Overview
Efficacy and Safety Study of MYOBLOC® in the Treatment of Sialorrhea in Pediatric Subjects
Status:
Not yet recruiting
Not yet recruiting
Trial end date:
2024-03-30
2024-03-30
Target enrollment:
0
0
Participant gender:
All
All
Summary
This study will evaluate the efficacy and safety of MYOBLOC for the Treatment of Chronic Sialorrhea in Pediatric Subjects.Phase:
Phase 3Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Supernus Pharmaceuticals, Inc.Collaborator:
Solstice NeurosciencesTreatments:
Botulinum Toxins, Type A
rimabotulinumtoxinB
Criteria
Inclusion Criteria:1. 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.
2. Written minor assent obtained from the subject, as applicable and in accordance with
local laws and IRB/IEC requirements.
3. Male or female ages 3 to < 17 years at the time of signing informed consent (and
assent, if applicable) at Screening.
4. Minimum weight of 10 kg at Screening and Baseline (prior to randomization).
5. 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.
6. A mTDS score ≥ 5 at Screening and Baseline (prior to randomization).
7. A minimum USFR of 0.2 g/min at Screening and Baseline (prior to randomization).
8. 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:
1. simultaneous use of male condom and intra-uterine contraceptive device placed
during screening period prior to baseline (randomization, injection)
2. 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;
3. established use of oral, injected or implanted hormonal methods of contraception;
4. 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).
9. 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.
Exclusion Criteria:
1. 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.
2. History of drug or alcohol abuse within 6 months before Screening.
3. Treatment with an investigational drug, device, or biological agent within 30 days
before Screening or while participating in this study.
4. 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).
5. Aspiration pneumonia within 6 months before Screening.
6. 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).
7. Requires general anesthesia for study drug administration.
8. 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.
9. 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.
10. History of lack of response to MYOBLOC.
11. 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.
12. Oxygen saturation < 95% on room air at Screening and Baseline (prior to
randomization).
13. 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.
14. 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.
15. 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).
16. 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.
17. 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.
18. 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.
19. 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:
1. Serum creatinine >1.5 times the upper limit of normal (ULN);
2. Serum total bilirubin >1.5 times ULN;
3. Serum alanine aminotransferase or aspartate aminotransferase >2 times ULN.
20. Has any of the following cardiac findings at Screening:
1. Abnormal ECG that is, in the Investigator's opinion/evaluation, clinically
significant;
2. PR interval > 150 msec (2-5 years old); > 170 msec (6-11 years old); or > 180
msec (12-17 years old)
3. QRS interval > 80 msec (2-5 years old); or > 90 msec (6-17 years old)
4. QTcF interval >450 msec (for males), or >470 msec (for females) (QT corrected
using Fridericia's method);
5. Second-or third-degree atrioventricular block;
6. Any rhythm, other than sinus rhythm, that is interpreted or assessed by the
Investigator to be clinically significant.
21. Has received COVID-19 vaccine (single or 2nd dose) in the last 30 days prior to
Screening.
22. 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.
23. 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.