The TrAnsform Study for MPS IIIB

A Phase 3 research study of an investigational treatment for select children with MPS IIIB.

The TrAnsform Study (Protocol 250-301) is a clinical research study. It is looking at how safe and how well an investigational treatment (treatment or drug that is not yet approved for the condition being studied), called tralesinidase alfa (TA), works in children with MPS IIIB. The study is sponsored by Spruce Biosciences.

What is tralesinidase alfa (TA)

Tralesinidase alfa (TA) is a form of the NAGLU (N-acetyl-alpha-glucosaminidase) enzyme that children with MPS IIIB are missing. It is given once a week, directly into the fluid around the brain, through a small, implanted device. If your child is eligible, the study team will explain the process of administration in greater detail.

Participation in this clinical trial means that you are helping the development of an investigational treatment for MPS IIIB.

Quick facts about the study

Here is a short summary of the TrAnsform Study. The rest of this page has more details.

  • Study name

    TrAnsform Study (Protocol 250-301)

  • Investigational treatment

    tralesinidase alfa (TA), given once a week over a 10 minute infusion

  • Who it is for

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

  • How long

    about 5 years (around 260 weeks)

  • Where

    study sites in several countries (see Locations below)

  • Sponsor

    Spruce Biosciences

What the study is trying to learn

The TrAnsform Study is trying to answer questions like:

  • How safe is TA for children with MPS IIIB?

  • How well is it tolerated when given each week over a long time?

  • How does it affect signs of MPS IIIB, including how children grow, learn, and behave?

Who can take part

Each clinical study has a list of “inclusion” rules (who may join) and “exclusion” rules (who may not). These rules help the study team learn what they need to learn, in the safest way possible for children. The list below is a simple summary. The study team will go over the full list with you.

Children may be able to take part if:

  • They have a confirmed diagnosis of MPS IIIB
  • They are between 1 and 5 years old at the start of the study
  • They have a BSID-II-C score less than 70
  • A parent or legal guardian gives written permission for them to take part
  • The family or care partner can come to regularly required study visits and follow the study plan

Children may not be eligible to take part if:

  • They have a health condition that makes the study’s surgical procedure deemed risky for them
  • They have seizures that are not well-controlled
  • They are taking part in another research study with an investigational drug
  • There is another reason the doctor feels the study is not a good fit for them

The best way to know if your child may be able to take part is to complete the sign-up form to determine eligibility and receive the latest updates. If the TrAnsform Study is a good fit, a study representative may contact you to discuss the study, answer your questions, and explain the next steps. Completing the form does not guarantee eligibility, study participation, or EAP access.

See if Your Child is Eligible icon-arrow-button

Where the study is open

The TrAnsform Study (Protocol 250-301) will be conducted in a few study sites around the world. Sites are planned in the following countries:

  • Argentina
  • Australia
  • Brazil
  • Germany
  • Saudi Arabia
  • Taiwan
  • Turkey
  • United States

The study may reimburse for certain travel costs.