Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)
Public ClinicalTrials.gov record NCT07733856. 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.
Official title
Open-Label Early Access Treatment With Intracerebroventricular Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is an intermediate-size patient population Early Access Program (EAP) providing access to intracerebroventricular (ICV) tralesinidase alfa (TA) for participants with mucopolysaccharidosis type IIIB (MPS IIIB, Sanfilippo Syndrome Type B). The primary objectives are to allow early access to TA treatment and to evaluate the safety and tolerability of TA ICV infusion. Approximately 10 participants will be enrolled at up to 3 US sites and treated with TA once weekly via ICV infusion for up to approximately 52 weeks, or until TA becomes commercially available.
Study identification
- NCT ID
- NCT07733856
- Recruitment status
- Available
- Study type
- Expanded access
- Phase
- Not listed
- Enrollment
- Not listed
Conditions and interventions
Eligibility (public fields only)
- Age range
- 12 Months and older
- Sex
- All
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Not listed
- Primary completion
- Not listed
- Completion
- Not listed
- Last update posted
- Oct 6, 2026
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Oakland Research Institution | Oakland | California | 94609 | Available |
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 NCT07733856, 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 Oct 6, 2026 · Synced Oct 7, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT07733856 live on ClinicalTrials.gov.