Pittsburgh, PANCT02716246Now EnrollingIRB Ready

Sanfilippo Syndrome Clinical Trial in Pittsburgh, PA

Access cutting-edge sanfilippo syndrome treatment through this clinical trial at a research site in Pittsburgh. Study-provided care at no cost to qualified participants.

Sponsored by Ultragenyx Pharmaceutical Inc

Quick Self-Assessment

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Expert Care in Pittsburgh

Access sanfilippo syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related sanfilippo syndrome treatment provided free

Apply for This Pittsburgh Location

Check if you qualify for this sanfilippo syndrome clinical trial in Pittsburgh, PA

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

Why Participate?

  • No-Cost Study Care

  • Local to Pittsburgh

    Convenient for PA 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 Pittsburgh site if eligible
  4. 4Begin participation

About This Sanfilippo Syndrome Study in Pittsburgh

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

Sponsor: Ultragenyx Pharmaceutical Inc

Who Can Participate

Inclusion Criteria

Diagnosis of MPS IIIA confirmed by the following methods:
No detectable or significantly reduced SGSH enzyme activity by leukocyte assay, and
Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene (based upon review of documented results from a qualified laboratory, and with confirmation with Medical Monitor)
For Cohort 1-3: From birth (participating sites in USA and Australia) OR 6 months (participating sites in Spain) to 2 years of age with no BSITD-III Cognitive Development Quotient (DQ) requirement, or older than 2 years with a BSITD-III Cognitive DQ of 60 or above (participating sites globally).
For Cohort 4 (participating sites in Spain): 3 months to ≤ 2 years of age with no BSITD-III Cognitive DQ requirement, or \> 2 years of age with a BSITD-III Cognitive DQ of 60 or above at Screening Visit 1. Up to 2 additional patients \> 2 years and ≤ 5 years of age with a BSITD-III Cognitive DQ \< 60

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 Pittsburgh?

Yes, this clinical trial (NCT02716246) has an active research site in Pittsburgh, PA 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.

Sanfilippo Syndrome Treatment Options in Pittsburgh, PA

If you're searching for sanfilippo syndrome treatment options in Pittsburgh, PA, this clinical trial (NCT02716246) 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 Pittsburgh research site is actively enrolling participants for this clinical trial. You'll receive care from experienced sanfilippo syndrome 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 sanfilippo syndrome clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Pittsburgh, PA