A Phase 2 Study of YA-101 in Patients With Multiple System Atrophy

ConditionMultiple System Atrophy

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age30+

SponsorDasher Neuroscience Inc.

About this trial

This is a Phase 2, double-blind, placebo-controlled, multi-center, Phase II, dose escalation study to evaluate the safety, tolerability, pharmacokinetics and efficacy of Ya-101 in subjects with multiple system atrophy.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Able to understand the process of the clinical trial and give informed consent for the participation of the study.

Diagnosis of MSA according to MDS clinical criteria (Wenning et al, 2022), including subjects with MSA of either subtype (MSA-P or MSA-C).

Males or non-pregnant, non-lactating females with no child-bearing potential, or agree to use 2 forms of contraception.

Able to take oral medications.

Disqualifiers

Positive urine test for drugs of abuse and/or alcohol test both at screening and Day 1.

Evidence of renal impairment or hepatic impairment.

Subject with a Mini-Mental State Examination (MMSE) score of 24 or lower.

Medical history includes severe systemic diseases such as cardiopulmonary failure, severe liver or kidney disease, and uncontrolled diabetes; significant central nervous system disorders like stroke, encephalitis, and epilepsy and severe head trauma; peptic ulcer in one year prior to screening.

Trial design

Design model

Sequential

Treatments tested in this trial

YA-101
Drug
Drug: YA-101 • YA-101 taken BID
Placebo
Drug
Placebo taken BID

Treatment groups

75 Participants

are divided into 2 treatment groups

Group A: YA-101Experimental treatment 1 intervention

Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

Incidence and severity of Adverse Events (AEs)

Incidence and severity of Adverse Events (AEs).

Time frame

Baseline to Day 112

Secondary outcomes

Maximum observed concentration (Cmax)

To describe the pharmacokinetics parameter (Cmax) of YA-101 using sparse PK sampling.

Time frame

Baseline to Day 84

Area under the concentration-time curve (AUC)

To describe the pharmacokinetics parameter (AUC) of YA-101 using sparse PK sampling.

Time frame

Baseline to Day 84

Time of maximum observed concentration (Tmax)

To describe the pharmacokinetics parameter (Tmax) of YA-101 using sparse PK sampling.

Time frame

Baseline to Day 84

Change from baseline in Unified Multiple System Atrophy Rating Scale (UMSARS) over time

The unified multiple system atrophy rating scale (UMSARS) is composed of four subscales: UMSARS-I (12 items) rates patient-reported functional disability, UMSARS-II (14 items) assesses motor impairment based on a clinical examination, UMSARS-III records blood pressure and heart rate in the supine and standing positions, and UMSARS-IV (1 item) rates chore-based disability. Higher scores on the UMSARS indicate greater disability.

Time frame

Baseline to Day 112