An Open-Label Phase II Feasibility Study for the Use of Ublituximab in Adults With Down Syndrome Regression Disorder
Public ClinicalTrials.gov record NCT07812181. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT07812181
- Recruitment status
- Not yet recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 20 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 40 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 30, 2026
- Primary completion
- Sep 30, 2030
- Completion
- Nov 30, 2030
- Last update posted
- Sep 9, 2026
2026 – 2030
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07812181, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 9, 2026 · Synced Sep 11, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07812181 live on ClinicalTrials.gov.