San Rafael, CANCT07593391Now EnrollingIRB Ready

Phelan-McDermid Syndrome Clinical Trial in San Rafael, CA

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

Sponsored by Neuren Pharmaceuticals Limited

Quick Self-Assessment

See if you qualify for this San Rafael location

Preparing your pre-screening questions…

Expert Care in San Rafael

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

Apply for This San Rafael Location

Check if you qualify for this phelan-mcdermid syndrome clinical trial in San Rafael, CA

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to San Rafael

    Convenient for CA 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 San Rafael site if eligible
  4. 4Begin participation

About This Phelan-McDermid Syndrome Study in San Rafael

This Phase 3, open-label extension, multicenter study will evaluate long-term safety, tolerability and efficacy of NNZ-2591 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 for the antecedent study.
Participant must have completed all applicable study visits for the antecedent study in which they participated.
Body weight ≥ 10 kg at Screening/Baseline.
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.
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 San Rafael?

Yes, this clinical trial (NCT07593391) has an active research site in San Rafael, CA 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 San Rafael, CA

If you're searching for phelan-mcdermid syndrome treatment options in San Rafael, CA, this clinical trial (NCT07593391) 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 San Rafael 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.

More Heart Attack Trials in San Rafael, CA

See all heart attack clinical trials recruiting in San Rafael — not just this study.

Browse Heart Attack Trials in San Rafael →

Browse More Trials by Condition

Heart Attack TrialsPulmonary Hypertension TrialsMyocardial Infarction TrialsMyalgic Encephalomyelitis TrialsAll Conditions →

Ready to Join in San Rafael?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · San Rafael, CA