Atlanta, GANCT06150716Now EnrollingIRB Ready

Pelizaeus-Merzbacher Disease Clinical Trial in Atlanta, GA

Access cutting-edge pelizaeus-merzbacher disease treatment through this phase 1 clinical trial at a research site in Atlanta. Study-provided care at no cost to qualified participants.

Sponsored by Ionis Pharmaceuticals, Inc.

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Expert Care in Atlanta

Access pelizaeus-merzbacher disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related pelizaeus-merzbacher disease treatment provided free

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Check if you qualify for this pelizaeus-merzbacher disease clinical trial in Atlanta, GA

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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 Atlanta

    Convenient for GA 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 Atlanta site if eligible
  4. 4Begin participation

About This Pelizaeus-Merzbacher Disease Study in Atlanta

The primary purpose of this study is to evaluate the safety and tolerability of ION356.

Sponsor: Ionis Pharmaceuticals, Inc.

Who Can Participate

Inclusion Criteria

Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback regarding the participant's symptoms, and can comply with all study requirements.
Diagnosis of PMD with genetic confirmation of PLP1 gene duplication.
Clinical phenotype and brain imaging consistent with a diagnosis of PMD.
Male between the ages of 2 and 17 years, inclusive, at the time of informed consent.
Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and visits.

Exclusion Criteria

Clinically significant abnormalities in medical history, laboratory tests or physical examination.
Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator.
Any contraindication or unwillingness to undergo magnetic resonance imaging (MRI).
Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of the investigational agent, whichever is longer.
Previous treatment with an oligonucleotide (including small interfering ribonucleic acid) within 4 months of Screening if a single dose was received, or within 12 months of Screening if multiple doses were received. This exclusion does not apply to vaccines (both messenger ribonucleic acid \[mRNA\] and viral vector vaccines).
History of gene therapy or cell transplantation, or any experimental brain surgery.
Current obstructive hydrocephalus.
Known brain or spinal disease or previous spinal surgery that would interfere with the lumbar puncture (LP) process, CSF circulation, or safety assessment.
Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study.
Have any other conditions, which, in the opinion of the Investigator, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.

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 Atlanta?

Yes, this clinical trial (NCT06150716) has an active research site in Atlanta, GA 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.

Pelizaeus-Merzbacher Disease Treatment Options in Atlanta, GA

If you're searching for pelizaeus-merzbacher disease treatment options in Atlanta, GA, this clinical trial (NCT06150716) 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 Atlanta research site is actively enrolling participants for this phase 1 clinical trial. You'll receive care from experienced pelizaeus-merzbacher 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 pelizaeus-merzbacher disease clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Atlanta, GA