Efficacy and Safety Study of Botulinum Toxin Type A Against Placebo to Treat Abnormal Contraction or Twitch of the Eyelid
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT01896895 |
|
Recruitment Status :
Completed
First Posted : July 11, 2013
Results First Posted : March 1, 2018
Last Update Posted : March 1, 2018
|
Sponsor:
Merz Pharmaceuticals GmbH
Information provided by (Responsible Party):
Merz Pharmaceuticals GmbH
- Study Details
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
Brief Summary:
This phase 3 study will serve to collect efficacy and safety data of two different doses of NT 201 in subjects suffering from Bilateral Blepharospasm (BEB) who are BTX treatment-naïve.
In this study, BTX treatment-naïve subjects are defined as those who have not received BTX treatment within the last 12 months for the treatment of BEB. This definition aims to avoid bias by comparison of treatment effects in the subject's assessments. Furthermore, this study will substantiate the existing efficacy and safety database for the indication BEB.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Bilateral Blepharospasm (BEB) | Drug: IncobulinumtoxinA (Xeomin), 25 Units Drug: IncobotulinumtoxinA (Xeomin), 12.5 Units Drug: Placebo Drug: IncobotulinumtoxinA (Xeomin), 35 Units | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 61 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | Prospective, Double-blind, Placebo-controlled, Randomized, Parallel-group, Multi-center Study With an Open-label Extension Period to Investigate the Efficacy and Safety of Two Different Doses of NT 201 in Botulinum Toxin Treatment-naïve Subjects With Blepharospasm |
| Study Start Date : | November 2013 |
| Actual Primary Completion Date : | April 2016 |
| Actual Study Completion Date : | November 2016 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Benign essential blepharospasm
| Arm | Intervention/treatment |
|---|---|
|
Experimental: IncobotulinumtoxinA (Xeomin) 25U per eye
Main Period: one injection session, 25 Units per eye. Open-Label Extension: one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection.
|
Drug: IncobulinumtoxinA (Xeomin), 25 Units
IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Main Period: 25 Units per eye.
Other Names:
Drug: IncobotulinumtoxinA (Xeomin), 35 Units IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Open-Label Extension: up to 35 Units per eye. Other Names:
|
|
Experimental: IncobotulinumtoxinA (Xeomin) 12.5U per eye
Main Period: one injection session, 12.5 Units per eye. Open-Label Extension Period: one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection.
|
Drug: IncobotulinumtoxinA (Xeomin), 12.5 Units
IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Main Period: 12.5 Units per eye.
Other Names:
Drug: IncobotulinumtoxinA (Xeomin), 35 Units IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Open-Label Extension: up to 35 Units per eye. Other Names:
|
|
Placebo Comparator: Placebo
Main Period: Placebo to IncobotulinumtoxinA (Xeomin)(12.5 or 25U/eye), one injection session. Open-Label Extension: IncobotulinumtoxinA (Xeomin), one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection. |
Drug: Placebo
Main Period: Placebo to IncobotulinumtoxinA (Xeomin), powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Drug: IncobotulinumtoxinA (Xeomin), 35 Units IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Open-Label Extension: up to 35 Units per eye. Other Names:
|
Primary Outcome Measures :
- Double-blind MP: Change From Baseline in JRS Severity Subscore at Day 43 (Visit 4) [ Time Frame: Baseline, Day 43 (Visit 4) ]JRS severity subscore was used to classify individual symptoms of blepharospasm and to determine therapeutic efficacy. JRS severity subscore ranges from 0 to 4, where 0: None; 1: increased blinking present with external stimuli; 2: Mild but spontaneous eyelid fluttering, definitely noticeable, possibly embarrassing, but not functionally disabling, 3: Moderate, very noticeable spasm of eyelids only, mildly incapacitating, 4: Severe, incapacitating spasm of eyelids and possibly other facial muscles. Values represent least square (LS) mean differences between baseline and visit 4 resulting from analysis of covariance (ANCOVA) with treatment group, pooled site, and gender as fixed factors and baseline JRS severity subscore and age as covariates and missings replaced using the last observation carried forward (LOCF) method. Negative values denote improvement, while positive values denote deterioration vs. baseline.
Secondary Outcome Measures :
- Double-blind MP: Change From Baseline in Blepharospasm Disability Index (BSDI) at Day 43 (Visit 4) [ Time Frame: Baseline, Day 43 (Visit 4) ]BSDI is a scale for assessment of impairment of specific activities of daily living caused by blepharospasm. BSDI consists of six items (driving a vehicle; reading; watching TV; shopping; getting about on foot (walking); doing everyday activities), each ranging from 0 (=no impairment) to 4 (=no longer possible due to illness). The BSDI total score is a mean score for non-missing items ranging from 0 to 4. It is calculated by adding scores of all applicable and answered items, and dividing the resulting sum by the number of items answered. Outcome values represent LS mean differences between baseline and visit 4 (visit 4 value minus baseline value) resulting from ANCOVA with treatment group, pooled site, gender as fixed factors and baseline BSDI total score, age as covariates. Missings were replaced by the LOCF method. Negative values denote an improvement, while positive values denote deterioration vs. baseline.
- Double-blind MP: Patient Evaluation of Global Response (PEGR) at Final Visit (Day 43-Day 141) [ Time Frame: Baseline, Final Visit (Day 43-Day 141) ]PEGR scale is a descriptive subjective 9-point response self-rating scale ranging from "complete abolishment of signs and symptoms" (value=+4) down to "very marked worsening" (value=-4). Outcome values represent least square means at visit 4 resulting from an ANCOVA with treatment group, pooled site, gender as fixed factors and age as covariates. Missing were set to a zero effect (value=0). Positive values denote an improvement, while negative values denote deterioration.
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 80 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Male or female out-patients age ≥ 18 and ≤ 80 years.
- A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of orbicular oculi muscles.
- A need for injection of BTX defined as a Jankovic Rating Scale [JRS] severity subscore ≥ 2.
- Treatment-naïve subject defined as at least 12 months without BTX of any serotype for the treatment of BEB before administration of IP.
Exclusion Criteria:
- Subject with any previous unsuccessful treatment with BTX of any serotype for the treatment of BEB.
- Atypical variant of BEB (e.g., apraxia of the eyelid opening) caused by inhibition of levator palpebrae muscle.
- Neuroleptic-induced blepharospasm.
- Myotomy or denervation surgery in the affected muscles (e.g., peripheral denervation, spinal cord stimulation) and surgery in the upper face.
- Generalized disorders of muscles activity (e.g., myasthenia gravis in particular ocularis, Lambert-Eaton-Syndrome, amyotrophic lateral sclerosis) or any other significant neuromuscular dysfunction which might interfere with the study.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01896895
Locations
| Greece | |
| Merz Investigational Site #030002 | |
| Athens, Greece, 11521 | |
| Merz Investigational Site #030001 | |
| Athens, Greece, 11526 | |
| Malaysia | |
| Merz Investigational Site #060007 | |
| Georgetown, Penang, Malaysia, 10990 | |
| Merz Investigational Site #060004 | |
| Kota Kinabalu, Sabah, Malaysia, 88586 | |
| Merz Investigational Site #060006 | |
| Kuala Lumpur, Malaysia, 50586 | |
| Merz Investigational Site #060002 | |
| Kuala Lumpur, Malaysia, 56000 | |
| Merz Investigational Site #060003 | |
| Selangor, Malaysia, 43000 | |
| Sri Lanka | |
| Merz Investigational Site #094001 | |
| Colombo, Sri Lanka, 07 | |
| Merz Investigational Site #094005 | |
| Colombo, Sri Lanka, 10350 | |
| Merz Investigational Site #094006 | |
| Kurunegala, Sri Lanka, 60000 | |
| Merz Investigational Site #094002 | |
| Nugegoda, Sri Lanka, 10250 | |
Sponsors and Collaborators
Merz Pharmaceuticals GmbH
Investigators
| Study Director: | Merz Medical Expert | Merz Pharmaceuticals GmbH |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Merz Pharmaceuticals GmbH |
| ClinicalTrials.gov Identifier: | NCT01896895 |
| Other Study ID Numbers: |
MRZ60201_3074_1 2012-004821-26 ( EudraCT Number ) |
| First Posted: | July 11, 2013 Key Record Dates |
| Results First Posted: | March 1, 2018 |
| Last Update Posted: | March 1, 2018 |
| Last Verified: | January 2018 |
Additional relevant MeSH terms:
|
Blepharospasm Eyelid Diseases Eye Diseases Botulinum Toxins Botulinum Toxins, Type A abobotulinumtoxinA incobotulinumtoxinA Acetylcholine Release Inhibitors |
Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Cholinergic Agents Neurotransmitter Agents Physiological Effects of Drugs Neuromuscular Agents Peripheral Nervous System Agents |