Lexington, MANCT07281079Now EnrollingIRB Ready

Phelan-McDermid Syndrome Clinical Trial in Lexington, MA

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

Sponsored by Neuren Pharmaceuticals Limited

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Expert Care in Lexington

Access phelan-mcdermid syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related phelan-mcdermid syndrome treatment provided free

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Check if you qualify for this phelan-mcdermid syndrome clinical trial in Lexington, MA

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Why Participate?

  • No-Cost Study Care

  • Local to Lexington

    Convenient for MA 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 Lexington site if eligible
  4. 4Begin participation

About This Phelan-McDermid Syndrome Study in Lexington

This Phase 3, randomized, double-blind, parallel-group (2-arm), placebo-controlled, multicenter study will evaluate the efficacy and safety of NNZ-2591 compared to placebo in pediatric participants with Phelan- McDermid Syndrome.

Sponsor: Neuren Pharmaceuticals Limited

Who Can Participate

Inclusion Criteria

Male or female pediatric participants with Phelan-McDermid syndrome ages 3 to 12 years (inclusive) at the time of signing the informed consent.
Clinical diagnosis of Phelan-McDermid syndrome with a documented disease-causing genetic abnormality of SHANK3.
Body weight ≥ 10 kg at Screening.
Participants with a PMSA-S overall score ≥ 3 at the Screening and Baseline visits.
Not actively undergoing regression or loss of skills.

Exclusion Criteria

Use of exclusionary medication or unstable treatment regimens of acceptable concomitant medications as required by the protocol.
Current treatment with more than 3 allowable psychotropic medications.
Participants with seizures must be controlled on no more than 2 anticonvulsant medications (not counting rescue medications).
Psychotropic medications or any other medication used for a chronic illness (not including antibiotics, pain relievers, anti-diarrheals, and laxatives) with doses and dosing regimen that have not been stable for at least 4 weeks before Screening. If the treatment was discontinued, the discontinuation must have occurred no fewer than 2 weeks before the start of Screening.
Any intercurrent seizures in the past 6 months and /or more than 1 seizure in the past 12 months. •A single febrile seizure in the 6 months prior to screening is allowable if no rescue medication was required.
Abnormal liver function laboratory results during the Screening period, as defined by the protocol
Abnormal QT interval on Screening ECG as defined by the protocol.

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 Lexington?

Yes, this clinical trial (NCT07281079) has an active research site in Lexington, MA 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.

Phelan-McDermid Syndrome Treatment Options in Lexington, MA

If you're searching for phelan-mcdermid syndrome treatment options in Lexington, MA, this clinical trial (NCT07281079) 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 Lexington research site is actively enrolling participants for this clinical trial. You'll receive care from experienced phelan-mcdermid 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 phelan-mcdermid syndrome clinical trials near you to find additional studies recruiting in your area.

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