Frequently asked questions

TrAnsform Study (Protocol 250-301) is a Phase 3 clinical research study for an investigational treatment (treatment or drug that is not yet approved for the condition being studied), tralesinidase alfa, for children with MPS IIIB. You can read more on the TrAnsform Study page.

Tralesinidase alfa is an investigational treatment. It is not yet approved by health authorities.

It is a form of the NAGLU (N-acetyl-alpha-glucosaminidase) enzyme that children with MPS IIIB are missing. It is given once a week through a small, implanted device into the fluid around the brain.

No, it is not a cure. Tralesinidase alfa is an investigational product in development for the treatment of MPS IIIB. TA is a form of the NAGLU (N-acetyl-alpha-glucosaminidase) enzyme that children with MPS IIIB are missing. The TrAnsform Study is being done to learn more about how safe it is and how it affects children with MPS IIIB.

TrAnsform is a controlled study. That means half of the children will get the investigational treatment and the other half will get the standard of care that children with MPS IIIB get today. The study team will explain how this works and answer your questions before you make a decision about participation.

The study is planned to last about 5 years (around 260 weeks). Visits are weekly during much of the study.

Submitting information does not guarantee access to the Expanded Access Program (EAP). The EAP is a limited U.S.-only pathway through which some children with MPS IIIB may be considered to access the investigational treatment outside the TrAnsform Study. If you have specific questions about eligibility, please talk with your child’s doctor about whether a clinical study or expanded access program is right for your family.

The study is for children with MPS IIIB who are between 1 and 5 years old. There are other rules, too. The study team can help you understand if your child may be eligible. Please see the “Who can take part” section on the TrAnsform Study page.

The TrAnsform Study is designed to carefully evaluate the investigational treatment and must strictly follow rules and protocols to support regulatory approval. If you have specific questions about eligibility, please talk with your child’s doctor about whether a clinical study or expanded access program is right for your family.

Yes. Spruce Biosciences also runs an Expanded Access Program (EAP), called Protocol 250-502, in the United States. The EAP is a potential option for some children with MPS IIIB who do not meet the rules for the TrAnsform Study. Please see our EAP page for more.

Eligibility and enrollment decisions are made by the clinical trial sites conducting the program. Exceptions to the study rules cannot be made. These requirements were made based on medical research and are reviewed by health authorities, ethics committees, and medical experts who oversee the study. Following these rules helps ensure the study is as safe as possible and can be used to support an application for regulatory approval.

You sign up by completing this form to learn more.

There is no cost and no commitment when you submit the form.

Anyone can sign up to learn more. To participate in the TrAnsform Study, you must live in or be willing to relocate to a country in which the study is being conducted (Argentina, Australia, Brazil, Germany, Saudi Arabia, Taiwan, Turkey, and the United States). The EAP is only available in the United States.

If the TrAnsform Study or EAP is a good fit, a study representative may contact you to discuss the study, answer your questions, and explain the next steps. Completing this form does not guarantee eligibility, study participation, or EAP access.

Yes. Healthcare providers, including physicians and researchers interested in learning more about the TrAnsform Study or the Expanded Access Program, are encouraged to contact Spruce Biosciences directly for medical and scientific information, site qualification details, or to discuss patient referrals.

There is no cost to receive the investigational treatment in the study. Other costs, like travel and lodging, will be discussed with the study site directly.

Yes, you may be reimbursed for certain travel costs if your child is participating in the TrAnsform study.

Visits are scheduled to be weekly at the same site. Visits can take a few hours. Some visits are longer, especially in the beginning. The site team will explain what to expect for each visit.

Yes. Participants receiving the investigational medicine in the study must have a surgical procedure to place a device that allows the treatment to be given each week. The surgery is completed by an experienced surgeon. The study team will go over the surgery process with you and answer your questions.

Like any research study or surgery, there are risks. These will be explained in detail before you decide to take part. The study team will go over the risks, the possible benefits, and what we do not yet know.

Your information is collected for the purpose of assessing preliminary eligibility for the TrAnsform Study and expanded access program. If your child appears to meet preliminary eligibility criteria, your contact information and health responses may be shared with the study sponsor, its contract research organization, and participating research sites to facilitate your potential enrollment. Your phone number is verified via a one-time SMS code sent by our service provider (Twilio). Your data is stored on servers located in the United States. You may withdraw your consent or request deletion of your data at any time via our Participant Privacy Portal. For full details, see our Privacy Policy