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Leucettinib-21 First-in-Human Phase 1 in Healthy Volunteers and Subjects With Down Syndrome and Alzheimer's Disease

Phase 1
Recruiting
Conditions
Healthy Volunteers
Down Syndrome
Alzheimer's Disease
Interventions
Registration Number
NCT06206824
Lead Sponsor
Perha Pharmaceuticals
Brief Summary

Leucettinib-21 First-in-Human Phase 1 Study in 4 Parts: Single (Part 1) and Multiple (Part 3) Ascending Doses, and Food-Effect (Part 2) in Healthy Subjects, and Single Dose (Part 4) in People with Down Syndrome (DS) and Alzheimer's Disease (AD).

For Parts 1, 3 and 4, safety and tolerability of an oral administration of Leucettinib-21 will be assessed as primary objectives. Pharmacokinetics and pharmacodynamic biomarkers will be investigated as secondary objectives.

For Part 2, the effect of high fat meal will be evaluated on the pharmacokinetics parameters after an oral administration of Leucettinib-21.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
Male
Target Recruitment
120
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Part 2 : Food-Effect in Healthy VolunteersLeucettinib-21A total of 12 healthy male volunteers will be randomly assigned to one of two sequences in this cross over study part. All subjects will receive the same one dose of Leucettinib-21 in each sequence.
Part 1 : Single Ascending Doses in Healthy VolunteersLeucettinib-21A total of 48 healthy male volunteers will be randomized into six consecutive single ascending dose cohorts of 8 subjects, 6 receiving one dose of Leucettinib-21 and 2 receiving placebo.
Part 3 : Multiple Ascending Doses over 14 days in Healthy VolunteersLeucettinib-21A total of 36 healthy male volunteers will be randomized into three consecutive multiple ascending doses cohorts of 12 subjects, 9 receiving one dose of Leucettinib-21 and 3 receiving placebo everyday for 14 days.
Part 4 : Single Dose in People with Down Syndrome and Alzheimer's DiseaseLeucettinib-21A total of 12 people with Down Syndrome and 12 people with Alzheimer's Disease will receive the same one dose of Leucettinib-21.
Primary Outcome Measures
NameTimeMethod
Part 1 : Safety and tolerability of Leucettinib-21 after single administration at 6 increasing doses in healthy male subjects.Part 1 : Up to 8 days following administration

Assessment of systemic tolerability and safety:

Part 2 : Effect of food on the PK parameters after an oral administration of Leucettinib-21 in healthy male subjects in high fat breakfast condition vs. under fasted conditions.Part 2 : Up to 8 days following administration

Plasma PK assessments

Part 3 : Safety and tolerability of Leucettinib-21 after multiple administration at 3 increasing doses in healthy male subjectsPart 3 : Up to 21 days following administration

Assessment of systemic tolerability and safety

Part 4 : Safety and tolerability of Leucettinib-21 after single administration at 1 dose in Down Syndrome individuals and patients with Alzheimer's diseasePart 4 : Up to 8 days following administration

Assessment of systemic tolerability and safety

Secondary Outcome Measures
NameTimeMethod
PK of Leucettinib-21Up to 24 hours following Leucettinib-21 administration

Assessment of the plasmatic PK of Leucettinib-21

PD of Leucettinib-21Up to 4 hours following Leucettinib-21 administration

Change in the proteome following Leucettinib-21 administration assessed by phosphoproteomics

Activity of DYRK1AUp to 8 hours following Leucettinib-21 administration

Change in DYRK1A activity following Leucettinib-21 administration assessed in Peripheral Blood Mononuclear Cells

Trial Locations

Locations (1)

Eurofins Optimed

🇫🇷

Gières, France

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