An Open-Label Phase II Feasibility Study for the Use of Ublituximab in Adults With Down Syndrome Regression Disorder

Part of paid clinical trials in Los Angeles, California.

Sponsor
Jonathan Santoro
Study ID
NCT07812181
Phase
PHASE2
Status
Not Yet Recruiting

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Conditions

  • Down Syndrome (Trisomy 21)
  • Down Syndrome Regression Disorder (DSRD)
  • Neuroinflammatory / Neuropsychiatric Disorder

Eligibility Criteria

Sex
ALL
Age
18 Years - 40 Years
Healthy Volunteers
Not accepted

Interventions

  • Ublituximab — DRUG
    Participants assigned to the Ublituximab arm will receive a fixed two-dose intravenous regimen consisting of 150 mg on Day 1 and 450 mg on Day 15. All participants will be followed for 24 weeks with regular safety monitoring, clinical assessments, and evaluation of cognitive, neuropsychiatric, and functional outcomes.

Study Details

The goal of this clinical trial is to evaluate whether the monoclonal antibody ublituximab can treat Down Syndrome Regression Disorder (DSRD) by assessing its safety, tolerability, and preliminary efficacy in affected individuals. This study is conducted in adults aged 18-40 years with Down syndrome who have DSRD and have had an inadequate or partial response to first-line therapies. The main questions it aims to answer are: * Does ublituximab demonstrate acceptable safety and tolerability, as measured by treatment-emergent adverse events from baseline through Week 24? * Does ublituximab lead to improvement in cognitive, neuropsychiatric, motor, and functional outcomes from baseline to Weeks 12 and 24? This is a single-arm study, so there is no comparison group. Participants will: * Receive two intravenous infusions of ublituximab (150 mg on Day 1 and 450 mg on Day 15) * Attend study visits at Screening, Baseline (Day 1) , Week 2 (Day 15), Week 3, Week 6, Week 12, and Week 24 * Undergo clinical assessments, including neurological and physical exams and safety monitoring labs throughout the study * Complete cognitive, behavioral, and functional evaluations at Baseline, Week 12, and Week 24 * Provide blood samples for safety monitoring and research biomarker analyses

Key Dates

First listed
Sep 10, 2026
Start date
Oct 1, 2026
Status verified
Sep 2026
Primary completion
Oct 1, 2030
Completion
Dec 1, 2030

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Ublituximab
    Participants assigned to the Ublituximab arm will receive a fixed two-dose intravenous regimen consisting of 150 mg on Day 1 and 450 mg on Day 15. All participants will be followed for 24 weeks with regular safety monitoring, clinical assessments, and evaluation of cognitive, neuropsychiatric, and functional outcomes.

Primary Outcome Measure

Total AEs on Ublituximab [ Time Frame: Baseline through Week 24 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital Los AngelesLos AngelesCalifornia90027
Mariam Yousuf
[email protected]
323-607-3505
Samuel Otey
[email protected]
323-607-3505
Jonathan Santoro, MD (PRINCIPAL_INVESTIGATOR)

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By research site
Children's Hospital Los Angeles· Los Angeles, CA

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