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

Supernus Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Huntington Disease

Treatments: Drug: SAGE-718

Study type: Interventional

Funder types: Industry

Identifiers

NCT03787758

718-CLP-102 B

Details and patient eligibility

About

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)

Full description

This posting addresses Part B

Enrollment: 6 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

SAGE-718

Experimental group

Treatment:

Drug: SAGE-718