 201 | Subjects were randomized to receive unilateral intramuscular injections of NT 201 into the muscles of the motor-dominant upper limb on Day 1 of Cycle 1 (Cycle length = 24 weeks). |
| FG001 | Unilateral Treatment Period: Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Unilateral Treatment: Cycle 1 (24 Weeks) |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 31, 2021 | Apr 7, 2026 |
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Placebo | Drug | Solution for injection prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). |
|
| Baseline up to Week 6 |
| Unilateral Treatment Period: Change From Baseline to Week 6 in the TETRAS Activities of Daily Living (ADL) UL Score | TETRAS ADL subscale was another component of TETRAS. ADL subscale of TETRAS was completed by study subjects through an interview procedure to assess impact of tremor on activities of daily living. TETRAS ADL UL score was the sum of eight ADL items (on UL tremor [items 2-9]). Items were rated on a 5-point response scale each ranged from 0 (normal status/impact) to 4 (severe status/impact). The ADL UL score ranged from 0 to 32. Higher scores indicated greater tremor severity. | Baseline up to Week 6 |
| Unilateral Treatment Period: Change From Baseline to Week 6 in TETRAS ADL Functional Impact Score | TETRAS ADL Functional Impact score was the sum of the following three ADL items: occupational impairment (item 10), overall disability (item 11), and social impact (item 12) of ET. The items are rated on a 5-point response scale each ranged from 0 (normal status/impact) to 4 (severe status/impact). The ADL Functional Impact score ranged from 0 to 12. Higher scores indicated greater tremor severity. | Baseline up to Week 6 |
| Unilateral Treatment Period: Subject's Global Impression of Change Scale (GICS) Score of Motor-dominant UL at Week 6 | The GICS was used to evaluate overall clinical impression of change after treatment by the subject. The response option was a common 9-point Likert scale ranged from -4 to +4, with the following values: +4 (very much improved); +3 (much improved); +2 (improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (worse); -3 (much worse); -4 (very much worse). A higher score indicated improvements. | Week 6 |
| Unilateral Treatment Period: Investigator's GICS Score of Motor-dominant UL at Week 6 | The GICS was used to evaluate the overall clinical impression of change after treatment by the investigator. The response option was a common 9-point Likert scale ranged from -4 to +4, with the following values: +4 (very much improved); +3 (much improved); +2 (improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (worse); -3 (much worse); -4 (very much worse). A higher score indicated improvements. | Week 6 |
| Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS Performance Dominant UL Score | TETRAS was a validated clinical scale for the assessment of essential tremor severity. The TETRAS performance subscale was utilized by the qualified investigators to assess the tremor severity. The TETRAS Performance dominant UL score included TETRAS Performance items 4 ((including subitems a, b, c for 3 manoeuvres), 6 to 8 of dominant UL. Each individual item score ranged from 0 to 4. The performance dominant UL score ranged from 0 to 24. Higher scores indicated more severe tremor. | Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks) |
| Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS Performance Subscale Score | The TETRAS was a validated clinical scale for the assessment of essential tremor severity. The TETRAS performance subscale was utilized by the qualified investigators to assess the tremor severity. TETRAS performance subscale consisted of 9 items: head, face, and voice tremor (items 1-3), UL tremor of right and left UL (item 4) in three tasks (forward outstretched postural tremor, lateral "wing-beating" postural tremor, kinetic tremor), Archimedes spirals with both hands, handwriting with motor-dominant hand, and dot approximation task with both hands (items 6-8), and lower limb tremor and standing tremor (items 5 and 9). Each item was rated from 0 (no tremor) to 4 (severe tremor) with overall performance score of 0 to 64, calculated as the sum of subscale items. Higher scores indicated greater tremor severity. | Baseline of Cycle 2 up to Week 6 (Cycle length =12 weeks) |
| Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS ADL UL Score | TETRAS ADL subscale was another component of TETRAS. ADL subscale of TETRAS was completed by study subjects through an interview procedure to assess impact of tremor on activities of daily living. TETRAS ADL UL score was the sum of eight ADL items on UL tremor (items 2-9). Items were rated on a 5-point response scale each ranged from 0 (normal status/impact) to 4 (severe status/impact). The ADL UL score ranged from 0 to 32. Higher scores indicated greater tremor severity. | Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks) |
| Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS ADL Functional Impact Score | TETRAS ADL Functional Impact score was the sum of the following three ADL items: occupational impairment (item 10), overall disability (item 11), and social impact (item 12) of ET. The items are rated on a 5-point response scale each ranged from 0 (normal status/impact) to 4 (severe status/impact). The ADL Functional Impact score ranged from 0 to 12. Higher scores indicated greater tremor severity. | Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks) |
| Percentage of Subjects With At-least One Treatment Related Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) is any untoward medical occurrence in subject administered pharmaceutical product. TEAEs of unilateral treatment period (Cycle 1) = AEs from first injection up to injection in the bilateral treatment period (Week 24). TEAEs of bilateral treatment period (Cycle 2) = AEs from injection in bilateral treatment period up to end of study (Week 36). | Unilateral Treatment Period: From first injection in unilateral treatment period up to re-injection in Bilateral Treatment Period (Week 24); Bilateral Treatment Period: From re-injection in Bilateral Treatment Period at Week 24 up to end of study(Week 36) |
| University of Nebraska Medical Center, Merz Investigational Site #0010269 |
| Omaha |
| Nebraska |
| 68131 |
| United States |
| Cleveland Clinic Lou Ruvo Center for Brain Health, Merz Investigational Site #0010457 | Las Vegas | Nevada | 89106 | United States |
| Mount Sinai Medical Center, Merz Investigational Site #0010191 | New York | New York | 10029 | United States |
| Vanderbilt University Medical Center, Neuroscience Institute, Merz Investigational Site #0010206 | Nashville | Tennessee | 37232 | United States |
| Houston Methodist Neurological Institute, Merz Investigational Site #0010226 | Houston | Texas | 77030 | United States |
| Medstar Georgetown Neurology, Merz Investigational Site #0010231 | McLean | Virginia | 22101 | United States |
| UW Medical Center - Montlake, Merz Investigational Site #0010450 | Seattle | Washington | 98195 | United States |
| Selkirk Neurology, Merz Investigational Site #0010456 | Spokane | Washington | 99202 | United States |
| London Health Sciences Centre, Merz Investigational Site #0010087 | London | Ontario | N6A 5A5 | Canada |
| Specjalistyczne Gabinety, Merz Investigational Site #0480059 | Krakow | 30-539 | Poland |
| NeuroKlinika Gabinet Lekarski, Merz Investigational Site #0480101 | Lodz | 90-640 | Poland |
| Instytut Zdrowia Dr. Boczarska-Jedynak | Oświęcim | 32-600 | Poland |
| Mazowiecki Szpital Bródnowski, Merz Investigational Site #0480064 | Warsaw | 03-242 | Poland |
Subjects were randomized to receive unilateral intramuscular injections of placebo into the muscles of the motor-dominant upper limb on Day 1 of Cycle 1 (Cycle length = 24 weeks). |
| FG002 | Bilateral Treatment Period: NT 201 | Subjects who received NT201 or placebo in the unilateral treatment period, transitioned to bilateral treatment period and who were eligible for re-injection, received bilateral intramuscular injections of NT 201 into the muscles of both upper limbs on Day 1 of Cycle 2 (Cycle length = 12 weeks). |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Bilateral Treatment: Cycle 2 (12 Weeks) |
|
|
Randomized population.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Unilateral Treatment Period: NT 201 | Subjects were randomized to receive unilateral intramuscular injections of NT 201 into the muscles of the motor-dominant upper limb on Day 1 of Cycle 1 (Cycle length = 24 weeks). |
| BG001 | Unilateral Treatment Period: Placebo | Subjects were randomized to receive unilateral intramuscular injections of placebo into the muscles of the motor-dominant upper limb on Day 1 of Cycle 1 (Cycle length = 24 weeks). |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
| |||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 | Unilateral Treatment Period: Change From Baseline to Week 6 in Maximum Tremor Amplitude of the Wrist | TremorTek kinematic analytic investigational device is combination of computer software and wearable movement sensors that allows to collect motion data when placed on individual's arm to quantify tremor in various muscle groups that impact shoulder, elbow, wrist. This assessment system was used to measure maximum angular tremor amplitude at wrist of injected limb (unit: degrees). Angular tremor amplitude was measure of tremor severity. Reduction of maximum angular tremor amplitude at wrist of injected limb represented tremor improvement. | FAS-UP was subset of subjects in safety evaluation set (SES-UP) for whom at least one score of tremor amplitude at wrist level (injected UL) at study baseline and at least at one post-baseline score was available. "Overall number of participants analyzed" signifies subjects evaluable for this outcome measure. The analysis was performed by randomized treatment (one subject randomized to NT 201 was erroneously treated with placebo and one subject randomized to placebo was not treated). | Posted | Least Squares Mean | Standard Error | degrees of arc | Baseline up to Week 6 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Unilateral Treatment Period: Change From Baseline to Week 6 in the Essential Tremor Rating Assessment Scale (TETRAS) Performance Dominant Upper Limb (UL) Score as Assessed by Investigator | The TETRAS was a validated clinical scale for the assessment of essential tremor severity. The TETRAS performance subscale was utilized by the qualified investigators to assess the tremor severity. The TETRAS Performance dominant UL score included TETRAS Performance items 4 (including subitems a, b, c for 3 manoeuvres), 6 to 8 of dominant UL. Each individual item score ranged from 0 to 4. The performance dominant UL score ranged from 0 to 24. Higher scores indicated more severe tremor. | Full analysis set for unilateral treatment period (FAS-UP) was subset of subjects in SES-UP for whom at least one score of tremor amplitude at wrist level (injected UL) at study baseline and at least at one post-baseline score was available. The analysis was performed by randomized treatment (one subject randomized to NT 201 was erroneously treated with placebo and one subject randomized to placebo was not treated). | Posted | Mean | Standard Deviation | score on a scale | Baseline up to Week 6 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Unilateral Treatment Period: Change From Baseline to Week 6 in the TETRAS Activities of Daily Living (ADL) UL Score | TETRAS ADL subscale was another component of TETRAS. ADL subscale of TETRAS was completed by study subjects through an interview procedure to assess impact of tremor on activities of daily living. TETRAS ADL UL score was the sum of eight ADL items (on UL tremor [items 2-9]). Items were rated on a 5-point response scale each ranged from 0 (normal status/impact) to 4 (severe status/impact). The ADL UL score ranged from 0 to 32. Higher scores indicated greater tremor severity. | FAS-UP was subset of subjects in SES-UP for whom at least one score of tremor amplitude at wrist level (injected UL) at study baseline and at least at one post-baseline score was available. The analysis was performed by randomized treatment (one subject randomized to NT 201 was erroneously treated with placebo and one subject randomized to placebo was not treated). | Posted | Mean | Standard Deviation | score on a scale | Baseline up to Week 6 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Unilateral Treatment Period: Change From Baseline to Week 6 in TETRAS ADL Functional Impact Score | TETRAS ADL Functional Impact score was the sum of the following three ADL items: occupational impairment (item 10), overall disability (item 11), and social impact (item 12) of ET. The items are rated on a 5-point response scale each ranged from 0 (normal status/impact) to 4 (severe status/impact). The ADL Functional Impact score ranged from 0 to 12. Higher scores indicated greater tremor severity. | FAS-UP was subset of subjects in SES-UP for whom at least one score of tremor amplitude at wrist level (injected UL) at study baseline and at least at one post-baseline score was available. The analysis was performed by randomized treatment (one subject randomized to NT 201 was erroneously treated with placebo and one subject randomized to placebo was not treated). | Posted | Mean | Standard Deviation | score on a scale | Baseline up to Week 6 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Unilateral Treatment Period: Subject's Global Impression of Change Scale (GICS) Score of Motor-dominant UL at Week 6 | The GICS was used to evaluate overall clinical impression of change after treatment by the subject. The response option was a common 9-point Likert scale ranged from -4 to +4, with the following values: +4 (very much improved); +3 (much improved); +2 (improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (worse); -3 (much worse); -4 (very much worse). A higher score indicated improvements. | FAS-UP was subset of subjects in SES-UP for whom at least one score of tremor amplitude at wrist level (injected UL) at study baseline and at least at one post-baseline score was available. The analysis was performed by randomized treatment (one subject randomized to NT 201 was erroneously treated with placebo and one subject randomized to placebo was not treated). | Posted | Mean | Standard Deviation | score on a scale | Week 6 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Unilateral Treatment Period: Investigator's GICS Score of Motor-dominant UL at Week 6 | The GICS was used to evaluate the overall clinical impression of change after treatment by the investigator. The response option was a common 9-point Likert scale ranged from -4 to +4, with the following values: +4 (very much improved); +3 (much improved); +2 (improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (worse); -3 (much worse); -4 (very much worse). A higher score indicated improvements. | FAS-UP was subset of subjects in SES-UP for whom at least one score of tremor amplitude at wrist level (injected UL) at study baseline and at least at one post-baseline score was available. The analysis was performed by randomized treatment (one subject randomized to NT 201 was erroneously treated with placebo and one subject randomized to placebo was not treated). | Posted | Mean | Standard Deviation | score on a scale | Week 6 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS Performance Dominant UL Score | TETRAS was a validated clinical scale for the assessment of essential tremor severity. The TETRAS performance subscale was utilized by the qualified investigators to assess the tremor severity. The TETRAS Performance dominant UL score included TETRAS Performance items 4 ((including subitems a, b, c for 3 manoeuvres), 6 to 8 of dominant UL. Each individual item score ranged from 0 to 4. The performance dominant UL score ranged from 0 to 24. Higher scores indicated more severe tremor. | The full analysis set for the bilateral treatment period (FAS-BP) was the subset of subjects in the safety evaluation set for the bilateral treatment period (SES-BP that is, all subjects who received NT 201 during the bilateral treatment period) for whom at least one score of tremor amplitude at wrist level at the Cycle 2 baseline visit and at least at one post-baseline score. | Posted | Mean | Standard Deviation | score on a scale | Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks) |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS Performance Subscale Score | The TETRAS was a validated clinical scale for the assessment of essential tremor severity. The TETRAS performance subscale was utilized by the qualified investigators to assess the tremor severity. TETRAS performance subscale consisted of 9 items: head, face, and voice tremor (items 1-3), UL tremor of right and left UL (item 4) in three tasks (forward outstretched postural tremor, lateral "wing-beating" postural tremor, kinetic tremor), Archimedes spirals with both hands, handwriting with motor-dominant hand, and dot approximation task with both hands (items 6-8), and lower limb tremor and standing tremor (items 5 and 9). Each item was rated from 0 (no tremor) to 4 (severe tremor) with overall performance score of 0 to 64, calculated as the sum of subscale items. Higher scores indicated greater tremor severity. | The FAS-BP was the subset of subjects in the SES-BP (that is, all subjects who received NT 201 during the bilateral treatment period) for whom at least one score of tremor amplitude at wrist level at the Cycle 2 baseline visit and at least at one post-baseline score. | Posted | Mean | Standard Deviation | score on a scale | Baseline of Cycle 2 up to Week 6 (Cycle length =12 weeks) |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS ADL UL Score | TETRAS ADL subscale was another component of TETRAS. ADL subscale of TETRAS was completed by study subjects through an interview procedure to assess impact of tremor on activities of daily living. TETRAS ADL UL score was the sum of eight ADL items on UL tremor (items 2-9). Items were rated on a 5-point response scale each ranged from 0 (normal status/impact) to 4 (severe status/impact). The ADL UL score ranged from 0 to 32. Higher scores indicated greater tremor severity. | The FAS-BP was the subset of subjects in the SES-BP (that is, all subjects who received NT 201 during the bilateral treatment period) for whom at least one score of tremor amplitude at wrist level at the Cycle 2 baseline visit and at least at one post-baseline score. | Posted | Mean | Standard Error | score on a scale | Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks) |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Bilateral Treatment Period: Change From Cycle 2 Baseline to Week 6 in TETRAS ADL Functional Impact Score | TETRAS ADL Functional Impact score was the sum of the following three ADL items: occupational impairment (item 10), overall disability (item 11), and social impact (item 12) of ET. The items are rated on a 5-point response scale each ranged from 0 (normal status/impact) to 4 (severe status/impact). The ADL Functional Impact score ranged from 0 to 12. Higher scores indicated greater tremor severity. | The FAS-BP was the subset of subjects in the SES-BP (that is, all subjects who received NT 201 during the bilateral treatment period) for whom at least one score of tremor amplitude at wrist level at the Cycle 2 baseline visit and at least at one post-baseline score. | Posted | Mean | Standard Deviation | score on a scale | Baseline of Cycle 2 up to Week 6 (Cycle length = 12 weeks) |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Subjects With At-least One Treatment Related Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) is any untoward medical occurrence in subject administered pharmaceutical product. TEAEs of unilateral treatment period (Cycle 1) = AEs from first injection up to injection in the bilateral treatment period (Week 24). TEAEs of bilateral treatment period (Cycle 2) = AEs from injection in bilateral treatment period up to end of study (Week 36). | The safety evaluation set for unilateral treatment period (SES-UP) included all subjects who received NT 201 or placebo during the unilateral treatment period and SES-BP included all subjects who received NT 201 during the bilateral treatment period. For the unilateral treatment period, the analysis performed according to actual treatment (one subject was treated with placebo even so randomized to NT 201 and one subject was not treated even so randomized to placebo). | Posted | Number | percentage of subjects | Unilateral Treatment Period: From first injection in unilateral treatment period up to re-injection in Bilateral Treatment Period (Week 24); Bilateral Treatment Period: From re-injection in Bilateral Treatment Period at Week 24 up to end of study(Week 36) |
|
Unilateral Treatment Period: From first injection in unilateral treatment period up to re-injection in Bilateral Treatment Period (Week 24); Bilateral Treatment Period: From re-injection in Bilateral Treatment Period at Week 24 up to end of study (Week 36)
Not provided
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 | Unilateral Treatment Period: NT 201 | Subjects were randomized to receive unilateral intramuscular injections of NT 201 into the muscles of the motor-dominant upper limb on Day 1 of Cycle 1 (Cycle length = 24 weeks). | 0 | 50 | 3 | 50 | 29 | 50 |
| EG001 | Unilateral Treatment Period: Placebo | Subjects were randomized to receive unilateral intramuscular injections of placebo into the muscles of the motor-dominant upper limb on Day 1 of Cycle 1 (Cycle length = 24 weeks). | 0 | 27 | 2 | 27 | 5 | 27 |
| EG002 | Bilateral Treatment Period: NT 201 | Subjects who received NT201 or placebo in the unilateral treatment period, transitioned to bilateral treatment period and who were eligible for re-injection, received bilateral intramuscular injections of NT 201 into the muscles of both upper limbs on Day 1 of Cycle 2 (Cycle length = 12 weeks). | 0 | 72 | 2 | 72 | 29 | 72 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bladder cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.1 | Systematic Assessment |
| |
| Papillary thyroid cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.1 | Systematic Assessment |
| |
| Skull fracture | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Subarachnoid haemorrhage | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Deafness unilateral | Ear and labyrinth disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.1 | Systematic Assessment |
| |
| Metastases to lung | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.1 | Systematic Assessment |
| |
| Rectal neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Essential hypertension | Vascular disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Peripheral venous disease | Vascular disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site haemorrhage | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Inflammation | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site nodule | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Negative thoughts | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Craniofacial fracture | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Craniofacial injury | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Post-traumatic pain | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Type V hyperlipidaemia | Congenital, familial and genetic disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Hypertensive heart disease | Cardiac disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cervical radiculopathy | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Facial paralysis | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Fine motor skill dysfunction | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Monoparesis | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Paresis | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Peritoneal adhesions | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Chronic gastritis | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Retching | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Spinal osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Intervertebral disc disorder | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Muscle fatigue | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Coccydynia | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Helicobacter infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
|
Publication of study information usually requires agreement with the sponsor. In case of justified doubts by the sponsor, the INVESTIGATOR will consider these doubts in the publication as long as the scientific neutrality is not affected.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Public Disclosure Manager | Merz Therapeutics GmbH | +49 69 1503 1 | clinicaltrials@merz.de |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 24, 2024 | Apr 7, 2026 | SAP_001.pdf |
| ID | Term |
|---|---|
| C545476 | incobotulinumtoxinA |
| D019274 | Botulinum Toxins, Type A |
| 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
| Title | Measurements |
|---|---|
|
| >= 85 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
Subjects were randomized to receive unilateral intramuscular injections of placebo into the muscles of the motor-dominant upper limb on Day 1 of Cycle 1 (Cycle length = 24 weeks).
|
|
|
|
|
|
|
|
|
|
| Counts |
|---|
| Participants |
|
|
|
|
|
|
|
Subjects were randomized to receive unilateral intramuscular injections of placebo into the muscles of the motor-dominant upper limb on Day 1 of Cycle 1 (Cycle length = 24 weeks). |
| OG002 | Bilateral Treatment Period: NT 201 | Subjects who received NT201 or placebo in the unilateral treatment period, transitioned to bilateral treatment period and who were eligible for re-injection, received bilateral intramuscular injections of NT 201 into the muscles of both upper limbs on Day 1 of Cycle 2 (Cycle length = 12 weeks). |
|
|