NCT07579910

Brief Summary

The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_3

Timeline
81mo left

Started Dec 2026

Longer than P75 for phase_3

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 28, 2026

Completed
14 days until next milestone

First Posted

Study publicly available on registry

May 12, 2026

Completed
7 months until next milestone

Study Start

First participant enrolled

December 1, 2026

Expected
6.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2033

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2033

Last Updated

May 12, 2026

Status Verified

May 1, 2026

Enrollment Period

6.7 years

First QC Date

April 28, 2026

Last Update Submit

May 6, 2026

Conditions

MPS IIIB

Outcome Measures

Primary Outcomes (1)

  • Change from Baseline in Bayley Scales of Infant and Toddler Development, Third Edition, Cognition Domain (BSID-III-C) Raw Score

    The BSID-III-C is a standardized tool that evaluates cognitive development in young children, capturing skills such as processing speed, problem solving, and play. Raw scores are used to sensitively measure individual change over time, independent of age. Assessments are administered by a trained, blinded rater and reviewed by a central blinded rater. The endpoint is the difference in mean change from baseline in BSID-III-C raw score between the tralesinidase alfa and control arms at Week 260

    Baseline to Week 260 (approximately 5 years)

Study Arms (2)

Tralesinidase alfa (TA)

EXPERIMENTAL
Drug: Tralesinidase alfa (TA)

Control

NO INTERVENTION

Interventions

Tralesinidase alfa (TA)DRUG

TA study drug is a sterile solution for ICV infusion.

Tralesinidase alfa (TA)

Eligibility Criteria

Age1 Year - 5 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Has a diagnosis of MPS IIIB confirmed by deficient NAGLU enzyme activity during screening.
  • Has nonattenuated severe MPS IIIB by the Genotype and Clinical Assessment Committee.
  • Is ≥1 and ≤5 years of age with BSID-III-C raw score of \<70. Is male or female as identified at birth.

You may not qualify if:

  • Genotyped for a known MPS IIIB variant associated with an attenuated phenotype or has a sibling with a known attenuated phenotype.
  • Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry.
  • Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Mucopolysaccharidosis III

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 28, 2026

First Posted

May 12, 2026

Study Start (Estimated)

December 1, 2026

Primary Completion (Estimated)

August 1, 2033

Study Completion (Estimated)

August 1, 2033

Last Updated

May 12, 2026

Record last verified: 2026-05