Nashville, TNNCT06430385Now EnrollingIRB Ready

MECP2 Duplication Syndrome Clinical Trial in Nashville, TN

Access cutting-edge mecp2 duplication syndrome treatment through this phase 1/2 clinical trial at a research site in Nashville. Study-provided care at no cost to qualified participants.

Sponsored by Ionis Pharmaceuticals, Inc.

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Expert Care in Nashville

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 Nashville, TN

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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 Nashville

    Convenient for TN 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 Nashville site if eligible
  4. 4Begin participation

About This MECP2 Duplication Syndrome Study in Nashville

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:
Males aged ≥ 2 to ≤ 65 years, depending on specific cohort and group, at the time of informed consent.
Group A: ≥ 8 to ≤ 65 years old
Group B: 2 to 7 years old, inclusive
Participant has at least one parent or caregiver ≥ 18 years old capable of providing informed consent and able to comply with all study requirements and activities.
Participant has a documented diagnosis of MDS with genetic confirmation of MECP2 duplication.
Is currently receiving stable doses of concomitant medications for at least 1 month prior to screening.
Able to complete all study procedures, measurements and visits to support primary and secondary endpoints, in the opinion of the Investigator. Key

Exclusion Criteria

for Part 1:
Documented diagnosis of severe MECP2 duplications including terminal duplication and/or translocation or MECP2 triplication OR clinical features associated with severe variant structure including (a) onset of seizures prior to age 5 (for those aged 5 and above at signing of ICF), (b) oxygen dependence, (c) microcephaly, IF MECP2 genetic structure information is unavailable.
Clinically significant vital sign or ECG abnormality at Screening
Known brain or spinal disease that would interfere with the LP procedure, or CSF circulation or presence of other factors would affect the safety of the LP procedure.
Has any concomitant disease or condition or circumstance, or any finding at Screening that, in the opinion of the Investigator, makes the participant unsuitable for enrollment or that could interfere with the conduct of the study or that would pose an unacceptable risk to the participant in this study.
Treatment with an investigational drug, biological agent, or device within 30 days of Screening, or 5 half-lives of investigational agent, whichever is longer.
Previous treatment with an oligonucleotide (including siRNA) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received (this exclusion does not apply to vaccines - both mRNA and viral vector vaccines are allowed including COVID-19). For centrally administered ASOs, a minimum of 12 months washout is required irrespective of the number of doses received.
Currently enrolled in a clinical trial of an investigational agent or device or has used any investigational agent or device within 5 half-lives of investigational agent, whichever is longer.
Has a history of gene therapy or cell transplantation or any other experimental brain surgery.
Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Baseline (Day 1).
Has experienced Status Epilepticus in the past 6 months. Key Inclusion criteria for Part 2:
Participants in ION440-CS1, Part 1/MAD who received at least one dose of Study Drug /Sham in Part 1/MAD, missed no more than 1 study visit, and attended the Follow Up visit (Visit 6).
All inclusion criteria in Part 1/MAD apply (participants will not be required to undergo new Screening bloodwork). Key Exclusion criteria for Part 2: 1\. Has developed any concomitant disease (e.g., gastrointestinal, renal, hepatic, endocrine, respiratory, or cardiovascular system disease) or condition or circumstance, or any finding during Part 1/MAD that, in the opinion of the Investigator, makes the participant unsuitable for continued treatment (e.g., could interfere with the conduct of the study or that would pose an unacceptable risk to the participant in this 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 Nashville?

Yes, this clinical trial (NCT06430385) has an active research site in Nashville, TN 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 Nashville, TN

If you're searching for mecp2 duplication syndrome treatment options in Nashville, TN, 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 Nashville research site is actively enrolling participants for this phase 1/2 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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