The Expanded Access Program

A way for some participants with MPS IIIB to receive the investigational treatment outside of the TrAnsform Study.

Only available in the United States.

With help from the MPS IIIB community, Spruce Biosciences offers an Expanded Access Program (EAP) for some children with MPS IIIB. The program is called Protocol 250-502. It gives early access to the investigational treatment, tralesinidase alfa (TA), for some participants.

The EAP is not the same as the TrAnsform Study. It is open only in the United States. It is meant to provide limited treatment access outside of TrAnsform until the treatment becomes commercially accessible (if approved).

If the EAP is a good fit, a study representative may contact you to discuss the study, answer your questions, and explain the next steps after you sign up.

Quick Facts about EAP

  • Study name

    Expanded Access Program (Protocol 250-502)

  • Investigational treatment

    Tralesinidase alfa, given once a week

  • Who it is for

    Participants with MPS IIIB

  • Where

    study sites in the United States only

  • Number of children

    about 10

  • How long

    up to about 1 year, or until tralesinidase alfa becomes commercially accessible for the participants (whichever happens first)

  • Sponsor

    Spruce Biosciences with support from the MPS IIIB community

Who the EAP is for

The EAP is for up to 10 participants with MPS IIIB.

How is the EAP different from the TrAnsform Study?

Both opportunities use the same investigational treatment, tralesinidase alfa (TA). They have many things in common. But they are not the same. The table below shows some of the main differences.

A Phase 3 clinical research study.
Type
An Expanded Access Program.
Mainly designed to study safety and efficacy.
Goal
Mainly designed to give access to the treatment.
Open in several countries.
Where
Open only in the United States.
Plans to enroll up to about 14 children.
Number of children
Plans to enroll about 10 children.
Lasts about 5 years.
Length
Lasts about 1 year, or until the treatment becomes commercially accessible.
Has its own list of rules.
Who it is for
Has its own list of rules.