A Study of TEV-50717 (Deutetrabenazine) for the Treatment of Dyskinesia in Cerebral Palsy in Children and Adolescents
Condition: Cerebral Palsy, Dyskinetic
MeSH terms: Cerebral Palsy; Dyskinesias
Intervention: TEV-50717; Placebo
Intervention description: Dosage ranging from 6 mg to 48 mg.; Dosage ranging from 6 mg to 48 mg.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 63.0
Sex: All
Minimum age: 6 Years
Maximum age: 18 Years
Healthy volunteers: No
Keywords: DCP

Summary:

      CP (cerebral palsy) refers to a group of neurological disorders that appear in infancy or
      early childhood and permanently affect body movement and muscle coordination. CP is caused by
      damage to or abnormalities inside the developing brain that disrupt the brain's ability to
      control movement and maintain posture and balance. The signs of CP usually appear in the
      early months of life, although specific diagnosis may be delayed until the age of 2 years or
      older. TEV-50717 (deutetrabenazine, also known as SD-809) has already provided evidence for
      safe and effective use in 2 other hyperkinetic movement disorders, namely chorea in
      Huntington's disease (HD) and tardive dyskinesia (TD). Currently, there is no approved
      treatment available for Dyskinesia in cerebral palsy (DCP). The available treatment options
      address some of the manifestations of DCP. The study population will include pediatric and
      adolescent participants (6 through 18 years of age) with DCP with predominant choreiform
      movement disorder, who have had nonprogressive CP symptoms since infancy (≤2 years of age).
      Diagnosis of DCP is based on the Surveillance of Cerebral Palsy in Europe criteria.

      This is a Phase 3 study that will evaluate the efficacy and safety of TEV-50717 administered
      as oral tablets at a starting dose of 6 mg once daily in participants (age 6 through 18
      years, inclusive) with DCP with predominant choreiform movement disorder. The study will be
      conducted in multiple centers and will use 2 parallel treatment groups (ie, TEV-50717 and
      placebo) in which participants will be randomized in a 2:1 ratio.

      "Predominant" in this instance indicates that the choreiform movement disorder is the main
      cause of impairment or distress.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Participant is 6 through 18 years of age (inclusive) at baseline.

          -  Participant weighs at least 26 pounds (12 kg) at baseline.

          -  Participant has had CP symptoms since infancy (≤2 years)

          -  Choreiform is the prevalent movement disorder as assessed by the EAB at screening.

          -  Participant has a diagnosis of DCP

          -  Participant is able to swallow study medication whole.

          -  Females who are postmenarchal or ≥12 years of age whose male partners are potentially
             fertile (ie, no vasectomy) must use highly effective birth control methods for the
             duration of the study

          -  Additional criteria apply, please contact the investigator for more information

        Exclusion Criteria:

          -  Participant has a predominant movement disorder other than dyskinesia.

          -  Participant's predominant motor symptoms are dystonic.

          -  Participant's predominant motor symptoms are spastic.

          -  Participant has choreiform movement disorder that has not been consistent throughout
             the life of the participant.

          -  Participant has clinically significant depression at screening or baseline.

          -  Note: Participants receiving antidepressant therapy may be enrolled if on a stable
             dose for at least 6 weeks before screening.

          -  Participant has a history of suicidal intent or related behaviors within 2 years of
             screening:

          -  Previous intent to act on suicidal ideation with a specific plan, irrespective of
             level of ambivalence, at the time of suicidal thought

          -  Previous suicidal preparatory acts or behavior

          -  Participant has a history of a previous actual, interrupted, or aborted suicide
             attempt.

          -  Participant has a first-degree relative who has completed suicide.

          -  Participant has received treatment with stem cells, deep brain stimulation,
             transmagnetic stimulation, or transcranial direct current stimulation for treatment of
             abnormal movements or CP within 6 months of the screening visit, or the participant is
             not in a stable clinical condition.

          -  Participant has recent surgical procedure or is anticipated to have a surgical
             procedure during the study that, in the opinion of the investigator, makes the
             Participant unsuitable for the study.

          -  Participant has a severe mental disability or an unstable or serious medical illness
             (eg, epilepsy) at screening or baseline that, in the opinion of the investigator,
             could jeopardize or would compromise the Participant's ability to participate in this
             study.

          -  Participant has a known allergy to any of the components of the investigational
             medicinal product (IMP).

          -  Participant is pregnant or breastfeeding.

          -  Participant has a history of or acknowledges alcohol or substance abuse in the 12
             months before screening

          -  Participants with a history of torsade de pointes, congenital long QT syndrome,
             bradyarrhythmias, other cardiac arrhythmias, or uncompensated heart failure.

          -  Additional criteria apply, please contact the investigator for more information