Overview
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
Status:
Completed
Completed
Trial end date:
2018-12-27
2018-12-27
Target enrollment:
0
0
Participant gender:
All
All
Summary
The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity. This study will also evaluate the efficacy of TAK-831 versus placebo on activities of daily living (ADL) and other secondary assessments.Phase:
Phase 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Neurocrine Biosciences
TakedaCollaborator:
Takeda
Criteria
Key Inclusion Criteria:1. Has a genetically-confirmed diagnosis (homozygous for guanine-adenine-adenine [GAA]
repeat expansions in the frataxin gene [FXN] in the affected range of Friedreich ataxia
[FRDA] or a compound heterozygous expansion with a point mutation or deletion), with an
established disease stage of 2 to 5, inclusive, as determined by the Functional Staging for
Ataxia, at Screening.
Key Exclusion Criteria:
1. Received a diagnosis of ataxic syndromes other than FRDA.
2. Has a history of cancer, except basal cell carcinoma or in situ cervical cancer that
has been in remission for greater than or equal to (>=5) years prior to first dose of
study drug.
3. Known to be currently infected or has been infected with human immunodeficiency virus
(HIV), hepatitis B virus, or hepatitis C virus.
4. Has a known hypersensitivity to any component of the formulation of TAK-831.
5. Has a history of drug abuse (defined as any illicit drug use) or a history of alcohol
abuse.
6. Has taken any excluded medication, or has had insufficient washout of medications or
is unable or unwilling to discontinue medications as required by the protocol.
7. If male, the participant intends to donate sperm during the course of this study or
for 95 days after the last dose of study drug.
8. If female, the participant is of childbearing potential and lactating, pregnant
(positive prerandomization serum pregnancy test), or plans to become pregnant before
participating in the study, during the study, or within 35 days after last dose of the
study drug, or intending to donate ova during such time period.
9. Has a history of neuroleptic malignant syndrome, water intoxication, or paralytic
ileus or other conditions that may interfere with absorption of study medication.