Trial Outcomes & Findings for Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 (NCT NCT03879655)

Last Updated: 2023-12-26

Results Overview

An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

2 participants

Primary outcome timeframe

Baseline up to Week 156

Results posted on

2023-12-26

Participant Flow

This study included participants who completed Study VTS301 (Parts A/B \[NCT02534844\] and Part C \[NCT04958642\]) and were judged to receive potential benefit from continued treatment with adrabetadex. The study has been terminated early by the Sponsor due to previous Sponsor decision.

Participant milestones

Participant milestones
Measure	Adrabetadex Participants received treatment with adrabetadex at the last dose level administered in Study VTS301, administered intrathecal (IT) via lumbar puncture (LP) infusion every 2 weeks, for up to a total duration of 3 years or until the investigator considered adrabetadex to be no longer beneficial to the participant, adrabetadex received marketing authorization, or the adrabetadex development program was discontinued.
Overall Study STARTED	2
Overall Study Received at Least 1 Dose of Study Drug	2
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Adrabetadex n=2 Participants Participants received treatment with adrabetadex at the last dose level administered in Study VTS301, administered IT via LP infusion every 2 weeks, for up to a total duration of 3 years or until the investigator considered adrabetadex to be no longer beneficial to the participant, adrabetadex received marketing authorization, or the adrabetadex development program was discontinued.
Age, Categorical <=18 years	2 Participants n=2 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=2 Participants
Age, Categorical >=65 years	0 Participants n=2 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 156

Population: All enrolled participants

Outcome measures

Outcome measures
Measure	Adrabetadex n=2 Participants Participants received treatment with adrabetadex at the last dose level administered in Study VTS301, administered IT via LP infusion every 2 weeks, for up to a total duration of 3 years or until the investigator considered adrabetadex to be no longer beneficial to the participant, adrabetadex received marketing authorization, or the adrabetadex development program was discontinued.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) All TEAEs	2 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) SAEs	0 Participants

Adverse Events

Adrabetadex

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Adrabetadex n=2 participants at risk Participants received treatment with adrabetadex at the last dose level administered in Study VTS301, administered IT via LP infusion every 2 weeks, for up to a total duration of 3 years or until the investigator considered adrabetadex to be no longer beneficial to the participant, adrabetadex received marketing authorization, or the adrabetadex development program was discontinued.
Injury, poisoning and procedural complications Joint injury	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Infections and infestations Influenza	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Ear and labyrinth disorders Hypoacusis	100.0% 2/2 • Baseline up to Week 156 All enrolled participants
General disorders Asthenia	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Nervous system disorders Gelastic seizure	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Infections and infestations Respiratory tract infection viral	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Gastrointestinal disorders Dysphagia	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Gastrointestinal disorders Anal incontinence	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Nervous system disorders Ataxia	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Nervous system disorders Disturbance in attention	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Nervous system disorders Hypokinesia	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Nervous system disorders Language disorder	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Psychiatric disorders Anhedonia	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Psychiatric disorders Staring	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
General disorders Fatigue	50.0% 1/2 • Baseline up to Week 156 All enrolled participants
Nervous system disorders Seizure	50.0% 1/2 • Baseline up to Week 156 All enrolled participants

Additional Information

Executive Vice President, Regulatory Affairs

Mandos, LLC

Phone: 619-905-0489

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place