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Efficacy and Safety Study of MYOBLOC® Followed by Open-Label Multiple-Treatment With MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects (MYSTICOL)

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ClinicalTrials.gov Identifier: NCT01994109
Recruitment Status : Completed
First Posted : November 25, 2013
Results First Posted : August 15, 2019
Last Update Posted : July 13, 2021
Sponsor:
Information provided by (Responsible Party):
Supernus Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Sialorrhea
Interventions Drug: MYOBLOC
Other: PLACEBO
Enrollment 187
Recruitment Details The study was conducted at 33 sites including United States, Russia and Ukraine.
Pre-assignment Details DB: Double Blind (Part A) OL: Open Label (Part B)
Arm/Group Title DB: MYOBLOC 2500 U DB: MYOBLOC 3500 U DB: Placebo OL: ALL MYOBLOC
Hide Arm/Group Description Participants will receive specified dose of MYOBLOC Participants will receive specified dose of MYOBLOC Participants will receive volume matched Placebo All Participants were to receive 3500 U MYOBLOC but could receive lower doses at investigator discretion.
Period Title: Double-blind Phase (Part A)
Started 63 64 60 0
Completed 62 61 53 0
Not Completed 1 3 7 0
Period Title: Open-label Phase (Part B)
Started 0 0 0 [1] 170 [2]
Completed 0 0 0 [1] 128
Not Completed 0 0 0 42
[1]
No placebo group in Part B.
[2]
61 from 2500 U group, 57 from 3500 U group, 52 from Placebo group continued to Part B
Arm/Group Title MYOBLOC 2500 U MYOBLOC 3500 U PLACEBO Total
Hide Arm/Group Description Subjects were dosed per treatment assignment of 2500 U MYOBLOC. Subjects were dosed per treatment assignment of 3500 U MYOBLOC. Placebo was dosed as an exact match of MYOBLOC. Total of all reporting groups
Overall Number of Baseline Participants 63 64 57 184
Hide Baseline Analysis Population Description
Demographic Characteristics of intent-to-treat (ITT) Population.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants 64 participants 57 participants 184 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
29
  46.0%
25
  39.1%
24
  42.1%
78
  42.4%
>=65 years
34
  54.0%
39
  60.9%
33
  57.9%
106
  57.6%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 63 participants 64 participants 57 participants 184 participants
62.6  (13.0) 64.6  (14.0) 64.1  (13.1) 63.8  (13.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants 64 participants 57 participants 184 participants
Female
15
  23.8%
9
  14.1%
16
  28.1%
40
  21.7%
Male
48
  76.2%
55
  85.9%
41
  71.9%
144
  78.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants 64 participants 57 participants 184 participants
Hispanic or Latino
5
   7.9%
2
   3.1%
1
   1.8%
8
   4.3%
Not Hispanic or Latino
58
  92.1%
62
  96.9%
56
  98.2%
176
  95.7%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 63 participants 64 participants 57 participants 184 participants
American Indian or Alaska Native
0
   0.0%
1
   1.6%
0
   0.0%
1
   0.5%
Asian
1
   1.6%
0
   0.0%
1
   1.8%
2
   1.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
   3.2%
1
   1.6%
1
   1.8%
4
   2.2%
White
60
  95.2%
60
  93.8%
55
  96.5%
175
  95.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
2
   3.1%
0
   0.0%
2
   1.1%
1.Primary Outcome
Title Unstimulated Salivary Flow Rate (USFR) at Week 4 Post-injection Visit (Part A)
Hide Description Change weight of expectorated saliva at a Week 4 post-injection visit.
Time Frame 4 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The per protocol included subjects with a minimum USFR of 0.2 g/min.
Arm/Group Title MYOBLOC 2500 U MYOBLOC 3500 U Placebo
Hide Arm/Group Description:
Subjects were dosed per treatment assignment of 2500 U MYOBLOC.
Subjects were dosed per treatment assignment of 3500 U MYOBLOC
Placebo was dosed as an exact match of MYOBLOC.
Overall Number of Participants Analyzed 63 64 57
Least Squares Mean (Standard Error)
Unit of Measure: g/minute
-0.37  (0.03) -0.36  (0.03) -0.07  (0.03)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MYOBLOC 3500 U, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection MYOBLOC 2500 U, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
2.Primary Outcome
Title Clinical Global Impression Change (CGI-C) at Week 4 Post-injection (Part A)
Hide Description CGI-C was assessed on a 7-point scale ranging from "very much improved" to "very much worse" with 1 assigned to "very much improved" and 7 assigned to "very much worse"; ranging from a minimum score of 1 and a maximum score of 7.
Time Frame 4 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Per protocol the intent-to-treat population who were injected with study medication and had at least 1 post injection CGI-C measurement up to week 4 (inclusive).
Arm/Group Title MYOBLOC 2500 U MYOBLOC 3500 U Placebo
Hide Arm/Group Description:
Subjects were dosed per treatment assignment of 2500 U MYOBLOC.
Subjects were dosed per treatment assignment of 3500 U MYOBLOC.
Placebo was dosed as an exact match of MYOBLOC.
Overall Number of Participants Analyzed 63 64 60
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
2.38  (0.12) 2.45  (0.12) 3.59  (0.13)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MYOBLOC 3500 U, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection MYOBLOC 2500 U, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Time Frame Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods
Adverse Event Reporting Description Part A (Session 1): Serious TEAEs Reported During Double-Blind Phase (Safety Population) Part B (Sessions 2-5): Serious TEAEs Reported During Open-Label Phase (OL Population)
 
Arm/Group Title MYOBLOC 2500 U (Part A) MYOBLOC 3500 U (Part A) Placebo (Part A) OL: ALL MYOBLOC (Part B)
Hide Arm/Group Description Participants received specified dose of MYOBLOC. Participants were evaluated for 13 weeks following the injection. Participants received specified dose of MYOBLOC. Participants were evaluated for 13 weeks following the injection. Participants received volume matched Placebo. Participants were evaluated for 13 weeks following the injection. Participants received Open Label treatment with 3500 U (decreased dose at investigator discretion) every 13 weeks for a maximum of 4 treatment sessions.
All-Cause Mortality
MYOBLOC 2500 U (Part A) MYOBLOC 3500 U (Part A) Placebo (Part A) OL: ALL MYOBLOC (Part B)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/63 (0.00%)   2/64 (3.13%)   1/60 (1.67%)   9/170 (5.29%) 
Hide Serious Adverse Events
MYOBLOC 2500 U (Part A) MYOBLOC 3500 U (Part A) Placebo (Part A) OL: ALL MYOBLOC (Part B)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/63 (1.59%)   4/64 (6.25%)   4/60 (6.67%)   32/170 (18.82%) 
Cardiac disorders         
Cardio-respiratory arrest   0/63 (0.00%)  1/64 (1.56%)  0/60 (0.00%)  2/170 (1.18%) 
Atrial fibrillaiton   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Cardiac arrest   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Eye disorders         
Visual acuity reduced   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Gastrointestinal disorders         
Small intestinal obstruction   0/63 (0.00%)  0/64 (0.00%)  1/60 (1.67%)  0/170 (0.00%) 
Dysphagia   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
General disorders         
Malaise   0/63 (0.00%)  1/64 (1.56%)  0/60 (0.00%)  1/170 (0.59%) 
Asthenia   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Fatigue   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Hypothermia   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Infections and infestations         
Pneumonia   0/63 (0.00%)  1/64 (1.56%)  0/60 (0.00%)  5/170 (2.94%) 
Urinary tract infection   1/63 (1.59%)  0/64 (0.00%)  0/60 (0.00%)  3/170 (1.76%) 
Urosepsis   0/63 (0.00%)  0/64 (0.00%)  1/60 (1.67%)  0/170 (0.00%) 
Cellulitis   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Fungal skin infection   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Herpes zoster   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Sepsis   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Staphylococcal infection   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Injury, poisoning and procedural complications         
Burns first degree   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Burns second degree   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Cervical vertebral fracture   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Facial bones fracture   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Fall   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Hip fracture   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Lumbar vertebral fracture   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Subdural haematoma   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Metabolism and nutrition disorders         
Malnutrition   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Musculoskeletal and connective tissue disorders         
Arthralgia   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Brain cancer metastatic   0/63 (0.00%)  1/64 (1.56%)  0/60 (0.00%)  0/170 (0.00%) 
Nodular melanoma   0/63 (0.00%)  0/64 (0.00%)  1/60 (1.67%)  0/170 (0.00%) 
Lung neoplasm   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Pharyngeal cancer   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Nervous system disorders         
Syncope   0/63 (0.00%)  1/64 (1.56%)  0/60 (0.00%)  2/170 (1.18%) 
Amyotrophic lateral sclerosis   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Dementia with Lewy bodies   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Encephalopathy   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  2/170 (1.18%) 
Ischaemic stroke   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Parkinson's disease   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Parkinsonism   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Radiculopathy   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Transient ischaemic attack   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Vocal cord paralysis   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Psychiatric disorders         
Aggression   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Respiratory, thoracic and mediastinal disorders         
Pneumonia aspiration   0/63 (0.00%)  1/64 (1.56%)  0/60 (0.00%)  1/170 (0.59%) 
Aspiration   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Pulmonary embolism   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  0/170 (0.00%) 
Respiratory failure   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  3/170 (1.76%) 
Vascular disorders         
Circulatory collapse   0/63 (0.00%)  0/64 (0.00%)  1/60 (1.67%)  0/170 (0.00%) 
Arteriosclerosis   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Deep vein thrombosis   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  1/170 (0.59%) 
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
MYOBLOC 2500 U (Part A) MYOBLOC 3500 U (Part A) Placebo (Part A) OL: ALL MYOBLOC (Part B)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   44/63 (69.84%)   38/64 (59.38%)   16/60 (26.67%)   143/170 (84.12%) 
Gastrointestinal disorders         
Dental caries   5/63 (7.94%)  3/64 (4.69%)  2/60 (3.33%)  50/170 (29.41%) 
Dry mouth   24/63 (38.10%)  29/64 (45.31%)  5/60 (8.33%)  73/170 (42.94%) 
Dysphagia   7/63 (11.11%)  3/64 (4.69%)  1/60 (1.67%)  21/170 (12.35%) 
Infections and infestations         
Urinary tract infection   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  12/170 (7.06%) 
Injury, poisoning and procedural complications         
Fall   4/63 (6.35%)  3/64 (4.69%)  4/60 (6.67%)  28/170 (16.47%) 
Contusion   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  11/170 (6.47%) 
Investigations         
Weight decreased   0/63 (0.00%)  0/64 (0.00%)  0/60 (0.00%)  10/170 (5.88%) 
Nervous system disorders         
Dizziness   4/63 (6.35%)  0/64 (0.00%)  0/60 (0.00%)  10/170 (5.88%) 
Respiratory, thoracic and mediastinal disorders         
Cough   0/63 (0.00%)  0/64 (0.00%)  4/60 (6.67%)  8/170 (4.71%) 
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Sponsor has the first right, within 9 months following the later to occur 1) close out meeting or 2) Sponsor possessing all data, to publish the lead paper. Following the foregoing, Institution has the right to publish the results provided the Institution provides a Publications Committee, which includes the Sponsor, with a draft at least 30 days before submission. At Sponsor's request, Institution shall withhold publication for 45 days to allow filing of a patent application.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Regulatory Affairs Associate
Organization: Solstice Neurosciences
EMail: regulatoryaffairs@usworldmeds.com
Layout table for additonal information
Responsible Party: Supernus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01994109    
Other Study ID Numbers: SN-SIAL-301
First Submitted: November 14, 2013
First Posted: November 25, 2013
Results First Submitted: May 2, 2019
Results First Posted: August 15, 2019
Last Update Posted: July 13, 2021