A Study to Evaluate the Safety, Tolerability, and Pharmac... | NCT05195008

ID	Type	Description	Link
2021-002903-36	EudraCT Number

Status Module

Description Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Part A [Single Ascending Dose (SAD)]: BIIB113 Cohort 1	Experimental	Participants aged 18 to 64 years will receive Dose 1 of BIIB113, orally, once daily (QD), on Day 1 of Part A of the study.	Drug: BIIB113
Part A (SAD): BIIB113 Cohort 2	Experimental	Participants aged 18 to 64 years will receive Dose 2 of BIIB113, orally, QD, on Day 1 of Part A of the study	Drug: BIIB113
Part A (SAD): BIIB113 Cohort 3	Experimental	Participants aged 18 to 64 years will receive Dose 3 of BIIB113, orally, QD, on Day 1 of Part A of the study.	Drug: BIIB113
Part A (SAD): BIIB113 Cohort 4	Experimental	Participants aged 18 to 64 years will receive Dose 4 of BIIB113, orally, QD, on Day 1 of Part A of the study.	Drug: BIIB113
Part A (SAD): BIIB113 Cohort 5	Experimental	Participants aged 18 to 64 years will receive Dose 5 of BIIB113, orally, QD, on Day 1 of Part A of the study	Drug: BIIB113

Interventions

Name	Type	Description	Arm Group Labels	Other Names
BIIB113	Drug	Administered as specified in the treatment arm.	Part A (SAD): BIIB113 Cohort 2 Part A (SAD): BIIB113 Cohort 3 Part A (SAD): BIIB113 Cohort 4

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Parts A, B and C: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death; in the view of the investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or is a medically important event.	Part A: Up to 54 days; Part B: Up to 53 days; Part C: Up to 71 days (SAD); Up to 85 days (MAD)
Parts A, B and C: Number of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Parameters, Vital Signs, and 12-Lead Electrocardiogram (ECG)		Part A: Baseline up to 54 days; Part B: Baseline up to 53 days; Part C: Baseline up to 71 days (SAD), Baseline up to 85 days (MAD)
Parts A, B and C: Number of Participants With Change in Columbia Suicide Severity Rating Scale (C-SSRS) Score	C-SSRS is used to assess the suicidality of participants during the study. The assessment includes "yes" or "no" responses for 5 questions each, related to suicidal ideation (wish to be dead, non-specific active suicidal thoughts, active suicidal ideation with any methods, active suicidal ideation with some intent, active suicidal ideation with specific plan) and suicidal behavior (preparatory acts or behavior, aborted attempt, interrupted attempt, actual attempt, suicide). Numeric ratings are provided for severity of ideation, from 1 to 5, with 5 being the most severe.	Part A: Up to 54 days; Part B: Up to 53 days; Part C: Up to 71 days (SAD); Up to 85 days (MAD)
Part C: Percent O-GlcNAcase-Positron Emission Tomography (OGA PET) Target Engagement/Occupancy (TO) as a Function of Dose and Time

Secondary Outcomes

Measure	Description	Time Frame
Parts A and B: Area Under the Concentration-Time Curve From Time Zero to Time of the Last Measurable Concentration (AUClast) of BIIB113	The parameter will be assessed under fed conditions for Part A Cohort 3.	Part A: Pre-dose and at multiple timepoints post-dose on Days 1 to 4; Part B: Pre-dose and at multiple timepoints post-dose on Days 1 to 15

Eligibility Module

Eligibility Criteria

Key Inclusion Criteria:

Female or infertile/vasectomized males aged 18 to 64 years (Parts A and B), 20 to 64 years (Part C), or 65 to 75 years (Part B Dose 7Cohort 9 only), inclusive, at the time of informed consent
Have a body mass index between 18 and 32 kilograms per square meter (kg/m^2), inclusive, at screening
Weight ≥50 kilograms (kg) at screening
Negative Polymerase Chain Reaction (PCR) test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) within 5 days of Day -1 prior to randomization

Key Exclusion Criteria:

History or positive test result at Screening for Human Immunodeficiency Virus (HIV)
Chronic, recurrent, or serious infection (e.g., pneumonia, septicemia), as determined by the investigator, within 90 days prior to screening or between screening and Day -1
History of severe allergic or anaphylactic reactions, or of any allergic reactions that in the opinion of the investigator are likely to be exacerbated by any component of the study treatment
History of systemic hypersensitivity reaction to BIIB113 or the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study
Has suicidal ideation with some intent to act within 6 months prior to the start of screening, per the investigator's clinical judgment or based on the C-SSRS, corresponding to a response of "Yes" on Item 4 (active suicidal ideation with some intent to act, without specific plan) or Item 5 (active suicidal ideation with specific plan and intent) for suicidal ideation on the C-SSRS, or a history of suicidal behaviour within one year prior to the start of screening.
Any condition affecting study treatment absorption (e.g., gastrectomy)
Previous exposure to an OGA inhibitor
Current enrolment in any other drug, biologic, device, or clinical study, or treatment with an investigational drug or approved therapy for investigational use within 90 days (6 months for biologics) prior to Day -1, or 5 half-lives of the agent, whichever is longer
For Part C only: Previously undergone PET scans for research purposes

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.