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This is a 5-week, multi-center, open-label, dose optimization trial in subjects aged 12-17 years with 22q11DS who have a diagnosis of anxiety disorder, and/or ADHD, and/or ASD. Approximately 12 subjects will be initiated, dose optimized, and maintained on NFC-1 over a period of 5 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NFC-1 | Experimental | Doses of NFC-1 will be administered as 50, 100, 200, or 400 mg twice daily as capsules for oral administration. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NFC-1 | Drug | Doses of NFC-1 will be administered as 50, 100, 200, or 400 mg twice daily as size 2, hard gelatin capsules for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Time to symptom relapse | Through study completion, up to 5 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Response to treatment based on Clinical Global Impression - Improvement scale | Through study completion, up to 5 weeks | |
| Safety and tolerability as determined by AEs, laboratory results, C-SSRS, and K-SADS | Through study completion, up to 5 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37120522 | Derived | Glessner JT, Khan ME, Chang X, Liu Y, Otieno FG, Lemma M, Slaby I, Hain H, Mentch F, Li J, Kao C, Sleiman PMA, March ME, Connolly J, Hakonarson H. Rare recurrent copy number variations in metabotropic glutamate receptor interacting genes in children with neurodevelopmental disorders. J Neurodev Disord. 2023 Apr 29;15(1):14. doi: 10.1186/s11689-023-09483-z. | |
| 35690720 |
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| ID | Term |
|---|---|
| D004062 | DiGeorge Syndrome |
| ID | Term |
|---|---|
| D058165 | 22q11 Deletion Syndrome |
| D019465 | Craniofacial Abnormalities |
| D009139 | Musculoskeletal Abnormalities |
| D009140 | Musculoskeletal Diseases |
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| Slaby I, Hain HS, Abrams D, Mentch FD, Glessner JT, Sleiman PMA, Hakonarson H. An electronic health record (EHR) phenotype algorithm to identify patients with attention deficit hyperactivity disorders (ADHD) and psychiatric comorbidities. J Neurodev Disord. 2022 Jun 11;14(1):37. doi: 10.1186/s11689-022-09447-9. |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D006331 | Heart Diseases |
| D044148 | Lymphatic Abnormalities |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D007011 | Hypoparathyroidism |
| D010279 | Parathyroid Diseases |
| D004700 | Endocrine System Diseases |