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NB-001 in Children and Adolescents With 22q11 Deletion Syndrome

Primary Purpose

22q11 Deletion Syndrome

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
NB-001
Placebo
Sponsored by
Nobias Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for 22q11 Deletion Syndrome focused on measuring 22q11 Deletion Syndrome, 22q11.2 Deletion Syndrome, 22q11DS, Children and Adolescents

Eligibility Criteria

6 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. The subject has a genotype with a pathologic deletion in the 22q11 region confirmed by documentation (e.g., genetic test results) available at the clinical trial site.
  2. The subject is aged 6 to 17 years old, inclusive.
  3. The subject has a CGI-S scale score of ≥4 (i.e., moderately, markedly, severely, or among the most extremely ill patients) at Screening. Note that the Severity score of 4 could be from a composite of 2 or more sub-threshold scores.

    And either:

    1. Psychiatric symptoms in the clinical range for at least 1 of 3 disorders, anxiety disorder, ADHD, or ASD, respectively, as demonstrated by score(s) at or above the following numbers on at least 1 of 3 scales:

      • PARS 5-Item Severity Score ≥12 (i.e., sum of items 2+3+5+6+7 ≥12)
      • ADHD-RS-5 Scores of 2 or 3 (i.e., "Often" or "Very Often") on at least 6 questions, with the majority of symptoms related to inattention (common in 22q11DS) rather than hyperactivity (less common in 22q11DS)
      • SRS-2 >60

      OR:

    2. Psychiatric symptoms in the subclinical range for at least 2 of 3 disorders, anxiety disorder, ADHD, and/or ASD, respectively, as demonstrated by scores at or above the following numbers on at least 2 of 3 scales:

      • PARS 5-Item Severity Score of 10 or 11 (i.e., sum of items 2+3+5+6+7=10 or 11)
      • ADHD-RS-5 Scores of 2 or 3 (i.e., "Often" or "Very Often") on 4 or 5 questions, with the majority of symptoms related to inattention (common in 22q11DS) rather than hyperactivity (less common in 22q11DS)
      • SRS-2 of 55-59
  4. The subject has adequate renal and hepatic function indicated by:

    • Estimated glomerular filtration rate (eGFR) ≥60 mL/min/1.73 m2 (per the revised Schwartz equation; Fadrowski and Furth 2011, Staples et al. 2010)
    • Serum bilirubin ≤2.5 × upper limit of normal (ULN; unless documented Gilbert's Disease); aspartate aminotransferase and alanine aminotransferase ≤2.5 × ULN
  5. If the subject is female and of reproductive potential, she has a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day 0.
  6. If the subject is of reproductive potential, s/he agrees to abstain from reproductive cell donation, per below, and, if ever heterosexually active, to use dual effective/highly effective contraception (including at least one effective and at least one highly effective contraceptive method; Section 9.2.1) from Screening through the End of Trial Visit.

    • If the subject is female and of reproductive potential, she agrees to abstain from oocyte donation from Screening through the End of Trial Visit.
    • If the subject is male and of reproductive potential, he agrees to abstain from sperm donation from Screening through the End of Trial Visit.
  7. The subject's parent/guardian understands the trial procedures and agrees to the subject's participation in the trial, as well as to the parent/guardian trial involvement, as indicated by parent/guardian signature on the informed consent form and, if applicable, subject signature on the subject assent form.

Exclusion Criteria:

  1. The subject or parent/guardian is, in the opinion of the Investigator, mentally or legally incapacitated, or has significant emotional problems at the time of Screening or expected emotional problems during the conduct of the trial which would interfere with the conduct of the trial evaluations.
  2. The subject has a history of psychotic symptoms, current psychotic symptoms, or a diagnosis of a psychotic disorder based on clinical assessment.
  3. The subject has a history of any illness that, in the opinion of the Investigator, might confound the results of the trial or pose an additional risk to the subject by participation in the trial.
  4. The subject has clinically significant unstable or uncontrolled endocrine, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases.
  5. The subject has uncontrolled, active seizure(s), within the 3 months prior to Screening.
  6. The subject has known human immunodeficiency virus (HIV), a detectable viral load for hepatitis C, or hepatitis B surface antigen indicative of chronic active infection.
  7. The subject is pregnant or is a nursing mother.
  8. The subject has suicidal ideation and behavior, based on Investigator assessment of the completed Columbia-Suicide Severity Rating Scale at Screening, or when repeated on Day 0 (if more than 21 days elapse between Screening and Day 1).
  9. The subject is currently taking medication(s) at a dose that has not been stable for ≥3 months prior to Day 1 or psychotherapy that has not been stable for ≥3 months prior to Day 1. If the subject is taking medication(s) or receiving psychotherapy, the subject and parent/guardian must agree to continue the intervention(s) at the same dose and frequency through the End of Trial Visit.
  10. The subject has received any investigational therapy (i.e., used for a non-approved indication and in the context of a research investigation) <14 days prior to the first dose of NB-001 (i.e., Day 1) or within 5 drug half-lives prior to the first dose of NB-001.
  11. The subject uses illicit drugs (e.g., marijuana, amphetamines or cocaine), or has known alcohol or drug abuse or dependence, as defined by the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (American Psychiatric Association 2013). Medically approved marijuana use, where usage is legal, is allowed; however, the dose and frequency of use should remain stable during trial participation.

Sites / Locations

  • Children's Hospital Colorado
  • Children's Hospital of Philadelphia (CHOP)
  • Seattle Children's Hospital
  • The Hospital for Sick Children (SickKids)

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

NB-001

Placebo

Arm Description

Active drug product, NB-001: Two (2) 100 mg capsules will be administered orally BID with liquids or, if the subject is unable to swallow a capsule whole, capsules may be opened, and the contents sprinkled on applesauce; total daily dose: 400 mg.

Placebo: Two (2) capsules (matching NB-001) will be administered orally BID with liquids or, if the subject is unable to swallow a capsule whole, capsules may be opened, and the contents sprinkled on applesauce.

Outcomes

Primary Outcome Measures

Number of participants with treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (TESAEs) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) Dictionary v.5.0
Clinical and laboratory adverse events (AEs) will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). System Organ Class (SOC) and Preferred Term (PT) will be attached to the clinical database. AE severity will be graded using the CTCAE. Summaries (number and percentage of subjects) of TEAEs and TESAEs by SOC and PT will be provided by treatment group. TEAEs will also be summarized by relationship to study drugs and severity. In addition, TEAEs leading to premature discontinuation of study drugs and study, and SAEs leading to death will be summarized and listed.

Secondary Outcome Measures

Change from baseline in Clinical Global Impression Improvement (CGI-I) Scale
Responder analyses will be performed for the CGI-I by classifying each subject as a responder or non-responder using an endpoint-specific threshold and computing the response rate in each treatment group. The CGI-S is a 7-point scale where 1=Very much improved and 7=Very much worse.
Change from baseline in Clinical Global Impression Severity (CGI-S) Scale
Responder analyses will be performed for the CGI-S by classifying each subject as a responder or non-responder using an endpoint-specific threshold and computing the response rate in each treatment group. The CGI-S is a 7-point scale where 1=Normal, Not at all impaired and 7=Among the most extremely impaired patients.
Treatment effect of NB-001 on the Pediatric Anxiety Rating Scale (PARS)
Treatment effect will be evaluated within the subset of subjects where the symptom of greatest impairment at baseline is anxiety as defined by the Pediatric Anxiety Rating Scale (PARS). A score of >12 on the PARS 5-item total severity score (items 2+3+5+6+7) is indicative of psychiatric symptoms in the clinical range for anxiety disorder, and a score of 10 or 11 is indicative of psychiatric symptoms in the subclinical range for anxiety disorder.
Treatment effect of NB-001 on the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5)
Treatment effect will be evaluated within the subset of subjects where the symptom of greatest impairment at baseline is attention deficit as assessed by the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5). A score of >6 on the ADHD-RS-5 is indicative of psychiatric symptoms in the clinical range for attention deficit disorder, and a score of 4 or 5 is indicative of psychiatric symptoms in the subclinical range for attention deficit disorder.
Treatment effect of NB-001 on the Social Responsiveness Scale, Second Edition (SRS-2)
Treatment effect will be evaluated within the subset of subjects where the symptom of greatest impairment at baseline is one or more symptoms in autism spectrum disorder as assessed by the Social Responsiveness Scale, Second Edition (SRS-2). A score of >60 on the SRSTM-2 is indicative of psychiatric symptoms in the clinical range for autism spectrum disorder, and a score of 55-59 is indicative of psychiatric symptoms in the subclinical range for autism spectrum disorder.

Full Information

First Posted
January 26, 2022
Last Updated
October 5, 2023
Sponsor
Nobias Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05290493
Brief Title
NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
Official Title
A Randomized, Placebo-Controlled Crossover Trial to Assess the Safety and Efficacy of NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Completed
Study Start Date
February 10, 2022 (Actual)
Primary Completion Date
June 1, 2023 (Actual)
Study Completion Date
June 9, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Nobias Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
22q11 Deletion Syndrome
Keywords
22q11 Deletion Syndrome, 22q11.2 Deletion Syndrome, 22q11DS, Children and Adolescents

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
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
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
NB-001 and matching placebo will be supplied to the Investigator or designee in blinded plastic bottles, each containing 40 capsules. Additionally, subject and parent/guardian, the Investigator, clinical trial site personnel, home health nurses, centralized rater(s), and the Sponsor will be blinded to treatment sequence assignment.
Allocation
Randomized
Enrollment
37 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NB-001
Arm Type
Active Comparator
Arm Description
Active drug product, NB-001: Two (2) 100 mg capsules will be administered orally BID with liquids or, if the subject is unable to swallow a capsule whole, capsules may be opened, and the contents sprinkled on applesauce; total daily dose: 400 mg.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo: Two (2) capsules (matching NB-001) will be administered orally BID with liquids or, if the subject is unable to swallow a capsule whole, capsules may be opened, and the contents sprinkled on applesauce.
Intervention Type
Drug
Intervention Name(s)
NB-001
Other Intervention Name(s)
fasoracetam monohydrate, (5R)-5-(pyridine-1-carbonyl)pyrrolidin-2-one monohydrate
Intervention Description
NB-001 is a non-stimulant activator of multiple metabotropic glutamate receptors (mGluRs).
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Matching, inactive placebo
Primary Outcome Measure Information:
Title
Number of participants with treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (TESAEs) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) Dictionary v.5.0
Description
Clinical and laboratory adverse events (AEs) will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). System Organ Class (SOC) and Preferred Term (PT) will be attached to the clinical database. AE severity will be graded using the CTCAE. Summaries (number and percentage of subjects) of TEAEs and TESAEs by SOC and PT will be provided by treatment group. TEAEs will also be summarized by relationship to study drugs and severity. In addition, TEAEs leading to premature discontinuation of study drugs and study, and SAEs leading to death will be summarized and listed.
Time Frame
6 weeks
Secondary Outcome Measure Information:
Title
Change from baseline in Clinical Global Impression Improvement (CGI-I) Scale
Description
Responder analyses will be performed for the CGI-I by classifying each subject as a responder or non-responder using an endpoint-specific threshold and computing the response rate in each treatment group. The CGI-S is a 7-point scale where 1=Very much improved and 7=Very much worse.
Time Frame
6 weeks
Title
Change from baseline in Clinical Global Impression Severity (CGI-S) Scale
Description
Responder analyses will be performed for the CGI-S by classifying each subject as a responder or non-responder using an endpoint-specific threshold and computing the response rate in each treatment group. The CGI-S is a 7-point scale where 1=Normal, Not at all impaired and 7=Among the most extremely impaired patients.
Time Frame
6 weeks
Title
Treatment effect of NB-001 on the Pediatric Anxiety Rating Scale (PARS)
Description
Treatment effect will be evaluated within the subset of subjects where the symptom of greatest impairment at baseline is anxiety as defined by the Pediatric Anxiety Rating Scale (PARS). A score of >12 on the PARS 5-item total severity score (items 2+3+5+6+7) is indicative of psychiatric symptoms in the clinical range for anxiety disorder, and a score of 10 or 11 is indicative of psychiatric symptoms in the subclinical range for anxiety disorder.
Time Frame
6 weeks
Title
Treatment effect of NB-001 on the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5)
Description
Treatment effect will be evaluated within the subset of subjects where the symptom of greatest impairment at baseline is attention deficit as assessed by the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5). A score of >6 on the ADHD-RS-5 is indicative of psychiatric symptoms in the clinical range for attention deficit disorder, and a score of 4 or 5 is indicative of psychiatric symptoms in the subclinical range for attention deficit disorder.
Time Frame
6 weeks
Title
Treatment effect of NB-001 on the Social Responsiveness Scale, Second Edition (SRS-2)
Description
Treatment effect will be evaluated within the subset of subjects where the symptom of greatest impairment at baseline is one or more symptoms in autism spectrum disorder as assessed by the Social Responsiveness Scale, Second Edition (SRS-2). A score of >60 on the SRSTM-2 is indicative of psychiatric symptoms in the clinical range for autism spectrum disorder, and a score of 55-59 is indicative of psychiatric symptoms in the subclinical range for autism spectrum disorder.
Time Frame
6 weeks
Other Pre-specified Outcome Measures:
Title
Subject experience and meaningfulness of the change(s) while participating in NB-001-01
Description
Optional qualitative exit interviews will be conducted with clinicians and parent/guardian(s) of approximately 10 subjects who complete NB-001-01 per protocol. Data collected will be coded using qualitative analysis software (e.g., MAXQDA) to faciliate the identification of concepts that are most important and relevant to subjects. Outputs will be generated in tabular, graphical, or text formats in MS Word or MS PowerPoint to summarize results.
Time Frame
End of Treatment; approximately 13 weeks of trial participation

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: The subject has a genotype with a pathologic deletion in the 22q11 region confirmed by documentation (e.g., genetic test results) available at the clinical trial site. The subject is aged 6 to 17 years old, inclusive. The subject has a CGI-S scale score of ≥4 (i.e., moderately, markedly, severely, or among the most extremely ill patients) at Screening. Note that the Severity score of 4 could be from a composite of 2 or more sub-threshold scores. And either: Psychiatric symptoms in the clinical range for at least 1 of 3 disorders, anxiety disorder, ADHD, or ASD, respectively, as demonstrated by score(s) at or above the following numbers on at least 1 of 3 scales: PARS 5-Item Severity Score ≥12 (i.e., sum of items 2+3+5+6+7 ≥12) ADHD-RS-5 Scores of 2 or 3 (i.e., "Often" or "Very Often") on at least 6 questions, with the majority of symptoms related to inattention (common in 22q11DS) rather than hyperactivity (less common in 22q11DS) SRS-2 >60 OR: Psychiatric symptoms in the subclinical range for at least 2 of 3 disorders, anxiety disorder, ADHD, and/or ASD, respectively, as demonstrated by scores at or above the following numbers on at least 2 of 3 scales: PARS 5-Item Severity Score of 10 or 11 (i.e., sum of items 2+3+5+6+7=10 or 11) ADHD-RS-5 Scores of 2 or 3 (i.e., "Often" or "Very Often") on 4 or 5 questions, with the majority of symptoms related to inattention (common in 22q11DS) rather than hyperactivity (less common in 22q11DS) SRS-2 of 55-59 The subject has adequate renal and hepatic function indicated by: Estimated glomerular filtration rate (eGFR) ≥60 mL/min/1.73 m2 (per the revised Schwartz equation; Fadrowski and Furth 2011, Staples et al. 2010) Serum bilirubin ≤2.5 × upper limit of normal (ULN; unless documented Gilbert's Disease); aspartate aminotransferase and alanine aminotransferase ≤2.5 × ULN If the subject is female and of reproductive potential, she has a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day 0. If the subject is of reproductive potential, s/he agrees to abstain from reproductive cell donation, per below, and, if ever heterosexually active, to use dual effective/highly effective contraception (including at least one effective and at least one highly effective contraceptive method; Section 9.2.1) from Screening through the End of Trial Visit. If the subject is female and of reproductive potential, she agrees to abstain from oocyte donation from Screening through the End of Trial Visit. If the subject is male and of reproductive potential, he agrees to abstain from sperm donation from Screening through the End of Trial Visit. The subject's parent/guardian understands the trial procedures and agrees to the subject's participation in the trial, as well as to the parent/guardian trial involvement, as indicated by parent/guardian signature on the informed consent form and, if applicable, subject signature on the subject assent form. Exclusion Criteria: The subject or parent/guardian is, in the opinion of the Investigator, mentally or legally incapacitated, or has significant emotional problems at the time of Screening or expected emotional problems during the conduct of the trial which would interfere with the conduct of the trial evaluations. The subject has a history of psychotic symptoms, current psychotic symptoms, or a diagnosis of a psychotic disorder based on clinical assessment. The subject has an intelligence quotient (IQ) score of <65 based on the WASI-II assessment. NOTE: A maximum of 3 (i.e., 10% of the total N) nonverbal subjects will be allowed in the trial on a first-come-first-served basis. The subject has a history of any illness that, in the opinion of the Investigator, might confound the results of the trial or pose an additional risk to the subject by participation in the trial. The subject has clinically significant unstable or uncontrolled endocrine, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases. The subject has uncontrolled, active seizure(s), within the 3 months prior to Screening. The subject has known human immunodeficiency virus (HIV), a detectable viral load for hepatitis C, or hepatitis B surface antigen indicative of chronic active infection. The subject is pregnant or is a nursing mother. The subject has suicidal ideation and behavior, based on Investigator assessment of the completed Columbia-Suicide Severity Rating Scale at Screening, or when repeated on Day 0 (if more than 21 days elapse between Screening and Day 1). The subject is currently taking neuropsychiatric medication(s) at a dose that has not been stable for ≥3 months prior to Day 1 or psychotherapy that has not been stable for ≥3 months prior to Day 1. If the subject is taking medication(s) or receiving psychotherapy, the subject and parent/guardian must agree to continue the intervention(s) at the same dose and frequency through the End of Trial Visit. The subject has received any investigational therapy (i.e., used for a non-approved indication and in the context of a research investigation) <14 days prior to the first dose of NB-001 (i.e., Day 1) or within 5 drug half-lives prior to the first dose of NB-001. The subject uses illicit drugs (e.g., marijuana, amphetamines or cocaine), or has known alcohol or drug abuse or dependence, as defined by the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (American Psychiatric Association 2013). Medically approved marijuana use, where usage is legal, is allowed; however, the dose and frequency of use should remain stable during trial participation.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Neil Inala
Organizational Affiliation
Nobias Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Children's Hospital of Philadelphia (CHOP)
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98115
Country
United States
Facility Name
The Hospital for Sick Children (SickKids)
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G1X8
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

NB-001 in Children and Adolescents With 22q11 Deletion Syndrome

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