Rochester, MNNCT07082543Now EnrollingIRB Ready

GM1 Gangliosidosis Clinical Trial in Rochester, MN

Access cutting-edge gm1 gangliosidosis treatment through this clinical trial at a research site in Rochester. Study-provided care at no cost to qualified participants.

Sponsored by Azafaros A.G.

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Expert Care in Rochester

Access gm1 gangliosidosis specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related gm1 gangliosidosis treatment provided free

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Check if you qualify for this gm1 gangliosidosis clinical trial in Rochester, MN

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Why Participate?

  • No-Cost Study Care

  • Local to Rochester

    Convenient for MN 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 Rochester site if eligible
  4. 4Begin participation

About This GM1 Gangliosidosis Study in Rochester

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis

Sponsor: Azafaros A.G.

Who Can Participate

Inclusion Criteria

Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant
Male and female participants aged 4 years and older at the time of informed consent
Onset of neurological symptoms from 1 to 10 years
Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline
Females of childbearing potential who are sexually active willing to follow the contraceptive guidance
Male participants with a female partner of childbearing potential willing to follow the contraceptive guidance

Exclusion Criteria

A history of medical conditions other than GM1 or GM2 gangliosidosis that, in the opinion of the Principal Investigator, would confound scientific rigor or the interpretation of results
Body weight of \<10 kg
The presence of another neurologic disease
The presence of moderate or severe hepatic impairment
The presence of moderate or severe renal impairment
Platelet count of \<100x10\^9/L
The dose of any anti-ep

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 Rochester?

Yes, this clinical trial (NCT07082543) has an active research site in Rochester, MN 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.

GM1 Gangliosidosis Treatment Options in Rochester, MN

If you're searching for gm1 gangliosidosis treatment options in Rochester, MN, this clinical trial (NCT07082543) 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 Rochester research site is actively enrolling participants for this clinical trial. You'll receive care from experienced gm1 gangliosidosis 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 gm1 gangliosidosis clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Rochester, MN