Trial Outcomes & Findings for A Study of FT 4101 in Overweight/Obese Participants With Non-alcoholic Steatohepatitis (NCT NCT04004325)

NCT ID: NCT04004325

Last Updated: 2025-10-20

Results Overview

TEAEs were defined as an adverse events (AEs) with an onset that occurred after receiving the first dose of the study drug (AE start date greater than or equal to \[\>=\] first dose date) and within 30 days after receiving the last dose of the study drug (AE start date - last dose date less than or equal to \[\<=\] 30). Number of participants with TEAEs are reported.

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 14 participants
• Primary outcome timeframe: From start of study drug administration up to 20 weeks
• Results posted on: 2025-10-20

Participant Flow

Participants were randomized at a ratio of 2:1 in FT-4101 treatment group and placebo.

Participant milestones

Measure	FT-4101 3.0 mg Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Overall Study STARTED	9	5
Overall Study COMPLETED	9	5
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study of FT 4101 in Overweight/Obese Participants With Non-alcoholic Steatohepatitis

Baseline characteristics by cohort

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Total n=14 Participants Total of all reporting groups
Age, Continuous	43.8 Years STANDARD_DEVIATION 10.52 • n=5 Participants	47.0 Years STANDARD_DEVIATION 15.76 • n=7 Participants	44.9 Years STANDARD_DEVIATION 12.13 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	2 Participants n=7 Participants	9 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	8 Participants n=5 Participants	5 Participants n=7 Participants	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants	5 Participants n=7 Participants	14 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: From start of study drug administration up to 20 weeks

Population: The Safety analysis set included all randomized participants who received investigational product (IP) (FT-4101 or placebo) or deuterated water.

TEAEs were defined as an adverse events (AEs) with an onset that occurred after receiving the first dose of the study drug (AE start date greater than or equal to [>=] first dose date) and within 30 days after receiving the last dose of the study drug (AE start date - last dose date less than or equal to [<=] 30). Number of participants with TEAEs are reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	3 Participants	3 Participants

PRIMARY outcome

Timeframe: Up to 20 weeks

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Number of participants with clinically significant changes in laboratory values (hematology, serum chemistry, and urinalysis) are reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Number of Participants With Clinically Significant Changes in Laboratory Values	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to 20 weeks

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Number of participants with clinically significant changes in physical examination are reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Number of Participants With Clinically Significant Changes in Physical Examination	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in blood pressure (systolic and diastolic) are reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Vital Signs: Blood Pressure (BP) Systolic BP	-0.2 millimeters of mercury (mmHg) Standard Deviation 16.45	-5.2 millimeters of mercury (mmHg) Standard Deviation 8.44
Change From Baseline in Vital Signs: Blood Pressure (BP) Diastolic BP	0.4 millimeters of mercury (mmHg) Standard Deviation 14.93	-2.4 millimeters of mercury (mmHg) Standard Deviation 6.11

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in heart rate is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Vital Signs: Heart Rate (HR)	4.4 Beats per minutes (beats/min) Standard Deviation 8.32	0.2 Beats per minutes (beats/min) Standard Deviation 2.59

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in respiratory rate is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Vital Signs: Respiratory Rate	-1.6 Breaths per minute (breaths/min) Standard Deviation 1.42	-0.6 Breaths per minute (breaths/min) Standard Deviation 1.34

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in temperature is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Vital Signs: Temperature	0.22 Degree Celsius (C) Standard Deviation 0.239	-0.08 Degree Celsius (C) Standard Deviation 0.409

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in ECG mean heart rate is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in 12-lead Electrocardiogram (ECG) Parameters: ECG Mean Heart Rate	0.3 beats/min Standard Deviation 11.46	2.6 beats/min Standard Deviation 6.23

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in QT interval is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in 12-lead ECG Parameters: QT Interval	0.2 milliseconds (msec) Standard Deviation 14.28	-4.2 milliseconds (msec) Standard Deviation 22.80

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in PR interval is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in 12-lead ECG Parameters: PR Interval	-7.0 msec Standard Deviation 13.63	2.2 msec Standard Deviation 10.64

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in QRS interval is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in 12-lead ECG Parameters: QRS Interval	1.9 msec Standard Deviation 8.62	0.8 msec Standard Deviation 8.38

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in RR interval is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in 12-lead ECG Parameters: RR Interval	1.7 msec Standard Deviation 138.73	-40.8 msec Standard Deviation 93.04

PRIMARY outcome

Timeframe: Baseline, Day 92

Population: The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Change from baseline in QTcF interval is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=9 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in 12-lead ECG Parameters: QTc (Corrected) Using the Fridericia Correction (QTcF) Interval	0.6 msec Standard Deviation 10.92	1.0 msec Standard Deviation 14.00

PRIMARY outcome

Timeframe: At week 12

Population: The preliminary efficacy analysis set included all participants who received FT-4101 or placebo with at least one quantifiable baseline and one post-baseline MRI-PDFF without major protocol deviations or violations that would have an impact on pharmacodynamics (PD) (Specifically MRI-PDFF).

Percent change from baseline in liver fat on MRI-PDFF is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Percent Change From Baseline in Liver Fat on Magnetic Resonance Imaging- Proton Density Fat Fraction (MRI-PDFF)	-18.02 Percent change of liver fat Standard Deviation 20.066	25.98 Percent change of liver fat Standard Deviation 15.670

PRIMARY outcome

Timeframe: At week 12

Population: The preliminary efficacy analysis set included all participants who received FT-4101 or placebo with at least one quantifiable baseline and one post-baseline MRI-PDFF without major protocol deviations or violations that would have an impact on PD (Specifically MRI-PDFF).

Change from baseline in percentage of liver fat on MRI-PDFF is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Liver Fat on Magnetic Resonance Imaging- Proton Density Fat Fraction (MRI-PDFF)	-3.65 Percentage of liver fat Standard Deviation 4.389	4.16 Percentage of liver fat Standard Deviation 2.236

SECONDARY outcome

Timeframe: At week 6

Population: The preliminary efficacy analysis set included all participants who received FT-4101 or placebo with at least one quantifiable baseline and one post-baseline MRI-PDFF without major protocol deviations or violations that would have an impact on PD (Specifically MRI-PDFF).

Change from baseline in percentage of liver fat on MRI-PDFF is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Liver Fat on MRI-PDFF	-0.10 Percentage of liver fat Standard Deviation 3.395	3.58 Percentage of liver fat Standard Deviation 4.060

SECONDARY outcome

Timeframe: At week 6

Population: The preliminary efficacy analysis set included all participants who received FT-4101 or placebo with at least one quantifiable baseline and one post-baseline MRI-PDFF without major protocol deviations or violations that would have an impact on PD (Specifically MRI-PDFF).

Percent change from baseline in liver fat on MRI-PDFF is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Percent Change From Baseline in Liver Fat on MRI-PDFF	-0.73 Percent change of liver fat Standard Deviation 16.272	22.07 Percent change of liver fat Standard Deviation 27.154

SECONDARY outcome

Timeframe: At week 12

Population: The preliminary efficacy analysis set included all participants who received FT-4101 or placebo with at least one quantifiable baseline and one post-baseline MRI-PDFF without major protocol deviations or violations that would have an impact on PD (Specifically MRI-PDFF).

Percentage of participants experiencing a relative reduction of 30% or greater of liver fat as assessed by MRI-PDFF at week 12 are reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Percentage of Participants Experiencing a Relative Reduction of 30% or Greater of Liver Fat as Assessed by MRI-PDFF	25.0 Percentage of participants	0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline, Day 92

Population: The pharmacodynamic (PD) analysis set was to include all participants who received FT-4101 or placebo without major protocol deviations or violations that would have had an impact on PD.

Change from baseline in ALT is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Alanine Aminotransferase (ALT)	-3.0 units per liter (U/L) Standard Error 6.29	40.8 units per liter (U/L) Standard Error 8.02

SECONDARY outcome

Timeframe: Baseline, Day 92

Population: The pharmacodynamic (PD) analysis set was to include all participants who received FT-4101 or placebo without major protocol deviations or violations that would have had an impact on PD.

Change from baseline in AST is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Aspartate Aminotransferase (AST)	-1.161 U/L Standard Error 4.2789	12.457 U/L Standard Error 5.4320

SECONDARY outcome

Timeframe: Baseline, Day 92

Population: The pharmacodynamic (PD) analysis set was to include all participants who received FT-4101 or placebo without major protocol deviations or violations that would have had an impact on PD.

Change from baseline in γGT is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Gamma-glutamyl Transferase (γGT)	-2.058 U/L Standard Error 9.1733	46.293 U/L Standard Error 11.6580

SECONDARY outcome

Timeframe: Baseline, Day 92

Population: The pharmacodynamic (PD) analysis set was to include all participants who received FT-4101 or placebo without major protocol deviations or violations that would have had an impact on PD.

Change from baseline in alkaline phosphatase is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Alkaline Phosphatase	1.023 U/L Standard Error 2.2841	13.764 U/L Standard Error 2.8903

SECONDARY outcome

Timeframe: Baseline, Day 92

Population: The pharmacodynamic (PD) analysis set was to include all participants who received FT-4101 or placebo without major protocol deviations or violations that would have had an impact on PD.

Change from baseline in total bilirubin is reported.

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 Participants Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Change From Baseline in Total Bilirubin	-0.59 micromoles per liter (umol/L) Standard Error 0.735	-1.09 micromoles per liter (umol/L) Standard Error 0.932

SECONDARY outcome

Timeframe: Cycle 1 (Day 1), Cycle 4 (Day 14); each Cycle length = 21 days

Population: The pharmacokinetic (PK) analysis set included all participants who received FT-4101 with sufficient evaluable PK concentration data appropriate for the evaluation of interest without major protocol deviations or violations that would have an impact on the absorption, distribution, metabolism, or excretion.

Maximum plasma concentration (Cmax) of FT-4101 is reported

Outcome measures

Measure	FT-4101 3.0 mg n=8 Participants Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Maximum Plasma Concentration (Cmax) of FT-4101 Cycle 1 (Day 1)	62.3 nanograms per milliliter (ng/mL) Standard Deviation 22.2	—
Maximum Plasma Concentration (Cmax) of FT-4101 Cycle 4 (Day 14)	118 nanograms per milliliter (ng/mL) Standard Deviation 38.6	—

SECONDARY outcome

Timeframe: Cycle 1 (Day 1), Cycle 4 (Day 14); each Cycle length = 21 days

Population: Data for this endpoint was not collected and analysed as there were no participants.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Cycle 1 (Day 1), Cycle 4 (Day 14); each Cycle length = 21 days

Population: Data for this endpoint was not collected and analysed as there were no participants.

Outcome measures

Outcome data not reported

Adverse Events

FT-4101 3.0 mg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	FT-4101 3.0 mg n=9 participants at risk Participants received 3.0 mg of FT-4101 capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).	Placebo n=5 participants at risk Participants received FT-4101 matching placebo capsule orally once daily for 2 weeks, followed by 1 week of no treatment. This cycle continued until the end of week 12, for up to 4 dosing cycle (each cycle 21 days).
Cardiac disorders Atrioventricular block first degree	0.00% 0/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	20.0% 1/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.
Infections and infestations Gastroenteritis	0.00% 0/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	20.0% 1/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.
Infections and infestations Urinary tract infection	11.1% 1/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	0.00% 0/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.
Investigations Alanine aminotransferase increased	0.00% 0/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	20.0% 1/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.
Investigations White blood cell count increased	11.1% 1/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	0.00% 0/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.
Nervous system disorders Headache	0.00% 0/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	20.0% 1/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.
Gastrointestinal disorders Abdominal pain	11.1% 1/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	0.00% 0/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.
General disorders Non-cardiac chest pain	11.1% 1/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	0.00% 0/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.
Skin and subcutaneous tissue disorders Urticaria	11.1% 1/9 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.	0.00% 0/5 • From start of study drug administration up to 20 weeks The Safety analysis set included all randomized participants who received IP (FT-4101 or placebo) or deuterated water.

Additional Information

Clinical Reporting Office (2834)

FORMA Therapeutics, Inc.

Phone: (+1) 866-867-7178

Email: clinicaltrials@novonordisk.com

Results disclosure agreements

• Principal investigator is a sponsor employee At the end of the trial, one or more scientific publications may be prepared collaboratively by the investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property
• Publication restrictions are in place

Restriction type: OTHER