NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
Condition: 22q11 Deletion Syndrome
MeSH terms: DiGeorge Syndrome; 22q11 Deletion Syndrome; Syndrome
Study type: Interventional
Phase: Phase 2

Summary: 
      This is a Phase 2, randomized, placebo-controlled crossover trial to assess the safety and
      efficacy of NB-001 in children and adolescents with 22q11DS that manifest commonly associated
      neuropsychiatric conditions.
    

Detailed description: 
      The trial is designed to allow all visits to be conducted via telephone and/or video (i.e.,
      telemedicine) or by home health nurse. An in-person visit is required at Screening unless
      site or government mandates restrict this due to coronavirus disease-2019 (COVID-19). Other
      in-person visit(s) may occur, if indicated, based on the Investigator's clinical judgement.
      Subjects will be screened to confirm eligibility and then randomized in a 1:1 ratio to one of
      two treatment sequences: NB-001 (active drug product) followed by placebo (treatment sequence
      A/P) or placebo followed by NB-001 (treatment sequence P/A). During the Double-Blind
      Treatment Phase of the trial, the subject and/or parent/legal guardian (henceforth,
      'parent/guardian') will be contacted at Day 0 to complete baseline symptom scales and will
      begin dosing with the investigational product (IP; NB-001 or placebo) on the morning of Day
      1. Subjects or their parent/guardian will administer the IP twice daily (BID) and will be
      contacted at Days 0, 1, 14, 28, 42, 49, 50, 63, 77 and 91 to evaluate measures of safety and
      efficacy, including the completion of symptom scales. In addition, the subject and/or
      parent/guardian will be contacted at Days 7, 21, 35, 56, 70 and 84 to assess subject safety.
      Blood samples for pharmacokinetic analysis, 4β-hydroxycholesterol and plasma proline will be
      collected at multiple timepoints. During the Double-Blind Treatment Phase, subjects will
      receive IP corresponding with their first treatment assignment for 6 weeks (Treatment Period
      1), followed by an intervening wash-out period of 1 week, and then will receive their second
      treatment assignment for the subsequent 6-week period (Treatment Period 2). All symptom
      scales will be centrally and/or locally administered. Approximately 10 parents/guardians and
      paired clinical trial site clinicians for subjects who complete the trial per protocol
      through Visit Day 91 will be invited to participate in an optional, one-hour (approximately),
      exit interview to discuss the observations of the subject's experience(s) and functioning
      while participating in the treatment periods of the trial. The subject and/or parent/guardian
      will be contacted for an End of Trial Visit to occur 4 weeks following the last dose of IP to
      assess safety.
    

Intervention name(s): NB-001; Placebo
Intervention type(s): Drug; Other
Intervention description(s): NB-001 is a non-stimulant activator of multiple metabotropic glutamate receptors (mGluRs).; Matching, inactive placebo
Other intervention name(s): fasoracetam monohydrate; (5R)-5-(pyridine-1-carbonyl)pyrrolidin-2-one monohydrate
Intervention MeSH terms: 5-((2-(6-Amino-9H-purin-9-yl) ethyl) amino)-1-pentanol
Allocation: Randomized
Intervention model: Crossover Assignment
Intervention model description: Subjects will be randomized in a 1:1 ratio to one of two treatment sequences: NB-001 (active drug product) followed by placebo (treatment sequence A/P) or placebo followed by NB-001 (treatment sequence P/A). Subjects will receive IP corresponding with their first treatment assignment for 6 weeks (Treatment Period 1), followed by an intervening wash-out period of 1 week, and then will receive their second treatment assignment for the subsequent 6-week period (Treatment Period 2).
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: 22q11 Deletion Syndrome; 22q11.2 Deletion Syndrome; 22q11DS; Children and Adolescents