Trial Outcomes & Findings for Aldafermin (NGM282) for Chronic Diarrhea Due to Bile Acid Malabsorption (BAM) (NCT NCT05130047)

NCT ID: NCT05130047

Last Updated: 2023-10-12

Results Overview

Fasting serum C4 is measured by liquid chromatography-mass spectrometry.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 30 participants
• Primary outcome timeframe: 28 days
• Results posted on: 2023-10-12

Participant Flow

Participant milestones

Measure	Aldafermin (NGM282) Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Overall Study STARTED	15	15
Overall Study COMPLETED	15	15
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution	Total n=30 Participants Total of all reporting groups
Age, Continuous	49.0 years n=15 Participants	43.0 years n=15 Participants	43.0 years n=30 Participants
Sex: Female, Male Female	12 Participants n=15 Participants	14 Participants n=15 Participants	26 Participants n=30 Participants
Sex: Female, Male Male	3 Participants n=15 Participants	1 Participants n=15 Participants	4 Participants n=30 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	15 participants n=15 Participants	15 participants n=15 Participants	30 participants n=30 Participants

PRIMARY outcome

Timeframe: 28 days

Fasting serum C4 is measured by liquid chromatography-mass spectrometry.

Outcome measures

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Fasting Serum C4 Levels	2.2 ng/ML Interval 1.6 to 3.3	50.4 ng/ML Interval 25.8 to 106.0

PRIMARY outcome

Timeframe: Baseline, 28 days

Stool consistency as reported by the patient in daily bowel pattern diaries. Stool consistency is based on Bristol Stool Form Scale (BSFS) 1: Hard lumps; 2:Lumpy sausage; 3: Cracked sausage; 4: Smooth sausage; 5: Soft lumps; 6: Mushy; 7: Watery. spectrometry.

Outcome measures

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Change in Stool Consistency From Baseline to Day 28	-0.4 score on a scale Interval -1.2 to 0.1	-0.02 score on a scale Interval -0.4 to 0.3

SECONDARY outcome

Timeframe: 14 days, 28 days

Stool consistency as reported by the patient in daily bowel pattern diaries. Stool consistency is based on Bristol Stool Form Scale (BSFS) 1: Hard lumps; 2:Lumpy sausage; 3: Cracked sausage; 4: Smooth sausage; 5: Soft lumps; 6: Mushy; 7: Watery.

Outcome measures

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Stool Consistency Day 14	4.6 score on a scale Interval 4.3 to 5.0	4.8 score on a scale Interval 4.0 to 5.3
Stool Consistency Day 28	4.4 score on a scale Interval 4.2 to 5.0	5.1 score on a scale Interval 4.4 to 5.5

SECONDARY outcome

Timeframe: baseline, 28 days

Abdominal pain rated by patient using an 11-point scale, 0 to 10 inclusive with 0 as none and 10 as worst imaginable abdominal pain.

Outcome measures

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Abdominal Pain Score Baseline	0.9 score on a scale Interval 0.0 to 3.6	0.4 score on a scale Interval 0.0 to 1.0
Abdominal Pain Score Day 28	0.6 score on a scale Interval 0.0 to 1.3	0.5 score on a scale Interval 0.1 to 1.1

SECONDARY outcome

Timeframe: baseline, 28 days

The total number of bowel movements per day reported by the participant in the daily bowel pattern diary.

Outcome measures

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Bowel Movements Baseline	2.3 bowel movements per day Interval 1.9 to 3.2	2.3 bowel movements per day Interval 1.8 to 3.0
Bowel Movements Day 28	2.3 bowel movements per day Interval 2.1 to 3.6	2.1 bowel movements per day Interval 1.7 to 2.7

SECONDARY outcome

Timeframe: Baseline, 14 days, 28 days

Fecal secretory bile acids are expressed as a percentage of the total bile acids as measured by high performance liquid chromatography-mass spectrometry.

Outcome measures

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Proportion of Fecal Secretory (CDCA + DCA) Bile Acid as Measured in a Random Stool Sample by a Validated Laboratory Assay. Day 28	45.5 percentage of the total bile acids Interval 39.8 to 55.6	60.0 percentage of the total bile acids Interval 46.3 to 65.1
Proportion of Fecal Secretory (CDCA + DCA) Bile Acid as Measured in a Random Stool Sample by a Validated Laboratory Assay. Baseline	63.8 percentage of the total bile acids Interval 58.3 to 66.7	63.2 percentage of the total bile acids Interval 57.4 to 66.6
Proportion of Fecal Secretory (CDCA + DCA) Bile Acid as Measured in a Random Stool Sample by a Validated Laboratory Assay. Day 14	43.2 percentage of the total bile acids Interval 32.5 to 53.5	59.2 percentage of the total bile acids Interval 53.1 to 63.9

SECONDARY outcome

Timeframe: Baseline,14 days, 28 days

Fecal primary bile acids are expressed as a percentage of the total bile acids as measured by high performance liquid chromatography-mass spectrometry.

Outcome measures

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Proportion of Fecal Primary (CDCA + CA) Bile Acid as Measured in a Random Stool Sample by a Validated Laboratory Assay. Baseline	5.4 percentage of the total bile acids Interval 1.9 to 14.6	5.2 percentage of the total bile acids Interval 2.0 to 12.9
Proportion of Fecal Primary (CDCA + CA) Bile Acid as Measured in a Random Stool Sample by a Validated Laboratory Assay. Day 14	10.3 percentage of the total bile acids Interval 3.3 to 15.4	2.8 percentage of the total bile acids Interval 0.7 to 9.8
Proportion of Fecal Primary (CDCA + CA) Bile Acid as Measured in a Random Stool Sample by a Validated Laboratory Assay. Day 28	9.9 percentage of the total bile acids Interval 3.0 to 20.0	14.3 percentage of the total bile acids Interval 7.4 to 27.4

SECONDARY outcome

Timeframe: baseline,14 days, 28 days

Total fecal bile acids concentration in micromoles of bile acid per gram stool as measured by high performance liquid chromatography-mass spectrometry.

Outcome measures

Measure	Aldafermin (NGM282) n=15 Participants Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 Participants A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Total Fecal Bile Acid Concentration in a Random Stool Sample as Measured by a Validated Laboratory Assay. Baseline	3.1 mmol/g Interval 1.9 to 4.0	3.5 mmol/g Interval 1.9 to 4.3
Total Fecal Bile Acid Concentration in a Random Stool Sample as Measured by a Validated Laboratory Assay. Day 14	0.5 mmol/g Interval 0.3 to 0.7	2.7 mmol/g Interval 1.2 to 5.1
Total Fecal Bile Acid Concentration in a Random Stool Sample as Measured by a Validated Laboratory Assay. Day 28	0.5 mmol/g Interval 0.3 to 0.8	3.6 mmol/g Interval 1.0 to 7.3

SECONDARY outcome

Timeframe: baseline, 14 days, 28 days

Population: Data was not collected nor analyzed

Validated aldafermin concentration (PK) laboratory assay

Outcome measures

Outcome data not reported

Adverse Events

Aldafermin (NGM282)

Serious events: 1 serious events

Other events: 14 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Measure	Aldafermin (NGM282) n=15 participants at risk Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 participants at risk A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Cardiac disorders Myocardial infarction	6.7% 1/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	0.00% 0/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days

Other adverse events

Measure	Aldafermin (NGM282) n=15 participants at risk Aldafermin (NGM282) is an investigational medication. It is an engineered analog of FGF-19 which reduces synthesis of bile acids and diarrhea caused by elevated bile acids. Participants received aldafermin (NGM282) 1 mg given by subcutaneous injection once daily for 28 days. Aldafermin: 1 mg solution	Placebo n=15 participants at risk A placebo looks exactly like the study drug but contains no active ingredients. It is used to learn if the effects seen are truly from the study drug. Participants received placebo solution matching aldafermin (NGM282) given by subcutaneous injection once daily for 28 days. Placebo: Aldafermin placebo solution
Gastrointestinal disorders Increased diarrhea	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	6.7% 1/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Gastrointestinal disorders Abdominal pain	6.7% 1/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Gastrointestinal disorders Other gastrointestinal symptoms	20.0% 3/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	20.0% 3/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Investigations Increased LDL Cholesterol	26.7% 4/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	0.00% 0/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Skin and subcutaneous tissue disorders Irritation at injection site	20.0% 3/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	6.7% 1/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Infections and infestations Infections	33.3% 5/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Nervous system disorders Headache	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Musculoskeletal and connective tissue disorders Musculoskeletal	20.0% 3/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Metabolism and nutrition disorders Metabolism and Nutrition	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	0.00% 0/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days
Renal and urinary disorders Reanl and urinary	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days	13.3% 2/15 • Adverse Events were collected from baseline to end of study for each participant, approximately 28 days

Additional Information

Michael Camilleri, M.D., D.Sc.

Mayo Clinic

Phone: 507-284-2687

Email: camilleri.michael@mayo.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place