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SialorrheaPhase 3NCT05097079

Efficacy and Safety Study of MYOBLOC in the Treatment of Sialorrhea in Pediatric Subjects

A Phase 3 study testing MYOBLOC Low Dose and MYOBLOC High Dose in people with Sialorrhea, run by Solstice Neurosciences, LLC, a subsidiary of MDD US Operations, LLC.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This study will evaluate the efficacy and safety of MYOBLOC for the treatment of chronic sialorrhea in pediatric subjects. Read more about this study

Who is it for?
Ages 3 to 17 years
Conditions the study is for
Sialorrhea
What is being tested?
MYOBLOC Low Dose and MYOBLOC High Dose
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
0 participants
Where
No sites listed yet
Study dates
Nov 2021to May 2023
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Written informed consent obtained from the subject's parent or legally authorized representative(s) (LAR)/guardian(s) in accordance with local laws and Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements.
  • Written minor assent obtained from the subject, as applicable and in accordance with local laws and IRB/IEC requirements.
  • Male or female ages 3 to < 17 years at the time of signing informed consent (and assent, if applicable) at Screening.
  • Minimum weight of 10 kg at Screening and Baseline (prior to randomization).
  • Chronic sialorrhea due to a neurological disorder (e.g., cerebral palsy (CP), or traumatic brain injury (TBI)) for at least 3 months prior to Screening.
  • A mTDS score ≥ 5 at Screening and Baseline (prior to randomization).
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  • A minimum USFR of 0.2 g/min at Screening and Baseline (prior to randomization).
  • Females of childbearing potential (FOCP) must be either sexually inactive (abstinent) or, if sexually active, must agree to use/practice one of the following acceptable methods of contraception beginning during screening period prior to baseline (randomization, injection), for the duration of the study, and 2 months after study completion:
  • simultaneous use of male condom and intra-uterine contraceptive device placed during screening period prior to baseline (randomization, injection)
  • barrier method: condom with spermicidal foam/gel/film/cream/suppository or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository;
  • established use of oral, injected or implanted hormonal methods of contraception;
  • surgically sterile male partner (e.g., vasectomized partner is sole partner). With approval by the Investigator, subjects' parents or legal guardians may select abstinence as a form of birth control if deemed more appropriate. For the purposes of this study, all females are considered to be of childbearing potential unless they are confirmed by the Investigator to be premenarchal, biologically sterile, or surgically sterile (e.g., hysterectomy with bilateral oophorectomy, tubal ligation).
  • Subject and the subject's parent/LAR are willing and able to comply with scheduled visits, treatment plan, laboratory tests, home monitoring, and other study procedures.

Who cannot join

  • FOCP subjects who are pregnant, lactating/breastfeeding and/or sexually active and not agreeing to use one of the acceptable birth control methods throughout the study.
  • History of drug or alcohol abuse within 6 months before Screening.
  • Treatment with an investigational drug, device, or biological agent within 30 days before Screening or while participating in this study.
  • Major surgery (requiring general anesthesia) within 3 months before screening, or any anticipated or scheduled surgery during the study period (with or without general anesthesia).
  • Aspiration pneumonia within 6 months before Screening.
  • History of moderate dysphagia or severe dysphagia (defined as an inability to swallow liquids, solids or both without choking or medical intervention) within 6 months before screening. Subjects who require gastrostomy tube feeding are not excluded provided tube placement was at least 30 days prior to Baseline (Day 1; injection).
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  • Requires general anesthesia for study drug administration.
  • Prior botulinum toxin type A (BoNT/A) or BoNT/B treatment for sialorrhea or cervical dystonia within 20 weeks before screening. Prior BoNT/A or BoNT/B treatment in other anatomical locations is not exclusionary, but must have occurred at least 12 weeks prior to screening. Prior toxin exposure must have been well tolerated and without any significant long-term side effects in cases of repeated prior exposure.
  • Subjects must not receive nor have any plans to receive any other form of botulinum toxin treatment, other than the study drug (MYOBLOC), from the time that the informed consent is obtained until participation in the study is complete.
  • History of lack of response to MYOBLOC.
  • Any previous known or suspected hypersensitivity to botulinum toxins type A (BoNT/A) or B (BoNT/B) or to any of the MYOBLOC solution components.
  • Oxygen saturation < 95% on room air at Screening and Baseline (prior to randomization).
  • Subjects taking medications with anticholinergic/antihistamine properties who have not been on a stable dose and regimen for at least 2 weeks before Day 1. The same dose and dosing regimen must be maintained through the Week 4 study visit.
  • Diagnosed with Obstructive Sleep Apnea which requires nightly Continuous Positive Airway Pressure (CPAP) or Bi-level Positive Airway Pressure (BiPAP) therapy, such that subject has been receiving nightly therapy for at least 30 days.
  • Current or recent treatment (exposure within 5 half-lives of screening) or treatment at any time during the study with aminoglycoside antibiotics, curare-like agents, other agents that interfere with neuromuscular function, or dopamine receptor blocking agents (e.g., clozapine).
  • 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.
  • Prior surgery or irradiation in the head and neck to control sialorrhea (including salivary gland surgery or salivary gland irradiation) within 1 year before screening or planned during the study.
  • Extremely poor dental and/or oral condition, including infection at injection site(s) that might preclude safe study participation according to the judgment of the Investigator.
  • Has one or more screening clinical laboratory test values outside the reference range that, in the opinion of the Investigator, are clinical significant, or any of the following:
  • Serum creatinine >1.5 times the upper limit of normal (ULN);
  • Serum total bilirubin >1.5 times ULN;
  • Serum alanine aminotransferase or aspartate aminotransferase >2 times ULN.
  • Has any of the following cardiac findings at Screening:
  • Abnormal ECG that is, in the Investigator's opinion/evaluation, clinically significant;
  • PR interval > 150 msec (2-5 years old); > 170 msec (6-11 years old); or > 180 msec (12-17 years old)
  • QRS interval > 80 msec (2-5 years old); or > 90 msec (6-17 years old)
  • QTcF interval >450 msec (for males), or >470 msec (for females) (QT corrected using Fridericia's method);
  • Second-or third-degree atrioventricular block;
  • Any rhythm, other than sinus rhythm, that is interpreted or assessed by the Investigator to be clinically significant.
  • Has received COVID-19 vaccine (single or 2nd dose) in the last 30 days prior to Screening.
  • Chronic or current use of inhaled corticosteroids, short acting beta-agonists or any other medication to manage asthma or other lung conditions such as emphysema.
  • Any other medical illness, condition, or clinical finding, including clinically significant abnormal laboratory values that in the opinion of the Investigator and/or the Sponsor, would put the subject at undue risk.

About this study

Official title: A Phase 3 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Single-Treatment Efficacy and Safety Study of MYOBLOC® for the Treatment of Chronic Sialorrhea in Pediatric Subjects

This study will evaluate the efficacy and safety of MYOBLOC for the treatment of chronic sialorrhea in pediatric subjects.

Read the detailed description

This is a multicenter, randomized, double-blind, placebo-controlled, single treatment study designed to assess the efficacy, safety, and tolerability of 2 dose levels of MYOBLOC vs placebo for the treatment of chronic sialorrhea in pediatric subjects ages 3 to < 17 years. Eligible subjects will be randomized 1:1:1 to receive single treatment of the low dose of MYOBLOC (60 Units/kg), the high dose of MYOBLOC (100 Units/kg), or volume-matched placebo via intraglandular injection.

Conditions
Sialorrhea
Being tested
MYOBLOC Low Dose, MYOBLOC High Dose, Placebo
Medical subject headings
Sialorrhea

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MYOBLOC Low DoseDrug

    Also called rimabotulinumtoxinB

    Subjects assigned to the MYOBLOC Low Dose treatment group will be administered MYOBLOC (60 units/kg; maximum 1800 units) as a single treatment via intraglandular injection (5.0 units/kit per submandibular gland [maximum 150 units] and 25.0 units/kg per parotid gland [maximum 750 units])

  • MYOBLOC High DoseDrug

    Also called rimabotulinumtoxinB

    Subjects assigned to the MYOBLOC High Dose treatment group will be administered MYOBLOC (100 units/kg; maximum 3000 units) as a single treatment via intraglandular injection (25.0 units/kit per submandibular gland [maximum 300 units] and 40.0 units/kg per parotid gland [maximum 1200 units])

  • PlaceboDrug

    Also called PBO

    Subjects assigned to the Placebo treatment group will be administered volume-matched placebo (1:1)

Study groups in NCT05097079
Study groupRoleWhat they receive
MYOBLOC Low DoseExperimentalMYOBLOC Low Dose will be administered as a single treatment via intraglandular injection (60 Units/kg)
Receives: Drug: MYOBLOC Low Dose
MYOBLOC High DoseExperimentalMYOBLOC High Dose will be administered as a single treatment via intraglandular injection (100 units/kg)
Receives: Drug: MYOBLOC High Dose
PlaceboPlacebo comparatorVolume-matched placebo will be administered as a single treatment via intraglandular injection
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The change from baseline in the Unstimulated Saliva Flow Rate (USFR) at Week 4 post-injection.

    At each study visit, the subject's saliva is collected for at least 3 minutes and weighed (grams). The Unstimulated Saliva Flow Rate (USFR; grams/minute) for each visit is calculated for by dividing the 'total grams of saliva collected during the collection period' by the 'total minutes of the collection period. The USFR at Week 4 post-injection visit subtracted from the USFR at Baseline visit (prior to injection) yields the change from baseline USFR. A change from baseline USFR <0 grams/minutes represents a better outcome.

    Measured: Baseline and Week 4

  2. The Clinical Global Impression of Change (CGI-C) score at Week 4 post-injection.

    The Clinical Global Impression of Change (CGI-C) scale is a single item clinician assessment of how much the subject's condition (sialorrhea) has improved, worsened or has not changed relative to subject's state at baseline prior to treatment (injection). The CGI-C is rated on a 7-point scale from 1 to 7, where 1 = "Very much improved", 2 = "Much improved", 3 = "Minimally improved", 4 = "No change", 5 = "Minimally worse", 6 = "Much worse", and 7 = "Very much worse". A CGI-C score <4 represents a better outcome.

    Measured: Week 4

Secondary outcomes (6)

  1. The Parent/Guardian Global Impression of Change (PGI-C) score at Week 4 post-injection.

    The Parent/Guardian Global Impression of Change (PGI-C) scale is a single item parent/guardian assessment of how much the subject's condition (sialorrhea) has improved, worsened or has not changed relative to subject's state at baseline prior to treatment (injection). The PGI-C is rated on a 7-point scale from 1 to 7, where 1 = "Very much improved", 2 = "Much improved", 3 = "Minimally improved", 4 = "No change", 5 = "Minimally worse", 6 = "Much worse", and 7 = "Very much worse". A PGI-C score <4 represents a better outcome

    Measured: Week 4

  2. The change from baseline in the Unstimulated Saliva Flow Rate (USFR) at Weeks 2, 8 and 13 post-injection.

    At each study visit, the subject's saliva is collected for at least 3 minutes and weighed (grams). The Unstimulated Saliva Flow Rate (USFR; grams/minute) for each visit is calculated for by dividing the 'total grams of saliva collected during the collection period' by the 'total minutes of the collection period'. The USFR at the post-injection visit subtracted from the USFR at Baseline visit (prior to injection) yields the change from baseline USFR. A change from baseline USFR <0 grams/minutes represents a better outcome.

    Measured: Baseline and Weeks 2, 8 and 13

  3. The Clinical Global Impression of Change (CGI-C) score at Weeks 2, 8, and 13 post-injection.

    The Clinical Global Impression of Change (CGI-C) scale is a single item clinician assessment of how much the subject's the subject's condition (sialorrhea) has improved, worsened or has not changed relative to subject's state at baseline prior to treatment (injection). The CGI-C is rated on a 7-point scale from 1 to 7, where 1 = "Very much improved", 2 = "Much improved", 3 = "Minimally improved", 4 = "No change", 5 = "Minimally worse", 6 = "Much worse", and 7 = "Very much worse". A CGI-C score <4 represents a better outcome.

    Measured: Weeks 2, 8 and 13

  4. The Parent/Guardian Global Impression of Change (PGI-C) score at Week 2, 8, and 13 post-injection.

    The Parent/Guardian Global Impression of Change (PGI-C) scale is a single item parent/guardian assessment of how much the subject's condition (sialorrhea) has improved, worsened or has not changed relative to subject's state at baseline prior to treatment (injection). The PGI-C is rated on a 7-point scale from 1 to 7, where 1 = "Very much improved", 2 = "Much improved", 3 = "Minimally improved", 4 = "No change", 5 = "Minimally worse", 6 = "Much worse", and 7 = "Very much worse". A PGI-C score <4 represents a better outcome.

    Measured: Weeks 2, 8, and 13

  5. The change from baseline in the Modified Teacher's Drooling Scale (mTDS) score at Week4 post injection.

    The Modified Teacher's Drooling Scale (mTDS) is a single-item scale used to assess subject's drooling severity and frequency. It is rated by the parent/guardian on a 9-point scale (1-9); where 1 = "Dry: never drools"; 2 = "Mild: only the lips are wet, occasionally"; 3 = "Mild: only the lips are wet, frequently"; 4 = "Moderate: wet on the lips and chin, occasionally"; 5 = "Moderate: wet on the lips and chin, frequently"; 6 = "Severe: drools to the extent that clothing becomes damp, occasionally"; 7 = "Severe: drools to the extent that clothing becomes damp, frequently"; 8 = "Profuse: clothing, hands, tray, and objects become wet, occasionally"; 9 = "Profuse: clothing, hands, tray, and objects become wet, frequently". The mTDS score at Week 4 Post injection subtracted from the mTDS score at Baseline yields the change from baseline mTDS score. A change from baseline mTDS score <0 represents a better outcome.

    Measured: Baseline and Week 4

  6. The change from baseline in the Drooling Impact Scale (DIS) score at Week 4 post-injection.

    The Drooling Impact Scale (DIS) is a 10-item questionnaire used to assess the frequency, severity, and impact of subject's drooling. Each item is rated by the parent/guardian on a 10-point end-anchor semantic differential scale (1 to 10; where 1 = "best outcome" to 10 = "worst outcome"). The sum of all 10 items yields a total score (ranging from 10-100). The DIS total score at Week 4 Post injection subtracted from the DIS total score at Baseline yields the change from baseline DIS total score. A change from baseline DIS score <0 represents a better outcome.

    Measured: Baseline and Week 4

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Oct 2021
  2. Study startNov 2021 (estimated)
  3. Primary completionMay 2023 (estimated)
  4. Study completionMay 2023 (estimated)

Why it stopped: Company/sponsor business decision

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
A Phase 3, multicenter, randomized, double-blind, placebo-controlled, single treatment study designed to assess the efficacy, safety, and tolerability of 2 dose levels of MYOBLOC compared to placebo for the treatment of chronic sialorrhea in pediatric subjects ages 3 to \< 17 years.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05097079
Sponsor's protocol ID
SN-SIAL-302
Status last verified
Sep 2024
First submitted
18 Oct 2021
Last updated
19 Sep 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing MYOBLOC Low Dose and MYOBLOC High Dose in people with Sialorrhea, run by Solstice Neurosciences, LLC, a subsidiary of MDD US Operations, LLC.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 32 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The change from baseline in the Unstimulated Saliva Flow Rate (USFR) at Week 4 post-injection. (time frame: Baseline and Week 4). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05097079.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.

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