Trial Outcomes & Findings for Efficacy and Safety Study of MYOBLOC® Followed by Open-Label Multiple-Treatment With MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects (NCT NCT01994109)

NCT ID: NCT01994109

Last Updated: 2021-07-13

Results Overview

Change weight of expectorated saliva at a Week 4 post-injection visit.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

187 participants

Primary outcome timeframe

4 Weeks

Results posted on

2021-07-13

Participant Flow

The study was conducted at 33 sites including United States, Russia and Ukraine.

DB: Double Blind (Part A) OL: Open Label (Part B)

Participant milestones

Participant milestones
Measure	DB: MYOBLOC 2500 U Participants will receive specified dose of MYOBLOC	DB: MYOBLOC 3500 U Participants will receive specified dose of MYOBLOC	DB: Placebo Participants will receive volume matched Placebo	OL: ALL MYOBLOC All Participants were to receive 3500 U MYOBLOC but could receive lower doses at investigator discretion.
Double-blind Phase (Part A) STARTED	63	64	60	0
Double-blind Phase (Part A) COMPLETED	62	61	53	0
Double-blind Phase (Part A) NOT COMPLETED	1	3	7	0
Open-label Phase (Part B) STARTED	0	0	0	170
Open-label Phase (Part B) COMPLETED	0	0	0	128
Open-label Phase (Part B) NOT COMPLETED	0	0	0	42

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	MYOBLOC 2500 U n=63 Participants Subjects were dosed per treatment assignment of 2500 U MYOBLOC.	MYOBLOC 3500 U n=64 Participants Subjects were dosed per treatment assignment of 3500 U MYOBLOC.	PLACEBO n=57 Participants Placebo was dosed as an exact match of MYOBLOC.	Total n=184 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	29 Participants n=5 Participants	25 Participants n=7 Participants	24 Participants n=5 Participants	78 Participants n=4 Participants
Age, Categorical >=65 years	34 Participants n=5 Participants	39 Participants n=7 Participants	33 Participants n=5 Participants	106 Participants n=4 Participants
Age, Continuous	62.6 years STANDARD_DEVIATION 13.0 • n=5 Participants	64.6 years STANDARD_DEVIATION 14.0 • n=7 Participants	64.1 years STANDARD_DEVIATION 13.1 • n=5 Participants	63.8 years STANDARD_DEVIATION 13.4 • n=4 Participants
Sex: Female, Male Female	15 Participants n=5 Participants	9 Participants n=7 Participants	16 Participants n=5 Participants	40 Participants n=4 Participants
Sex: Female, Male Male	48 Participants n=5 Participants	55 Participants n=7 Participants	41 Participants n=5 Participants	144 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	5 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	8 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	58 Participants n=5 Participants	62 Participants n=7 Participants	56 Participants n=5 Participants	176 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	4 Participants n=4 Participants
Race (NIH/OMB) White	60 Participants n=5 Participants	60 Participants n=7 Participants	55 Participants n=5 Participants	175 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants

PRIMARY outcome

Timeframe: 4 Weeks

Population: The per protocol included subjects with a minimum USFR of 0.2 g/min.

Change weight of expectorated saliva at a Week 4 post-injection visit.

Outcome measures

Outcome measures
Measure	MYOBLOC 2500 U n=63 Participants Subjects were dosed per treatment assignment of 2500 U MYOBLOC.	MYOBLOC 3500 U n=64 Participants Subjects were dosed per treatment assignment of 3500 U MYOBLOC	Placebo n=57 Participants Placebo was dosed as an exact match of MYOBLOC.
Unstimulated Salivary Flow Rate (USFR) at Week 4 Post-injection Visit (Part A)	-0.37 g/minute Standard Error 0.03	-0.36 g/minute Standard Error 0.03	-0.07 g/minute Standard Error 0.03

PRIMARY outcome

Timeframe: 4 weeks

Population: 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).

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.

Outcome measures

Outcome measures
Measure	MYOBLOC 2500 U n=63 Participants Subjects were dosed per treatment assignment of 2500 U MYOBLOC.	MYOBLOC 3500 U n=64 Participants Subjects were dosed per treatment assignment of 3500 U MYOBLOC	Placebo n=60 Participants Placebo was dosed as an exact match of MYOBLOC.
Clinical Global Impression Change (CGI-C) at Week 4 Post-injection (Part A)	2.38 score on a scale Standard Error 0.12	2.45 score on a scale Standard Error 0.12	3.59 score on a scale Standard Error 0.13

Adverse Events

MYOBLOC 2500 U (Part A)

Serious events: 1 serious events

Other events: 44 other events

Deaths: 0 deaths

MYOBLOC 3500 U (Part A)

Serious events: 4 serious events

Other events: 38 other events

Deaths: 2 deaths

Placebo (Part A)

Serious events: 4 serious events

Other events: 16 other events

Deaths: 1 deaths

OL: ALL MYOBLOC (Part B)

Serious events: 32 serious events

Other events: 143 other events

Deaths: 9 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	MYOBLOC 2500 U (Part A) n=63 participants at risk Participants received specified dose of MYOBLOC. Participants were evaluated for 13 weeks following the injection.	MYOBLOC 3500 U (Part A) n=64 participants at risk Participants received specified dose of MYOBLOC. Participants were evaluated for 13 weeks following the injection.	Placebo (Part A) n=60 participants at risk Participants received volume matched Placebo. Participants were evaluated for 13 weeks following the injection.	OL: ALL MYOBLOC (Part B) n=170 participants at risk Participants received Open Label treatment with 3500 U (decreased dose at investigator discretion) every 13 weeks for a maximum of 4 treatment sessions.
Gastrointestinal disorders Dental caries	7.9% 5/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	4.7% 3/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	3.3% 2/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	29.4% 50/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)
Gastrointestinal disorders Dry mouth	38.1% 24/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	45.3% 29/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	8.3% 5/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	42.9% 73/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)
Gastrointestinal disorders Dysphagia	11.1% 7/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	4.7% 3/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	1.7% 1/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	12.4% 21/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)
Injury, poisoning and procedural complications Fall	6.3% 4/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	4.7% 3/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	6.7% 4/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	16.5% 28/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)
Nervous system disorders Dizziness	6.3% 4/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	5.9% 10/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	6.7% 4/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	4.7% 8/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)
Infections and infestations Urinary tract infection	0.00% 0/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	7.1% 12/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)
Injury, poisoning and procedural complications Contusion	0.00% 0/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	6.5% 11/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)
Investigations Weight decreased	0.00% 0/63 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/64 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	0.00% 0/60 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)	5.9% 10/170 • Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods 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)

Additional Information

Regulatory Affairs Associate

Solstice Neurosciences

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER