Clinical trial NCT03787758: A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B
Official title: A Phase 1, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Determine the Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Healthy Adults With an Open-label Cohort of Patients With Huntington's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Huntington Disease
Lead sponsor: Sage Therapeutics
Sponsor class: INDUSTRY
First posted: 2018-12-26
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is a phase 1, double-blind, placebo-controlled, multiple ascending dose study to determine the safety, tolerability, and pharmacokinetics of SAGE-718 oral solution in healthy adults (Part A) with an open-label cohort of patients with Huntington's disease (Part B)

Detailed description: This posting addresses Part B

Eligibility criteria: Inclusion Criteria:

1. Subject is positive for mutant HTT (documented CAG repeats ≥ 40 units).
2. Subject has a body weight ≥50 kg and body mass index ≥18.0 and ≤30.0 kg/m2 at screening.

Exclusion Criteria:

1. Subject has any clinically significant abnormal finding on the physical exam at screening or admission.
2. Subject has a history or presence of a neurologic disease or condition (other than Huntington's disease), including but not limited to severe chorea, epilepsy, closed head trauma with clinically significant sequelae, or a prior seizure.
3. Subject has a family history of epilepsy.
4. Subject has a positive screening test for alcohol or drugs of abuse (including marijuana) at screening or admission.

Primary outcomes:
measure: Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.
timeFrame: 21 Days
measure: Percentage of participants with change from baseline in vital signs.
timeFrame: 21 Days
measure: Change from baseline in electrocardiograms (ECGs) including PR interval, QT interval, QTc interval, QTcF, and rhythm abnormalities
timeFrame: 21 Days
measure: Percentage of participants with change from baseline in clinical laboratory parameters.
timeFrame: 21 Days
measure: Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS).
timeFrame: 21 Days
Secondary outcomes:
measure: PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by area under the curve [AUC].
timeFrame: 17 Days
measure: PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by maximum observed concentration [Cmax].
timeFrame: 17 Days
measure: PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by time of occurrence of Cmax [tmax].
timeFrame: 17 Days
Other outcomes:
Locations:
city: Long Beach
country: United States
facility: Sage Investigational Site
state: California
city: Marlton
country: United States
facility: Sage Investigational Site
state: New Jersey
Study officials:
References: