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M5049

Phase 1

Healthy | Small molecule | Other |Merck KGaA|Last Updated: Feb 21, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment96

FDA Designations

No designations recorded

Clinical trial landscape

M5049 · 2 trials · 3 indications

Phase 1 2
NCT04647708Study of M5049 in CLE and SLE ParticipantsSystemic Lupus Erythematosus
COMPLETED25 Analytics
NCT03676322Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) Study of M5049 in Healthy ParticipantsHealthy
COMPLETED96 Analytics
PHASE1COMPLETED
Study of M5049 in CLE and SLE Participants
Systemic Lupus ErythematosusUnlock trial analytics
PHASE1COMPLETED
Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) Study of M5049 in Healthy Participants
HealthyUnlock trial analytics

Study Endpoints

Primary Endpoints

Part A: Cohort 1 and 2: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Event of Special Interest (AESI), TEAEs Leading to Permanent Treatment Discontinuation and Treatment-Related TEAEs
Up to Day 102
Part A: Cohort 3 and 4; Part B: Cohort 5: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Event of Special Interest (AESI), TEAEs Leading to Permanent Treatment Discontinuation and Treatment-Related TEAEs
Up to Day 186
Part A: Cohort 1 and 2: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Based on Severity
Up to Day 102
Part A: Cohort 3 and 4; Part B: Cohort 5: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) Based on Severity
Up to Day 186
Part A: Cohort 1 and 2: Number of Participants with Clinically Significant Changes from Baseline in Laboratory Parameters, Vital Signs, Electroencephalogram (EEG) and Electrocardiogram (ECG) Findings
Up to Day 102
Part A: Cohort 3 and 4; Part B: Cohort 5: Number of Participants with Clinically Significant Changes from Baseline in Laboratory Parameters, Vital Signs, Electroencephalogram (EEG) and Electrocardiogram (ECG) Findings
Up to Day 186
Part A: Cohort 1 and 2: Number of Participants with Confirmed Signs and Symptoms of Prodromal Seizure
Up to Day 102
Part A: Cohort 3 and 4; Part B: Cohort 5: Number of Participants with Confirmed Signs and Symptoms of Prodromal Seizure
Up to Day 186
Part A: Cohort 1 and 2: Number of Participants with Suicidal Behavior and Suicidal Ideation as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS)
Up to Day 102
Part A: Occurrence and Severity of Treatment-emergent Adverse Events (TEAEs) and Serious AEs (SAEs)
Day 1 up to Day 21
Part A: Number of Participants With Clinically Significant Changes in Vital Signs, Laboratory Parameters and 12-lead Electrocardiogram (ECG) Findings
Day 1 up to Day 21

Number of participants with clinically significant changes will be reported.

Part B: Occurrence and Severity of TEAEs and SAEs
Day 1 up to Day 33
Part B: Number of Participants With Clinically Significant Changes in Vital Signs, Laboratory Parameters and 12-lead Electrocardiogram (ECG) Findings
Day 1 up to Day 33

Number of participants with clinically significant changes will be reported.

Part C: Maximum Observed Plasma Concentration (Cmax) of M5049
Pre-dose up to Day 6
Part C: Time to Reach Maximum Plasma Concentration (tmax) of M5049
Pre-dose up to Day 6
Part C: Elimination Rate Constant (λz) of M5049
Pre-dose up to Day 6
Part C: Apparent Terminal Half-life (t1/2) of M5049
Pre-dose up to Day 6
Part C: Area Under the Plasma Concentration-Time Curve From Time Zero to the Last Sampling Time (AUC 0-t) of M5049
Pre-dose up to Day 6
Part C: Area Under the Concentration-Time Curve Over Entire Dosing Time Period From Time Zero Extrapolated to Infinity (AUC0-inf)
Pre-dose up to Day 6
Part C: Total Body Clearance (CL/f) of M5049
Pre-dose up to Day 6
Part C: Apparent Volume of Distribution (Vz/f) of M5049
Pre-dose up to Day 6

Secondary Endpoints

Part A and Part B: Maximum Observed Plasma Concentration (Cmax) of M5049
Pre-dose up to Day 85 for Cohort 1 and 2 of Part A, up to Day 169 for Cohort 3, 4 of Part A and Cohort 5 of Part B
Part A and Part B: Dose Normalized Maximum Observed Plasma Concentration (Cmax/Dose) of M5049
Day 1 and Day 29
Part A and Part B: Time to Reach Maximum Plasma Concentration (tmax) of M5049
Pre-dose up to Day 85 for Cohort 1 and 2 of Part A, up to Day 169 for Cohort 3, 4 of Part A and Cohort 5 of Part B
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part A (Cohort 1): M5049 Dose AEXPERIMENTAL -
Part A (Cohort 2): M5049 Dose BEXPERIMENTAL -
Part A (Cohort 3): M5049 Dose CEXPERIMENTAL -
Part A (Cohort 4): M5049 Dose DEXPERIMENTAL -
Part A: PlaceboPLACEBO_COMPARATOR -
Part B (Cohort 5): M5049 Dose EEXPERIMENTAL -
Part B: PlaceboPLACEBO_COMPARATOR -
Part A: M5049EXPERIMENTAL -
Part B: M5049EXPERIMENTAL -
Part C: M5049EXPERIMENTAL -

Interventions

NameTypeDescription
M5049DRUGParticipants will receive low oral dose of M5049, twice daily in Part A.
PlaceboDRUGParticipants will receive placebo matched to M5049.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: * Active systemic lupus erythematosus (SLE) with a Cutaneous lupus erythematosus disease area and activity index (CLASI-A) greater than or equal to \[\>= \] 6 and/or at least one active SLE clinical manifestation according to Systemic Lupus Erythematosus Disease Activity Index 2...

Countries:BulgariaGermanyMoldovaNorth MacedoniaSpainUkraine
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Frequently asked questions about M5049

What is M5049 used for?

M5049 is an investigational small molecule being studied for Systemic Lupus Erythematosus (SLE) and Cutaneous Lupus Erythematosus (CLE). It has also been evaluated in healthy participants to assess safety and tolerability. The drug is in Phase 1 clinical development and is not yet approved by regulatory authorities.

Who makes M5049?

M5049 is being developed by Merck KGaA, a science and technology company. The company's stock is traded over-the-counter under the ticker MKGAF. Merck KGaA is conducting clinical trials to evaluate the safety and efficacy of M5049 in lupus indications.

What phase is M5049 in?

M5049 is in Phase 1 clinical development. Two Phase 1 trials have been completed: one in healthy participants and one in participants with Systemic Lupus Erythematosus and Cutaneous Lupus Erythematosus. The drug remains investigational and has not received FDA approval.

What clinical trials is M5049 in?

M5049 has been studied in two completed Phase 1 trials. NCT03676322 was a single and multiple ascending dose study in 96 healthy participants in Germany. NCT04647708 evaluated M5049 in 25 participants with Systemic Lupus Erythematosus and Cutaneous Lupus Erythematosus across multiple European countries.

Is M5049 the same as any other drug?

No alternative names for M5049 have been disclosed. The drug is identified solely by its code name M5049 in clinical trial registrations and development documentation. It is a distinct investigational small molecule being developed by Merck KGaA.