Welcome to the TrAnsform Study

A clinical research study exploring a new potential treatment option for individuals living with Sanfilippo Syndrome Type B (MPS IIIB).

TrAnsform is the name of a clinical research study (Protocol 250-301) for an investigational treatment (a treatment which is not yet approved for the condition being studied) called tralesinidase alfa (TA) for children living with MPS IIIB.

With help from the MPS IIIB community, an Expanded Access Program (EAP) (Protocol 250-502) has also been created.

We know that families living with MPS IIIB face a long road. This site is here to help you learn about the TrAnsform Study and the EAP.

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What is MPS IIIB?

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The TrAnsform Study and Expanded Access Program

Spruce Biosciences is sponsoring two related opportunities for children with MPS IIIB. They are not the same. One is a clinical study, and the other is an Expanded Access Program (EAP). Each has eligibility rules that depends on your child’s age, their care needs, where you live, and other factors.

Protocol 250-301

A Phase 3 clinical research study

Considers the safety and efficacy of tralesinidase alfa (TA)

For children ages 1 to 5 with MPS IIIB with BSID-II-C score less than 70.

(BSID-II-C below 70 means that a child is performing in the bottom 2% compared to peers of the same-age, indicating significant developmental delays that may benefit from early intervention services.)

Being conducted at study sites in: Argentina, Australia, Brazil, Germany, Taiwan, Turkey, and United States.

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Protocol 250-502

A potential option for participants with MPS IIIB to receive the investigational treatment through a protocol (a defined set of plans and procedures) if they meet certain eligibility criteria

Only in the United States

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See if Your Child is Eligible

Meet Bruce from Spruce

Hi, I’m Bruce from Spruce, and I’m so happy to meet you and your child. I’ll be right here with you throughout the study or expanded access program while you navigate this journey.

See if Your Child is Eligible Find a study site near you Read the FAQs