New York, NYNCT06199531Now EnrollingIRB Ready

NGLY1 Deficiency Clinical Trial in New York, NY

Access cutting-edge ngly1 deficiency treatment through this clinical trial at a research site in New York. Study-provided care at no cost to qualified participants.

Sponsored by Grace Science, LLC

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Expert Care in New York

Access ngly1 deficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related ngly1 deficiency treatment provided free

Apply for This New York Location

Check if you qualify for this ngly1 deficiency clinical trial in New York, NY

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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 New York

    Convenient for NY 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 New York site if eligible
  4. 4Begin participation

About This NGLY1 Deficiency Study in New York

A non-randomized, open-label, dose escalation study of a single intracerebroventricular (ICV) administration of a gene replacement therapy in subjects who are 2 to 18 years old with NGLY1 Deficiency.

Sponsor: Grace Science, LLC

Who Can Participate

Inclusion Criteria

Patients must be 2 to 18 years of age, inclusive, at the time of signing the informed consent form (ICF)
Patients with a documented diagnosis of NGLY1 Deficiency based on detection of biallelic variants in the NGLY1 gene via molecular genetic sequencing
Elevated GNA levels may be considered alongside genetic sequencing data and other clinical data to assist with diagnosis confirmation
Patients with two or more of the following clinical features typical of NGLY1 Deficiency: 1. Global developmental delay and/or intellectual disability 2. Hyperkinetic movement disorder 3. Transient elevation of transaminases 4. (Hypo)alacrima 5. Peripheral neuropathy
For patients with epilepsy who require anti-seizure medications for seizure control: must be on a stable regimen for 28 days prior to enrollment
Patients willing and capable per investigator opinion to comply with study procedures and requirements
Females of childbearing potential must have a nega

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 New York?

Yes, this clinical trial (NCT06199531) has an active research site in New York, NY 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.

NGLY1 Deficiency Treatment Options in New York, NY

If you're searching for ngly1 deficiency treatment options in New York, NY, this clinical trial (NCT06199531) 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 New York research site is actively enrolling participants for this clinical trial. You'll receive care from experienced ngly1 deficiency 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 ngly1 deficiency clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · New York, NY