Chicago, ILNCT06662188Now EnrollingIRB Ready

Phelan-McDermid Syndrome Clinical Trial in Chicago, IL

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

Sponsored by Jaguar Gene Therapy, LLC

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Expert Care in Chicago

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 Chicago, IL

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Why Participate?

  • No-Cost Study Care

  • Local to Chicago

    Convenient for IL 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 Chicago site if eligible
  4. 4Begin participation

About This Phelan-McDermid Syndrome Study in Chicago

This is a Phase 1/2, first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of JAG201 administered via intracerebroventricular (ICV) injection in pediatric and adult participants with SHANK3 haploinsufficiency resulting from SHANK3 loss of function mutations and chromosomal deletions encompassing the SHANK3 gene. Clinical data will be evaluated for safety, tolerability, and preliminary clinical activity of JAG201 in pediatri

Sponsor: Jaguar Gene Therapy, LLC

Who Can Participate

Inclusion Criteria

Is male or female, and 2 to 9 years of age at the time of JAG201 administration
Has a molecular confirmation of a loss of function mutation in SHANK3 or a 22q13.3 deletion classified as a Class I deletion
Has evidence of developmental/cognitive delay of at least 2 standard deviations (SD) below the mean (i.e., ≤ 70) via either Intelligence Quotient (IQ) OR Developmental Quotient (DQ) assessment (as applicable)
Has an overall Phelan-McDermid Syndrome (PMS) Assessment of Severity (PMSA-S) Score of 3 or greater at Screening
Willing to initiate structured therapies and continue for the duration of the study as determined by the specific therapist (structured therapies may include, at a minimum, physical therapy, occupational therapy, speech therapy, and applied behavior analysis)
Is stable on any medication regimens (if being administered to control the signs and symptoms of underlying disease) for at least 3 months prior to the planned JAG201 study treatment
If undergoing any kind of behavioral or therapeutic intervention, then the level of intervention must have remained stable for at least 3 months prior to the planned JAG201 study treatment (exclusive of school vacations/illness).
Is a permanent legal resident of the U.S. residing within the continental U.S. Key

Exclusion Criteria

A pediatric participant who meets any of the following criteria will be excluded from this study:
Has history of developmental regression defined in this study as a prolonged loss of previously acquired skills (defined as skills maintained for at least 3 months) with loss of skills persisting for at least 3 months
Has known or suspected prion disease (e.g., Creutzfeldt-Jakob Disease)
Has poorly-controlled epilepsy (defined as an increase in the dose or addition of new anti-epileptic medications within the past 3 months) or any history of status epilepticus or seizure-induced hospitalizations within the last 12 months
Has history of acute cerebrovascular episodes
Has active autoimmune disease or prior treatment with immunomodulatory therapy, immunotherapy, and/or immunosuppressive drugs within 3 months prior to study enrollment (Note: Inhaled or topical steroids are permitted in the absence of active autoimmune disease)
Has infection (viral, bacterial, or fungal) that requires treatment \< 6 weeks before JAG201 administration (Note: JAG201 administration may be postponed until the infection has resolved and the participant is clinically stable)
Has medical illness or other concern that would cause the Investigator to conclude that the participant will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessments
Has known allergy or hypersensitivity to prednisolone or other glucocorticosteroids, or their excipients
Has received any vaccine \< 6 weeks before JAG201 administration
Has received any gene therapy

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 Chicago?

Yes, this clinical trial (NCT06662188) has an active research site in Chicago, IL 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 Chicago, IL

If you're searching for phelan-mcdermid syndrome treatment options in Chicago, IL, this clinical trial (NCT06662188) 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 Chicago research site is actively enrolling participants for this phase 1/2 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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