Time commitment/Requirements
Participation will last up to two years, depending on when you complete the screening, whether you are enrolled in the delayed treatment group, or whether you complete the optional follow-up visits. A breakdown of each visit is provided below:
Visit 1: Screening (no more than 12 weeks prior to Baseline)
Review eligibility and health history, DSRD diagnostic workup, consent to participate, screening labs (blood work), MRI, EEG, LP, EKG, chest X-Ray, safety monitoring (i.e. vitals and a brief physical exam), randomization into a treatment arm
Visit 2a: Baseline (this visit is the same as Visit 2b; it is only applicable to participants in the delayed treatment group)
Visit 2b: Baseline & Begin Treatment (Time 0)
Questionnaires, safety monitoring, research labs (blood work), neurologic exam, clinical and cognitive assessments
Visit 3: Treatment (Week 2)
Safety monitoring, research labs (blood work)
Visit 4: Treatment (Week 4)
Safety monitoring, research labs (blood work), EKG
Visit 5: Treatment (Week 8)
Safety monitoring, research labs (blood work)
Visit 6: Endpoint & End Treatment (Week 12)
Questionnaires, safety monitoring, research labs (blood work), neurologic exam, clinical and cognitive assessments, DSRD workup
Visit 7-9: Follow-ups (optional in-person or telephone follow-up visits at 1 month, 6 months, and 12 months after Endpoint visit)
12-point review of systems, review health history and medical records, physical and neurological exams (in-person only), safety and research labs (in-person only)
Eligibility
Age 8-30
Weight >40kg
Confirmed diagnosis of Down syndrome
Possible or probable diagnosis of DSRD per 2022 consensus
Agree to a washout of any medications intended to treat DSRD
Agree to random treatment assignment
No known allergies to lorazepam, IVIg, or tofacitinib
Not pregnant or breastfeeding
No past or current tobacco smoking
Be able to present with a parent or legal guardian to all study visits
No co-occurring conditions or medications/interventions (see full list at clinicaltrials.gov)
Note: Participants may be excluded for other unforeseen reasons or confounding reasons for DSRD symptoms at the study doctor’s discretion.
