WithdrawnPhase 3

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

Stopped: Company/sponsor business decision

0Started 2021-11

Plain-language summary

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

Age range3 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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).
✓. 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:

✕. 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).
✕. 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.

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

Timeframe: Baseline and Week 4

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

Timeframe: Week 4

NCT IDNCT05097079

SponsorSolstice Neurosciences, LLC, a subsidiary of MDD US Operations, LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-05

Who can participate

Age range3 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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).
✓. 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:

✕. 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).
✕. 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.