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A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-148009 in Untreated Parkinson's Disease

Phase 2
Active, not recruiting
Conditions
Parkinson Disease
Interventions
Drug: Placebo
Registration Number
NCT05424276
Lead Sponsor
ABLi Therapeutics, Inc.
Brief Summary

This study investigates the safety and tolerability of drug IkT-148009 in untreated Parkinson's disease volunteers (30 to 80 years old). It also looks at the pharmacokinetics of IkT-148009 in the body and evaluates the effect of IkT-148009 on motor and non-motor features of the disease. This 12 week study is designed to be 3:1 randomized across 3 doses of IkT-148009 or placebo. Each participant will self-administer one of 3 doses or placebo of IkT-148009 once daily (QD) with food for 12 weeks. For more information, visit our website: www.the201trial.com

Detailed Description

This is a 12-Week, randomized, double-blind, multi-center, placebo-controlled dose-ranging clinical trial of three IkT 148009 doses in patients with untreated PD designed to assess safety, tolerability, and pharmacokinetics of IkT-148009, an oral, once daily c-Abl tyrosine kinase inhibitor. Secondary and exploratory assessments will evaluate the effect of IkT-148009 on motor and non-motor features of the disease. 120 participants are anticipated to be enrolled at up to 34 sites across the US.

Participants will undergo screening to evaluate their eligibility to participate in the study to include evaluation of Parkinson's diagnosis, vital signs, blood chemistry, hematology and urinalysis and complete listing of concomitant medications. An Enrollment Authorization Committee (EAC) will be responsible for reviewing screening data and confirming the eligibility and suitability of participants. Those selected will be enrolled and randomized to one of three active IkT-148009 arms (50/100/200 mg) or a placebo arm (1:1:1:1). All clinical staff, study investigators, and participants will be blinded to study assignments throughout the trial.

A Data Safety Monitoring Committee (DSMB) will evaluate all available safety, tolerability, and PK and Parkinson's disease-related data for each cohort on a monthly to quarterly basis. Adverse event reporting will be evaluated in real-time.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
120
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
50mg IkT-148009IkT-148009This arm will consist of thirty (30) patients on 50mg of active treatment.
100mg IkT-148009IkT-148009This arm will consist of thirty (30) patients on 100mg of active treatment.
200mg IkT-148009IkT-148009This arm will consist of thirty (30) patients on 200mg of active treatment.
PlaceboPlaceboThis arm will consist of thirty (30) patients on placebo.
Primary Outcome Measures
NameTimeMethod
Incidence and temporal profile of treatment-emergent adverse events (TEAEs) evaluated by type/nature, severity/intensity, seriousness, and relationship to study interventionDay 1 through week 16
Proportion of those randomized in each dosing cohort who discontinued the assigned regimenDay 1 through week 12
Secondary Outcome Measures
NameTimeMethod
Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II + IIIChange from Baseline to Week 12

Higher scores mean worse outcome from 0 to 52

Patient Global Impression-Severity (PGI-S)Change from Baseline to Week 12

Scale varies from None to Very Severe

Clinician Global Impression of Severity (CGI-S)Change from Baseline to Week 12

Higher scores mean worse outcome between 1 and 7.

Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part IChange from Baseline to Week 12

Higher scores mean worse outcome from 0 to 52.

Non-Motor Symptom Scale (NMSS)Change from Baseline to Week 12

Higher scores mean worse outcome from 0 to 360.

Complete Spontaneous Bowel Movement (CSBM)Change from Baseline to Week 12

A value \< 3 implies constipation

Epworth Sleepiness Scale (ESS)Change from Baseline to Week 12

Higher score means worse outcome from 0 to 24.

Schwab and England Activities of Daily Living (SE-ADL) ScaleChange from Baseline to Week 12

Higher score means worse outcome from 0% to 100%

Parkinson's Disease Questionnaire (PDQ-39)Change from Baseline to Week 12

Higher scores mean worse outcome from 0 to 100.

Patient Assessment of Upper Gastrointestinal Disorders Severity Index (PAGI-SYM)Change from Baseline to Week 12

Higher score means worse outcome from 0 to 100.

Patient Assessment of Constipation Quality of Life (PAC-QOL)Change from Baseline to Week 12

Higher score means worse outcome from 0 to 150.

Patient Assessment of Gastrointestinal Disorders Severity Quality of Life (PAGI- QOL)Change from Baseline to Week 12

Higher score means worse outcome from 0 to 150.

Trial Locations

Locations (2)

Neurology

🇺🇸

Milwaukee, Wisconsin, United States

Neurologist

🇺🇸

Boca Raton, Florida, United States

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