Fairfax, VANCT07082725Now EnrollingIRB Ready

Niemann-Pick Disease Clinical Trial in Fairfax, VA

Access cutting-edge niemann-pick disease treatment through this phase 3 clinical trial at a research site in Fairfax. Study-provided care at no cost to qualified participants.

Sponsored by Azafaros A.G.

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Expert Care in Fairfax

Access niemann-pick disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related niemann-pick disease treatment provided free

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Check if you qualify for this niemann-pick disease clinical trial in Fairfax, VA

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Why Participate?

  • No-Cost Study Care

  • Local to Fairfax

    Convenient for VA 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 Fairfax site if eligible
  4. 4Begin participation

About This Niemann-Pick Disease Study in Fairfax

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 Niemann-Pick type C disease

Sponsor: Azafaros A.G.

Who Can Participate

Inclusion Criteria

Signed informed consent
Confirmed diagnosis of NPC disease
Patient is unable or unwilling to take miglustat, or is, in the opinion of the investigator, unsatisfactorily treated with miglustat
Male and female participants aged 4 years and older at the time of informed consent
Onset of neurological symptoms from 2 to 15 years
Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline
Female 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 NPC disease 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-epileptic treatment(s) was not stable (required a change in dose within the previous 3 months) and/or a new anti-epileptic treatment (drug or procedure) was prescribed in the month before Baseline
Prior use of an investigational drug within the 3 months before Screening; or prior participation in a clinical study involving gene therapy or stem cell transplantation within 2 years prior to Screening
A positive serum pregnancy test (for women of childbearing potential)
Current treatment with miglustat, provided the patient has been using the recommended dose for most of the past 12 months AND is, in the opinion of the investigator, satisfactorily treated with miglustat. Any participants receiving miglustat are required to undergo a 1-month washout period before starting study medication

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 Fairfax?

Yes, this clinical trial (NCT07082725) has an active research site in Fairfax, VA 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.

Niemann-Pick Disease Treatment Options in Fairfax, VA

If you're searching for niemann-pick disease treatment options in Fairfax, VA, this clinical trial (NCT07082725) 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 Fairfax research site is actively enrolling participants for this phase 3 clinical trial. You'll receive care from experienced niemann-pick disease 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 niemann-pick disease clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Fairfax, VA