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Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adults (OPTIMYST)

Primary Purpose

Sialorrhea

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

MYOBLOC

About this trial

This is an interventional treatment trial for Sialorrhea focused on measuring Cerebral Palsy, ALS, Stroke, Neuroleptics

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Seeking treatment for troublesome sialorrhea for at least 3 months that is occurring secondary to any disorder or related to any cause, including, but not limited to, Parkinson's Disease (PD), adult cerebral palsy, amyotrophic lateral sclerosis (ALS), stroke, traumatic brain injury, oral cancer, and side effects of other medications.
Able to read and provide written informed consent before enrollment into the study, or the subject's caregiver (Legally Authorized Representative) can provide written informed consent.
Male or female, 18 to 85 years of age (inclusive).
Minimum unstimulated salivary flow rate of 0.2 g/min at screening
Minimum Investigator's Drooling Frequency and Severity Scale (DFSS) score of 4 at screening.
Ability and availability to participate in the study for up to 1 year (ALS subjects: ability and availability to participate in the study for at least 6 months), based on overall health of the subject and disease prognosis

Exclusion Criteria:

A moderate to high risk of aspiration will exclude participation in this study. Subjects whose risk of aspiration are judged by the Investigator to be satisfactorily controlled by placement of PEG tube or G-tube for nutritional support are eligible to participate.
Respiratory forced vital capacity (FVC) of <20% of predicted
Prior botulinum toxin type A or B treatment in the salivary gland(s) identified for treatment in this study within 24 weeks before screening. Prior botulinum toxin type A or B treatment into other anatomical regions not selected for treatment in this study is not exclusionary, but must have occurred at least 12 weeks before screening
Subjects should be excluded if, in the Investigator's opinion, the subject failed to respond to previous treatment with botulinum toxin. Subjects should not receive nor have any plans for receiving any botulinum toxin treatment, other than the study drug (MYOBLOC), during the entire course of the study (from the point the informed consent is signed until subject's participation is complete).
Concomitant use, or exposure within 5 half-lives of screening, of aminoglycoside antibiotics, curare-like agents, or other agents that interfere with neuromuscular function
Prior salivary gland surgery
Current treatment or treatment at any time during the study with Coumadin® (warfarin) or similar anti-coagulant medications. Anti-platelet medications are not specifically exclusionary
Evidence of any clinically significant neurologic disease
Pregnancy or lactation
Anticipated or scheduled surgery during the study period. A PEG tube/G tube may be placed for nutritional support at any time during the study and will not exclude the subject from continued study participation.
Major surgery (requiring general anesthesia, except PEG tube/G tube placement ) within the previous 6 months before screening.
Current infection at the sialorrhea treatment injection site(s)
History of drug or alcohol abuse currently or within the previous 6 months
Participation in another clinical drug, device, or biological agent study within 30 days of screening or while participating in this study

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

MYOBLOC Injection

Arm Description

After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections over the course of 1 year.

Outcomes

Primary Outcome Measures

Occurrence, Seriousness, Severity, and Causality Assessment of Treatment Emergent Adverse Events (TEAE)

TEAEs Related to Study Medication include TEAEs classified as Possibly, Probably and Definitely Related. Treatment Session (TS)

Occurrence of Adverse Events of Special Interest (AESI)

Treatment Session (TS) AESI includes: Aspiration, Aspirational pneumonia, Choking and Dysphagia.

Columbia Suicide Rating Scale (C-SSRS): Suicidal Ideation, Behavior Scores and Intensity Scores

Participants Analyzed does not match the Participant Flow because one participant discontinued prior to the first post-injection visit. Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U).

Occurrence of Dental Adverse Events

Treatment Session (TS)

Secondary Outcome Measures

Change From Baseline in Unstimulated Salivary Flow Rate (USFR): Treatment Sessions 1-4

For Treatment Sessions 2-4, the change from baseline is calculated using the Week 13 value from the previous Treatment Session as the new baseline. Week 8 was only assessed in Treatment Session 1. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit.

Change From Baseline in Clinical Global Impression of Severity (CGI-S)

CGI-S will be used to record the severity of illness (sialorrhea) on a 7-point scale ranging from a score of 1, "normal" to a score of 7, "among the most extremely ill patients". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit.

Clinical Global Impression of Change (CGI-C)

CGI-C will be used to record change or improvement of illness (sialorrhea) on a 7-point scale ranging from a score of 1, "very much improved" to a score of 7, "very much worse". Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. Treatment Session (TS)

Change From Baseline in Drooling Frequency and Severity Scale Performed by Trained Assessor (DFSS-I)

The DFSS will be used to assess the frequency and severity of drooling on a 5-point scale ranging from a score of 1, dry never drools to a score of 5, profuse clothing hands tray objects become wet. Drooling frequency will be assessed on a 4-point scale ranging from a score of 1, never drools to a score of 4, constantly drools. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. The severity score and frequency score are summed together to create the DFSS total score (range 2-9). A decrease from baseline indicates improvement.

Change From Baseline in Drooling Frequency and Severity Scale Performed by Subject (DFSS-S)

The DFSS will be used to assess the frequency and severity of drooling on a 5-point scale ranging from a score of 1, dry never drools to a score of 5, profuse clothing hands tray objects become wet. Drooling frequency will be assessed on a 4-point scale ranging from a score of 1, never drools to a score of 4, constantly drools. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit. The severity score and frequency score are summed together to create the DFSS total score (range 2 -9). A decrease from baseline indicates improvement.

Change From Baseline in Patient Global Impression of Severity (PGI-S)

The PGI-S scale will be used by the subject to rate the severity of his/her illness (sialorrhea) on an 7-point scale ranging from a score of 1, "normal" to a score of 7, "among the most extremely ill". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit.

Patient Global Impression of Change (PGI-C)

The PGI-C scale will be used by the subject to rate the change in symptoms of his/her illness (sialorrhea) on an 7-point scale ranging from a score of 1, "very much improved" to a score of 7, "very much worse". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit.

Change From Baseline in Drooling Impact Score (DIS)

The DIS is a 10-item questionnaire with each item rated by the subject to evaluate the impact of sialorrhea on daily activities (e.g., speech, social activities). Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit. Subject is requested to compare their current status to their last study visit using a 4-point scale ranging from a score of 1, not at all to a score of 4, very much. The DIS total score ranges from 10 to 40. A negative change from baseline indicates a positive clinical outcome.

Full Information

NCT ID

NCT02610868

First Posted

November 2, 2015

Last Updated

July 9, 2021

Sponsor

Supernus Pharmaceuticals, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02610868

Brief Title

Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adults

Acronym

OPTIMYST

Official Title

A Phase 3, Long-Term, Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects

Study Type

Interventional

2. Study Status

Record Verification Date

November 2019

Overall Recruitment Status

Completed

Study Start Date

October 2015 (undefined)

Primary Completion Date

June 2017 (Actual)

Study Completion Date

June 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Supernus Pharmaceuticals, Inc.

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

Multicenter, open-label, outpatient study of the safety and effectiveness of repeated doses of MYOBLOC over a 1-year duration in adult subjects with troublesome sialorrhea.

Detailed Description

Multicenter, open-label, outpatient study of the safety and effectiveness of repeated doses of MYOBLOC over a 1-year duration in adult subjects with troublesome sialorrhea. The primary goal is to determine the long-term safety and tolerability of MYOBLOC (administered intraglandularly as single total dose of 3,500 Units) treatments every 13 weeks over a maximum possible duration of 1 year in adult subjects with troublesome sialorrhea. The secondary goal is to assess the magnitude of therapeutic response of MYOBLOC (administered intraglandularly as a single total dose of 3,500 Units) using effectiveness assessments performed at intervals after treatments every 13 weeks over a maximum possible duration of 1 year.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sialorrhea

Keywords

Cerebral Palsy, ALS, Stroke, Neuroleptics

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

187 (Actual)

8. Arms, Groups, and Interventions

Arm Title

MYOBLOC Injection

Arm Type

Experimental

Arm Description

After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections over the course of 1 year.

Intervention Type

Drug

Intervention Name(s)

MYOBLOC

Other Intervention Name(s)

rimabotulinumtoxinB, botulinum toxin type B

Intervention Description

After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments via injections into the submandibular and parotid glands. Subjects will undergo safety and effectiveness assessments at Weeks 4, 8, 13, 17, 26, 30, 39, 43, and 52, and telephone follow up will occur at Weeks 21, 34, and 47

Primary Outcome Measure Information:

Title

Occurrence, Seriousness, Severity, and Causality Assessment of Treatment Emergent Adverse Events (TEAE)

Description

TEAEs Related to Study Medication include TEAEs classified as Possibly, Probably and Definitely Related. Treatment Session (TS)

Time Frame

Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1

Title

Occurrence of Adverse Events of Special Interest (AESI)

Description

Treatment Session (TS) AESI includes: Aspiration, Aspirational pneumonia, Choking and Dysphagia.

Time Frame

Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1

Title

Columbia Suicide Rating Scale (C-SSRS): Suicidal Ideation, Behavior Scores and Intensity Scores

Description

Time Frame

Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Title

Occurrence of Dental Adverse Events

Description

Treatment Session (TS)

Time Frame

Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1

Secondary Outcome Measure Information:

Title

Change From Baseline in Unstimulated Salivary Flow Rate (USFR): Treatment Sessions 1-4

Description

Time Frame

Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Title

Change From Baseline in Clinical Global Impression of Severity (CGI-S)

Description

Time Frame

Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Title

Clinical Global Impression of Change (CGI-C)

Description

Time Frame

TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Title

Change From Baseline in Drooling Frequency and Severity Scale Performed by Trained Assessor (DFSS-I)

Description

Time Frame

Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Title

Change From Baseline in Drooling Frequency and Severity Scale Performed by Subject (DFSS-S)

Description

The DFSS will be used to assess the frequency and severity of drooling on a 5-point scale ranging from a score of 1, dry never drools to a score of 5, profuse clothing hands tray objects become wet. Drooling frequency will be assessed on a 4-point scale ranging from a score of 1, never drools to a score of 4, constantly drools. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit. The severity score and frequency score are summed together to create the DFSS total score (range 2 -9). A decrease from baseline indicates improvement.

Time Frame

Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Title

Change From Baseline in Patient Global Impression of Severity (PGI-S)

Description

Time Frame

Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Title

Patient Global Impression of Change (PGI-C)

Description

Time Frame

TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Title

Change From Baseline in Drooling Impact Score (DIS)

Description

Time Frame

Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

85 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Seeking treatment for troublesome sialorrhea for at least 3 months that is occurring secondary to any disorder or related to any cause, including, but not limited to, Parkinson's Disease (PD), adult cerebral palsy, amyotrophic lateral sclerosis (ALS), stroke, traumatic brain injury, oral cancer, and side effects of other medications. Able to read and provide written informed consent before enrollment into the study, or the subject's caregiver (Legally Authorized Representative) can provide written informed consent. Male or female, 18 to 85 years of age (inclusive). Minimum unstimulated salivary flow rate of 0.2 g/min at screening Minimum Investigator's Drooling Frequency and Severity Scale (DFSS) score of 4 at screening. Ability and availability to participate in the study for up to 1 year (ALS subjects: ability and availability to participate in the study for at least 6 months), based on overall health of the subject and disease prognosis Exclusion Criteria: A moderate to high risk of aspiration will exclude participation in this study. Subjects whose risk of aspiration are judged by the Investigator to be satisfactorily controlled by placement of PEG tube or G-tube for nutritional support are eligible to participate. Respiratory forced vital capacity (FVC) of <20% of predicted Prior botulinum toxin type A or B treatment in the salivary gland(s) identified for treatment in this study within 24 weeks before screening. Prior botulinum toxin type A or B treatment into other anatomical regions not selected for treatment in this study is not exclusionary, but must have occurred at least 12 weeks before screening Subjects should be excluded if, in the Investigator's opinion, the subject failed to respond to previous treatment with botulinum toxin. Subjects should not receive nor have any plans for receiving any botulinum toxin treatment, other than the study drug (MYOBLOC), during the entire course of the study (from the point the informed consent is signed until subject's participation is complete). Concomitant use, or exposure within 5 half-lives of screening, of aminoglycoside antibiotics, curare-like agents, or other agents that interfere with neuromuscular function Prior salivary gland surgery Current treatment or treatment at any time during the study with Coumadin® (warfarin) or similar anti-coagulant medications. Anti-platelet medications are not specifically exclusionary Evidence of any clinically significant neurologic disease Pregnancy or lactation Anticipated or scheduled surgery during the study period. A PEG tube/G tube may be placed for nutritional support at any time during the study and will not exclude the subject from continued study participation. Major surgery (requiring general anesthesia, except PEG tube/G tube placement ) within the previous 6 months before screening. Current infection at the sialorrhea treatment injection site(s) History of drug or alcohol abuse currently or within the previous 6 months Participation in another clinical drug, device, or biological agent study within 30 days of screening or while participating in this study

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Najeebah Abdul-Musawir, MD,MBA

Organizational Affiliation

Supernus Pharmaceuticals, Inc.

Official's Role

Study Director

Facility Information:

City

Phoenix

State/Province

Arizona

ZIP/Postal Code

85013

Country

United States

City

Scottsdale

State/Province

Arizona

ZIP/Postal Code

85258

Country

United States

City

Scottsdale

State/Province

Arizona

ZIP/Postal Code

85259

Country

United States

City

Loma Linda

State/Province

California

ZIP/Postal Code

92354

Country

United States

City

Los Angeles

State/Province

California

ZIP/Postal Code

90033

Country

United States

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

City

Atlantis

State/Province

Florida

ZIP/Postal Code

33462

Country

United States

City

Boca Raton

State/Province

Florida

ZIP/Postal Code

33486

Country

United States

City

Orlando

State/Province

Florida

ZIP/Postal Code

32806

Country

United States

City

Port Charlotte

State/Province

Florida

ZIP/Postal Code

33980

Country

United States

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30329

Country

United States

City

Kansas City

State/Province

Kansas

ZIP/Postal Code

66160

Country

United States

City

Overland Park

State/Province

Kansas

ZIP/Postal Code

66211

Country

United States

City

Elkridge

State/Province

Maryland

ZIP/Postal Code

21075

Country

United States

City

Farmington Hills

State/Province

Michigan

ZIP/Postal Code

48334

Country

United States

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

City

Las Vegas

State/Province

Nevada

ZIP/Postal Code

89145

Country

United States

City

Edison

State/Province

New Jersey

ZIP/Postal Code

08818

Country

United States

City

Centerville

State/Province

Ohio

ZIP/Postal Code

45459

Country

United States

City

Tulsa

State/Province

Oklahoma

ZIP/Postal Code

74136

Country

United States

City

Greenville

State/Province

South Carolina

ZIP/Postal Code

29615

Country

United States

City

Cordova

State/Province

Tennessee

ZIP/Postal Code

38018

Country

United States

City

San Antonio

State/Province

Texas

ZIP/Postal Code

78229

Country

United States

City

Kirkland

State/Province

Washington

ZIP/Postal Code

98034

Country

United States

City

Spokane

State/Province

Washington

ZIP/Postal Code

99202

Country

United States

City

Tacoma

State/Province

Washington

ZIP/Postal Code

98409

Country

United States

City

Grodno

ZIP/Postal Code

230017

Country

Belarus

City

Dnipropetrovsk

ZIP/Postal Code

49027

Country

Ukraine

City

Ivano-Frankivsk

ZIP/Postal Code

76008

Country

Ukraine

City

Kharkiv

ZIP/Postal Code

61068

Country

Ukraine

City

Lviv

ZIP/Postal Code

79010

Country

Ukraine

City

Odesa

ZIP/Postal Code

65025

Country

Ukraine

City

Rivne

ZIP/Postal Code

33010

Country

Ukraine

City

Uzhgorod

ZIP/Postal Code

88018

Country

Ukraine

City

Zaporizhzhya

ZIP/Postal Code

69600

Country

Ukraine

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adults

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