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MSD-001

Phase 1

Mental Health Disorders | Small molecule | Other |Design Therapeutics, Inc.|Last Updated: Sep 16, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment47

FDA Designations

No designations recorded

Clinical trial landscape

MSD-001 · 1 trial · 1 indication

Phase 1 1
NCT06702332Single Ascending Dose Study of MSD-001 in Healthy ParticipantsMental Health Disorders
COMPLETED47 Analytics
PHASE1COMPLETED
Single Ascending Dose Study of MSD-001 in Healthy Participants
Mental Health DisordersUnlock trial analytics

Study Endpoints

Primary Endpoints

Assess the safety and tolerability of MSD-001 in healthy adult participants by assessing the number, duration, severity, drug relatedness and type of adverse events
up to 30 days

Treatment emergent adverse events/ treatment emergent serious adverse events - their frequency, duration and severity, clinically significant changes in lab parameters, ECG, and vital signs

Secondary Endpoints

Plasma Pharmacokinetics (PK) of a single dose of MSD-001: Maximum Plasma Concentration (Cmax)
Pre-dose and up to 24 hours post dose
Plasma Pharmacokinetics (PK) of a single dose of MSD-001: time to attain Cmax (Tmax)
Pre-dose and up to 24 hours post dose
Plasma Pharmacokinetics (PK) of a single dose of MSD-001: Area under plasma Concentration (AUC)
Pre-dose and up to 24 hours post dose
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Active Treatment: MSD-001ACTIVE_COMPARATORPlanned doses of MSD-001; N = 42
Placebo ComparatorPLACEBO_COMPARATORNon-active study drug N = 10

Interventions

NameTypeDescription
MSD-001DRUGMSD-001 is being developed as part of future two-agent combination treatment approaches for the management of mental health indications
Placebo ComparatorDRUGMatching Placebo
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Eligibility Criteria

Age Range18 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: * Male or female healthy consented participants 18 to 55 years of age * CYP2D6 phenotype established based on pharmacogenetic testing. * Free from psychoactive drug use from 4 weeks before dosing and until the last follow up visit. Key Exclusion Criteria: * Any current cli...

Countries:Netherlands
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Competitive Landscape -Mental Health Disorders 10 trials

No competing companies are currently tracked in active development for Mental Health Disorders.
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Frequently asked questions about MSD-001

What is MSD-001 used for?

MSD-001 is an investigational small molecule being developed for the treatment of mental health disorders. It is currently in Phase 1 clinical development and has been studied in healthy volunteers to evaluate its safety and tolerability.

Who makes MSD-001?

MSD-001 is being developed by Design Therapeutics, Inc., a biopharmaceutical company traded on the stock exchange under the ticker DSGN. The company is advancing this small molecule through early-stage clinical trials for mental health disorders.

What phase is MSD-001 in?

MSD-001 is in Phase 1 clinical development. A Phase 1 trial has been completed, and the drug is not yet approved by regulatory authorities. It remains an investigational agent under study for mental health disorders.

What clinical trials is MSD-001 in?

MSD-001 has one completed Phase 1 clinical trial, identified as NCT06702332, titled 'Single Ascending Dose Study of MSD-001 in Healthy Participants.' This randomized, double-blind, placebo-controlled study enrolled 47 healthy adults in the Netherlands.

Is MSD-001 FDA approved?

MSD-001 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for mental health disorders. The completed Phase 1 trial assessed safety and tolerability in healthy volunteers, but the drug has not yet received regulatory approval.