| Eligibility |
Inclusion Criteria:To be eligible to participate in this study, all of the following
criteria must be met:
1. Diagnosis of 16p11.2 BP4-BP5 deletion.
2. Male or female subjects, 5 through 17 years of age, at Screening.
3. Neurodevelopmental disability requiring current educational or other therapeutic
support.
4. Any educational, behavioral, other therapeutic, and/or dietary interventions must have
been continuous during the 2 months prior to Screening (with the exception of the
anti-epileptic drug (AED) 6-month requirement in Inclusion Criterion #5). Subjects and
their parent/caregiver/legally authorized representative (LAR) may not electively
initiate new, or modify ongoing, interventions for the duration of the study. Typical
school vacations are not considered modifications of stable programming.
5. Subjects with a history of seizure disorder must have been seizure-free and on a
stable antiepileptic therapy regimen for 6 months OR must have been seizure-free for
the 3 years prior to Screening if not currently receiving antiepileptics. If currently
receiving treatment with antiepileptics that are typically monitored by serum
concentration, serum concentrations of the antiepileptic drugs must be tested and
confirmed to be within the therapeutic range at Screening.
6. All medication regimens must be stable for 30 days prior to Screening.
7. Prior to the conduct of any study-specific procedures, the subject must provide assent
to participate in the study (if developmentally appropriate), and the
parent/caregiver/LAR must provide written informed consent. If the caregiver attending
the clinic visits is not the parent or LAR, written consent must also be obtained from
the parent or LAR for the subject's participation in the study.
8. The subject's parent/caregiver/LAR must be able to speak and understand English
sufficiently to understand the nature of the study and to allow for the completion of
all study assessments. The parent/caregiver/LAR should be capable of providing
reliable information about the subject's condition, agree to oversee the
administration of the study drug, and accompany the subject to all clinic visits. The
same parent/caregiver/LAR should accompany the subject to each visit.
9. Negative pregnancy test for females who are 9 years of age or older. Both male and
female subjects who are sexually active must agree to consistently use an accepted
form of contraception (i.e., surgical sterilization, intrauterine contraceptive
device, subcutaneous implant, oral contraceptive, or diaphragm or condom in
combination with contraceptive cream/jelly, or abstinence) throughout the trial and
for 30 days (females) or 90 days (males) following last study drug administration.
Exclusion Criteria:Subjects are not permitted to enroll in the study if any of the
following criteria are met:
1. Subjects with additional known genetic disorder besides 16p11.2 BP4-BP5 deletion.
2. Subjects receiving remote or hybrid schooling at Screening.
3. Subjects with an additional neurologic or psychiatric condition that might confound
performance on assessments measures, e.g., significant impairment in hearing or
vision, severe motor impairment (e.g., non-ambulatory) from cerebral palsy, birth
injury, or other injury, or cleft lip or palate (including submucous cleft).
4. Subjects with any seizures within the previous 6 months; subjects who are not
currently receiving antiepileptics AND have had one or more seizures during the 3
years prior to Screening; and subjects who are shown to have non-therapeutic AED
levels at Screening.
5. Subjects with any medical or psychiatric condition that might interfere with the
conduct of the study, confound interpretation of the study results, or endanger their
own well-being. This includes, but is not limited to substance use disorders,
impairment of renal function, evidence or history of malignancy or any significant
hematological, endocrine, cardiovascular, respiratory, hepatic, or gastrointestinal
disease.
6. Subjects who plan to initiate or change pharmacologic or non-pharmacologic
interventions during the course of the study.
7. Subjects who are currently treated or have been treated with racemic baclofen in the
last 30 days.
8. Subjects currently treated with antipsychotic medication(s).
9. Subjects currently treated with more than 2 psychoactive medications, including
antiepileptics used as an anti-seizure treatment, but not including sleep aids used on
an as-needed basis.
10. Subjects currently treated with drugs having anxiolytic properties, including but not
limited to: buspirone and beta-blockers. Benzodiazepines administered on a regular
schedule (more than 3x/week) are not permitted. Use of antidepressants may be
permitted with approval of the Medical Monitor. Other prohibited and restricted
medications are shown in Appendix A.
11. Subjects currently treated with vigabatrin, tiagabine, or riluzole.
12. Subjects taking another investigational drug currently or within the last 30 days.
13. Subjects who are not able or willing to take oral disintegrating tablets.
14. Subjects who have a history of hypersensitivity to racemic baclofen.
15. Subjects who, in the Investigator's opinion, might not be suitable for the study.
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