RECRUITING

JAG201 Gene Therapy Study in Children & Adults with SHANK3 Haploinsufficiency

Conditions

SHANK3 Haploinsufficiency Phelan-McDermid Syndrome SHANK3

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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. The pediatric cohorts will start enrolling first and the enrollment for adult cohorts may be initiated at a later timepoint in the study.

Official Title

A Phase 1/2, Multicenter, Open-Label, Dose-Escalation, Safety, Tolerability, and Clinical Activity Study of a Single Dose of JAG201 Gene Therapy Delivered Via Intracerebroventricular Administration in Participants with SHANK3 Haploinsufficiency

Quick Facts

Study Start:2024-01-07

Study Completion:2031-06

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06662188

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 9 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
1. Is male or female, and 2 to 9 years of age at the time of JAG201 administration 2. Has a molecular confirmation of a loss of function mutation in SHANK3 or a 22q13.3 deletion classified as a Class I deletion 3. 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) 4. Has an overall Phelan-McDermid Syndrome (PMS)-specific Clinical Global Impression-Severity (CGI-S) Score of 3 or greater at Screening 5. 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, and speech therapy) 6. 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 7. 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).	1. Has history of developmental regression defined as losing a skill previously maintained consistently for ≥ 3 months 2. Has known or suspected prion disease (e.g., Creutzfeldt-Jakob Disease) 3. 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 4. Has history of acute cerebrovascular episodes 5. 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) 6. 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) 7. 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 8. Has known allergy or hypersensitivity to prednisolone or other glucocorticosteroids, or their excipients 9. Has received any vaccine \< 6 weeks before JAG201 administration 10. Has received any gene therapy

Inclusion Criteria

Exclusion Criteria

1. Is male or female, and 2 to 9 years of age at the time of JAG201 administration
2. Has a molecular confirmation of a loss of function mutation in SHANK3 or a 22q13.3 deletion classified as a Class I deletion
3. 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)
4. Has an overall Phelan-McDermid Syndrome (PMS)-specific Clinical Global Impression-Severity (CGI-S) Score of 3 or greater at Screening
5. 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, and speech therapy)
6. 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
7. 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).

1. Has history of developmental regression defined as losing a skill previously maintained consistently for ≥ 3 months
2. Has known or suspected prion disease (e.g., Creutzfeldt-Jakob Disease)
3. 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
4. Has history of acute cerebrovascular episodes
5. 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)
6. 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)
7. 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
8. Has known allergy or hypersensitivity to prednisolone or other glucocorticosteroids, or their excipients
9. Has received any vaccine \< 6 weeks before JAG201 administration
10. Has received any gene therapy

Contacts and Locations

Study Contact

Jaguar Gene Therapy

CONTACT

224-303-0701

medinfo@jaguargenetherapy.com

Principal Investigator

Dan Gallo, PhD

STUDY_DIRECTOR

Jaguar Gene Therapy

Study Locations (Sites)

Rush University

Chicago, Illinois, 60612

United States

Seaver Autism Center at Mount Sinai

New York City, New York, 10029

United States

Collaborators and Investigators

Sponsor: Jaguar Gene Therapy, LLC

Dan Gallo, PhD, STUDY_DIRECTOR, Jaguar Gene Therapy

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-01-07

Study Completion Date2031-06

Study Record Updates

Study Start Date2024-01-07

Study Completion Date2031-06

Terms related to this study

Keywords Provided by Researchers

SHANK3 Haploinsufficiency
SHANK3
Phelan-McDermid Syndrome

Additional Relevant MeSH Terms

SHANK3 Haploinsufficiency
Phelan-McDermid Syndrome