Philadelphia, PANCT06430385Now EnrollingIRB Ready

MECP2 Duplication Syndrome Clinical Trial in Philadelphia, PA

Access cutting-edge mecp2 duplication syndrome treatment through this clinical trial at a research site in Philadelphia. Study-provided care at no cost to qualified participants.

Sponsored by Ionis Pharmaceuticals, Inc.

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Expert Care in Philadelphia

Access mecp2 duplication syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related mecp2 duplication syndrome treatment provided free

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Check if you qualify for this mecp2 duplication syndrome clinical trial in Philadelphia, 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 Philadelphia

    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 Philadelphia site if eligible
  4. 4Begin participation

About This MECP2 Duplication Syndrome Study in Philadelphia

The primary purpose of this study is to evaluate the safety and tolerability of ION440.

Sponsor: Ionis Pharmaceuticals, Inc.

Who Can Participate

Inclusion Criteria

for Part 1: 1. Males age ≥ 2 years to ≤ 65 years old, depending on specific cohort and group, at the time of informed consent. 1. Group A: ≥ 8 to ≤ 65 years old 2. Group B: 2 to 7 years old, inclusive 2. Participant has at least one parent or caregiver ≥ 18 years old capable of providing informed consent (signed and dated), and able to attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol, and able to comply with all study requirements and activities, 3. Participant has a documented diagnosis of MDS, with genetic confirmation of MECP2 duplication 4. Is currently receiving stable doses of concomitant medications for at least 3 months prior to baseline. If recent changes (\< 3 months stable) in medications, the participant may be allowed per Investigator judgment in consult with Sponsor Medical Monitor. 5. Able to complete all study procedures, measurements and visits and caregiv

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 Philadelphia?

Yes, this clinical trial (NCT06430385) has an active research site in Philadelphia, 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.

MECP2 Duplication Syndrome Treatment Options in Philadelphia, PA

If you're searching for mecp2 duplication syndrome treatment options in Philadelphia, PA, this clinical trial (NCT06430385) 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 Philadelphia research site is actively enrolling participants for this clinical trial. You'll receive care from experienced mecp2 duplication 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 mecp2 duplication syndrome clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Philadelphia, PA