A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B

Phase 1

Completed

• Conditions: Huntington Disease
• Interventions: Drug: SAGE-718

• Registration Number: NCT03787758
• Lead Sponsor: Sage Therapeutics

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

Study Timeline

• Study First Submitted: 2018-12-17
• Study First Submitted QC: 2018-12-21
• Study First Posted: 2018-12-26
• Study Start: 2019-02-28
• Primary Completion: 2019-10-07
• Study Completion: 2019-10-07
• Status Verified: 2022-01
• Last Update Submitted: 2022-01-20
• Last Update Posted: 2022-01-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
SAGE-718	SAGE-718	-

Primary Outcome Measures

Name	Time	Method
Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.	21 Days
Percentage of participants with change from baseline in vital signs.	21 Days
Percentage of participants with change from baseline in clinical laboratory parameters.	21 Days
Change from baseline in electrocardiograms (ECGs) including PR interval, QT interval, QTc interval, QTcF, and rhythm abnormalities	21 Days
Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS).	21 Days

Secondary Outcome Measures

Name	Time	Method
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by area under the curve [AUC].	17 Days
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by maximum observed concentration [Cmax].	17 Days
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by time of occurrence of Cmax [tmax].	17 Days

Trial Locations

Locations (1)

Sage Investigational Site; 🇺🇸 Marlton, New Jersey, United States