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Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)

NCT07733856 · Status: AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2026-07-29

No results posted yet for this study

Summary

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.

Conditions

  • Mucopolysaccharidosis Type IIIB
  • MPS IIIB (Sanfilippo B Syndrome)

Interventions

DRUG

Tralesinidase alfa (TA)

Tralesinidase alfa (TA) is a sterile solution of recombinant human alpha-N-acetylglucosaminidase fused with insulin-like growth factor 2 (rhNAGLU-IGF2), formulated at 30 mg/mL for ICV infusion. Dose: 200 mg (6.7 mL) for participants age ≥12 to \<24 months; 300 mg (10 mL) for participants age ≥24 months. Administered once weekly via ICV reservoir following isovolumetric removal of up to 10 mL CSF, infused over approximately 10 minutes. Stored frozen at -40°C (±10°C).

Sponsors & Collaborators

  • Spruce Biosciences

    lead INDUSTRY

Eligibility

Min Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07733856 on ClinicalTrials.gov