Oakland, CANCT07640984Now EnrollingIRB Ready

Mucopolysaccharidosis IIIB Clinical Trial in Oakland, CA

Access cutting-edge mucopolysaccharidosis iiib treatment through this phase 1/2 clinical trial at a research site in Oakland. Study-provided care at no cost to qualified participants.

Sponsored by JCR Pharmaceuticals Co., Ltd.

Quick Self-Assessment

See if you qualify for this Oakland location

Preparing your pre-screening questions…

Expert Care in Oakland

Access mucopolysaccharidosis iiib specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related mucopolysaccharidosis iiib treatment provided free

Apply for This Oakland Location

Check if you qualify for this mucopolysaccharidosis iiib clinical trial in Oakland, CA

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Oakland

    Convenient for CA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Oakland site if eligible
  4. 4Begin participation

About This Mucopolysaccharidosis IIIB Study in Oakland

This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.

Sponsor: JCR Pharmaceuticals Co., Ltd.

Who Can Participate

Inclusion Criteria

Patients with confirmed MPS IIIB with a phenotype of MPS IIIB that is not classified as slowly progressive (i.e., not attenuated).
A chronological age \<6 years.
Ability to complete and achieve a Standard score ≥55, using either Bailey Scale of Infant Development-4 (BSID-4) Cognitive domain or Kaufman Assessment Battery for Children-II (KABC-II) Non-Verbal Index, whichever tool is most appropriate for the participant's chronological age.

Exclusion Criteria

Contraindication for lumbar puncture or MRI.
A participant who has a medical condition or extenuating circumstance that, in the opinion of the principal investigator or sub-investigator, might compromise the participant's ability to comply with protocol requirements, the participant's well-being or safety, or the interpretability of the participant's clinical data.
A participant who has received any other investigational drug product (including but not limited to, tralesinidase alfa enzyme replacement therapy \[TA-ERT\], Genistein, KINERET \[anakinra\], ambroxol, miglustat) within 4 months (or 5 half-lives, whichever is longer) before the time of providing informed consent.
A participant who has received gene therapy treatment or hematopoietic stem cell transplantation (HSCT) with successful engraftment.
Serious drug allergy or hypersensitivity to any components of JR-446 or medications likely prescribed during the trial.
A participant has a history of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture.
A patient with recurrent epileptic seizures not adequately controlled with anti-seizure medication, and which, in the clinical judgment of the principal investigator, would preclude safe participation in the trial.
Serology consistent with human immunodeficiency virus (HIV) exposure or consistent with active hepatitis B (HepB) or hepatitis C (HepC) infection.
A participant/family, who, in the opinion of the investigator, may not be able to comply with protocol requirements and cooperate fully with the trial assessments, procedures, and scheduling for JR-446 IMP dose administrations.
Study participants for whom informed consent is unable to be provided by a parent or legal guardian; or when applicable for a study participant who is unable to provide assent with respect to study participation in conjunction with parental or legal guardian consent for participation on study. The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Oakland?

Yes, this clinical trial (NCT07640984) has an active research site in Oakland, CA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Mucopolysaccharidosis IIIB Treatment Options in Oakland, CA

If you're searching for mucopolysaccharidosis iiib treatment options in Oakland, CA, this clinical trial (NCT07640984) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Oakland research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced mucopolysaccharidosis iiib specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all mucopolysaccharidosis iiib clinical trials near you to find additional studies recruiting in your area.

More Ulcerative Colitis Trials in Oakland, CA

See all ulcerative colitis clinical trials recruiting in Oakland — not just this study.

Browse Ulcerative Colitis Trials in Oakland →

Browse More Trials by Condition

Ulcerative Colitis TrialsAll Conditions →

Ready to Join in Oakland?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Oakland, CA