Therapies for Down Syndrome Regression Disorder

Part of paid clinical trials in Los Angeles, California.

Sponsor
University of Colorado, Denver
Study ID
NCT05662228
Phase
PHASE2
Status
Active Not Recruiting

Conditions

  • Down Syndrome
  • Regression

Eligibility Criteria

Sex
ALL
Age
8 Years - 30 Years
Healthy Volunteers
Not accepted

Interventions

  • Lorazepam — DRUG
    Lorazepam will be administered as an oral pill over the first 15 days of study in a daily titration, starting at 0.5 mg BID and increasing to up to 2 mg three times daily, as tolerated. Dosing will continue at the maximum tolerated dose through the 12-week endpoint. Participants will be titrated off lorazepam over at least four weeks after completing the endpoint visit. Taper will be tailored to individuals for safety reasons with a goal of decreasing dosage by 25% weekly. Phone check ins will be conducted every three days to monitor patient.
  • Intravenous immunoglobulin (IVIG) — DRUG
    IVIG will be administered as a series of four intravenous infusions at a dose of 1 mg/kg with pre-infusion medications of 1 mg/kg diphenhydramine and 15 mg/kg acetaminophen. The first two infusions occur at baseline and one day after (induction dosing), followed by one infusion at 4 weeks and one infusion at 8 weeks.
  • Tofacitinib — DRUG
    Tofacitinib will be administered as an oral pill at 5 mg twice daily over the 12-week study.

Study Details

Individuals with Down syndrome (DS) have an increased risk of numerous co-occurring conditions, including the neuropsychiatric condition known as Down Syndrome Regression Disorder (DSRD). A DSRD diagnosis often includes a sub-acute onset of catatonia, mutism, depersonalization, loss of ability to perform activities of daily living, hallucinations, delusions, and aggression and is most commonly observed in adolescents and young adults. The study evaluates the safety and efficacy of three currently prescribed therapies: lorazepam, intravenous immunoglobulin (IVIG) and tofacitinib.

Key Dates

Start date
Jun 29, 2023
Status verified
Feb 2026
Primary completion
Dec 31, 2026
Completion
Dec 31, 2026

Study Design

Enrollment
66 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Lorazepam
    Participants will receive lorazepam as an oral pill three times daily for 12 weeks as well as titration doses for an additional 4 weeks (approximately).
  • Experimental: Intravenous immunoglobulin (IVIG)
    Participants will receive 4 doses of IVIG treatment over 12 weeks.
  • Experimental: Tofacitinib
    Tofacitinib will be administered as an oral pill at 5 mg twice daily over the 12-week study.

Primary Outcome Measure

Comparison of number and severity of all adverse events. [ Time Frame: Baseline to 14 weeks ]

Locations (2)

FacilityCityStateZIPSite coordinators
Children's Hospital Los AngelesLos AngelesCalifornia90027-
Children's Hospital ColoradoAuroraColorado80045-

Find similar trials in Los Angeles, CA

By research site
Children's Hospital Los Angeles· Los Angeles, CAChildren's Hospital Colorado· Aurora, CO

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