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New treatment option for SHANK3 Haploinsufficiency
Official title JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency
ClinicalTrials.gov ID: NCT06662188
What this study is testing
What is JAG201?
JAG201 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for shank3 haploinsufficiency.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 pediatric and adult participants with SHANK3 haploinsufficiency.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 9
You may be able to join if
- 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...
- 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...
You likely can't join if
- 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...
- 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...
- 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...
See the full eligibility 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...
- 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...
- Is a permanent legal resident of the U.S. residing within the continental U.S. Key
- 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...
- 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...
- 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...
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 9 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Chicago, Illinois, United States; Boston, Massachusetts, United States; New York, New York, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.