Trials Sponsors Conditions Drugs Pricing

Conditions

22q11 Deletion Syndrome

Keywords

22q11 Deletion Syndrome22q11.2 Deletion Syndrome22q11DSChildren and Adolescents

Outcome Measures

Primary Outcomes (1)

Safety and Tolerability of NB-001
Type, frequency, severity, and causality of treatment-emergent adverse events (TEAEs),treatment-emergent serious adverse events (TESAEs), clinically significant changes from baseline in laboratory tests, electrocardiograms (ECGs), vital signs, and physical examination findings during treatment with NB-001.
6 weeks (Day 42/ET)

Secondary Outcomes (6)

Treatment Effect of NB-001 on the Clinical Global Impression Improvement (CGI-I) Scale
6 weeks (Day 42/ET)
Treatment Effect of NB-001 on the Clinical Global Impression Severity (CGI-S) Scale
6 weeks (Day 42/ET)
Treatment Effect of NB-001 on the Pediatric Anxiety Rating Scale (PARS)
6 weeks (Day 42/ET)
Treatment Effect of NB-001 on the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5) - Inattention Score
6 weeks (Day 42/ET)
Treatment Effect of NB-001 on the Attention Deficit Hyperactivity Disorder-Rating Scale (ADHD-RS-5) - Hyperactivity Score
6 weeks (Day 42/ET)
+1 more secondary outcomes

Other Outcomes (1)

Exit Interview
End of Treatment; approximately 13 weeks of trial participation

Study Arms (2)

NB-001

EXPERIMENTAL

Two (2) 100 mg capsules administered orally BID (400 mg total daily dose) with liquids or, if the subject is unable to swallow a capsule whole, capsules were opened, and the contents sprinkled on applesauce.

Drug: NB-001

Placebo

PLACEBO COMPARATOR

Two (2) capsules (matching, inactive) administered orally BID with liquids or, if the subject is unable to swallow a capsule whole, capsules were opened, and the contents sprinkled on applesauce.

Other: Placebo

Interventions

NB-001Drug

Non-stimulant modulator of metabotropic glutamate receptors (mGluRs)

Also known as: fasoracetam monohydrate, (5R)-5-(piperidine-1-carbonyl)pyrrolidin-2-one monohydrate

NB-001

PlaceboOther

Matching, inactive placebo

Placebo

Eligibility Criteria

Age6 Years - 17 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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.

Sponsors & Collaborators

Nobias Therapeutics, Inc.
Industrylead

Study Sites (4)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States

Seattle Children's Hospital

Seattle, Washington, 98115, United States

The Hospital for Sick Children (SickKids)

Toronto, Ontario, M5G1X8, Canada

MeSH Terms

Conditions

22q11 Deletion SyndromeDiGeorge Syndrome

Interventions

5-((2-(6-Amino-9H-purin-9-yl) ethyl) amino)-1-pentanol

Condition Hierarchy (Ancestors)

Craniofacial AbnormalitiesMusculoskeletal AbnormalitiesMusculoskeletal DiseasesHeart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesLymphatic AbnormalitiesLymphatic DiseasesHemic and Lymphatic Diseases

Results Point of Contact

Title: Patrick Dougherty
Organization: Nobias Therapeutics, Inc.

Publication Agreements

PI is Sponsor Employee: No
Restrictive Agreement: No

Enrollment

37 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 2
Allocation: RANDOMIZED
Masking: QUADRUPLE
Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details: NB-001 and matching placebo were 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.
Purpose: TREATMENT
Intervention Model: CROSSOVER
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

January 26, 2022

You may not qualify if:

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.

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

Study Start

First Posted

Primary Completion

Study Completion

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (6)

Other Outcomes (1)

Study Arms (2)

NB-001

Placebo

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (4)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Results Point of Contact

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

Study Start

First Posted

Primary Completion

Study Completion

Results Posted

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (6)

Other Outcomes (1)

Study Arms (2)

NB-001

Placebo

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (4)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Results Point of Contact

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations