Trial Outcomes & Findings for Evaluation of the Safety, Tolerability, and Efficacy of MDGN-002 in Adults With Moderate to Severe Active Crohn's Disease or Ulcerative Colitis (NCT NCT03169894)

Last Updated: 2024-02-26

Results Overview

The SES-CD is assessed through endoscopic review of 5 predefined gastrointestinal (GI) segments (ileum; right colon; transverse colon; left colon; rectum). For each segment, 4 endoscopic variables are assessed (presence of ulcers, ulcerated surface, affected surface, and presence of narrowing). Each variable is scored from 0 to 3 with higher scores indicating more severe symptoms. For each variable, the total score is calculated as the sum across all segments of the GI tract. The SES-CD total score, ranging from 0-60, is calculated as the sum of all variable total scores with a higher score indicating more severe endoscopic activity

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

8 participants

Primary outcome timeframe

Baseline to Visit 10 (Day 56) or early termination

Results posted on

2024-02-26

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1 MDGN-002 1.0 mg/kg	Cohort 2 MDGN-002 3.0 mg/kg
Overall Study STARTED	4	4
Overall Study Safety Analysis Set	4	4
Overall Study Full Analysis Set	4	4
Overall Study PK Analysis Set	4	3
Overall Study COMPLETED	4	4
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

BMI not available for all subjects

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: Baseline to Visit 10 (Day 56) or early termination

Population: Full Analysis Set, with data analyzed and presented only for subjects with both a Baseline and Visit 10 or Early Termination assessment

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants MDGN-002 1.0 mg/kg	Cohort 2 n=4 Participants MDGN-002 3.0 mg/kg
Change From Baseline in Simple Endoscopic Score for Crohn's Disease (SES-CD)	-4.0 score on a scale Standard Deviation 4.36	1.3 score on a scale Standard Deviation 7.50

SECONDARY outcome

Timeframe: Baseline to Visit 10 (Day 56) or early termination.

Population: Full Analysis Set, with data analyzed and presented only for subjects with both a Baseline and Visit 10 or Early Termination assessment

The Crohn's Disease Activity Index (CDAI) consists of the following 8 items: abdominal pain, number of liquid stools, general well-being, extraintestinal complication, use of antidiarrheal drugs, abdominal mass, hematocrit, and body weight. Information on abdominal pain, general well-being, and frequency of loose and watery stools was taken from a daily diary completed by the subject. Total CDAI scores can range from 0 to approximately 600 with higher scores indicating more active disease. Disease severity as measured by CDAI is categorized as: Remission (\<150), Mildly active disease (150 - 219); Moderately active disease (220 - 450); Severe disease (\> 450).

Outcome measures

Outcome measures
Measure	Cohort 1 n=2 Participants MDGN-002 1.0 mg/kg	Cohort 2 n=1 Participants MDGN-002 3.0 mg/kg
Change From Baseline in Crohn's Disease Activity Index (CDAI)	-19.2 score on a scale Standard Deviation 38.34	-123.6 score on a scale Standard Deviation NA a standard deviation cannot be calculated for a single measure.

SECONDARY outcome

Timeframe: Baseline to Visit 10 (Day 56) or Early Termination

Population: Full Analysis Set

The IBD-Q is a 32-item questionnaire validated to measure quality of life in Crohn's disease subjects. The IBD-Q assesses the dimensions of bowel function, emotional status, systemic symptoms, and social function. Each of the 32 items is scored on a 1 to 7 scale, where higher scores represent a more positive response, and better outcome. The IBD-Q total score is calculated as the sum of all 32 items in the questionnaire, ranging from 32 to 224.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants MDGN-002 1.0 mg/kg	Cohort 2 n=4 Participants MDGN-002 3.0 mg/kg
Change From Baseline in Inflammatory Bowel Disease Questionnaire (IBD-Q).	43.5 score on a scale Standard Deviation 29.01	47.0 score on a scale Standard Deviation 31.59

SECONDARY outcome

Timeframe: Baseline to Visit 10 (Day 56) or Early Termination

Population: Full Analysis Set, with data analyzed and presented only for subjects with both a Baseline and Visit 10 or Early Termination assessment

Subjects reported their daily stool frequency including loose and/or watery stools via a diary. The stool frequency including the number of loose and/or watery stools per day, equivalent to a score of a 6 or 7 on the Bristol Stool Scale, was recorded. Loose stools were described as fluffy pieces with ragged edges, a mushy stool. Watery stools were described as watery, no solid pieces.

Outcome measures

Outcome measures
Measure	Cohort 1 n=2 Participants MDGN-002 1.0 mg/kg	Cohort 2 n=2 Participants MDGN-002 3.0 mg/kg
Change From Baseline in Total Number of Stools Daily	1.37 number of total daily stools Standard Deviation 0.286	-0.99 number of total daily stools Standard Deviation 4.462

SECONDARY outcome

Timeframe: Baseline to Visit 10 (Day 56) or Early Termination

Population: Full Analysis Set with data analyzed and presented only for subjects with both a Baseline and Visit 10 or Early Termination assessment

Subjects reported their daily assessment of stool frequency including loose and/or watery stools via a diary. The stool frequency including the number of loose and/or watery stools per day, equivalent to a score of a 6 or 7 on the Bristol Stool Scale, was recorded. Loose stools were described as fluffy pieces with ragged edges, a mushy stool. Watery stools were described as watery, no solid pieces.

Outcome measures

Outcome measures
Measure	Cohort 1 n=2 Participants MDGN-002 1.0 mg/kg	Cohort 2 n=2 Participants MDGN-002 3.0 mg/kg
Change From Baseline in Total Number of Loose/Watery Stools Daily	0.90 number of daily loose/watery stools Standard Deviation 1.077	-1.07 number of daily loose/watery stools Standard Deviation 5.758

SECONDARY outcome

Timeframe: Baseline to Visit 10 (Day 56) or Early Termination

Population: Full Analysis Set, with data analyzed and presented only for subjects with both a Baseline and Visit 10 or Early Termination assessment

Subjects reported their daily assessment of abdominal pain via a diary. Abdominal pain was assessed on a scale of 0 to 3 with higher values indicating greater pain severity.

Outcome measures

Outcome measures
Measure	Cohort 1 n=2 Participants MDGN-002 1.0 mg/kg	Cohort 2 n=2 Participants MDGN-002 3.0 mg/kg
Change From Baseline in Abdominal Pain	-0.75 score on a scale Standard Deviation 0.354	0.18 score on a scale Standard Deviation 0.455

SECONDARY outcome

Timeframe: Baseline to Visit 10 (Day 56) or Early Termination

Population: Full Analysis Set, with data analyzed and presented only for subjects with both a Baseline and Visit 10 or Early Termination assessment

Subjects reported their daily assessment of well-being via a diary. General well being was assessed on a scale of 0 to 4, with higher values indicating a poorer condition of health.

Outcome measures

Outcome measures
Measure	Cohort 1 n=2 Participants MDGN-002 1.0 mg/kg	Cohort 2 n=2 Participants MDGN-002 3.0 mg/kg
Change From Baseline in General Well-Being	-0.11 score on a scale Standard Deviation 0.859	-0.27 score on a scale Standard Deviation 0.623

Adverse Events

Cohort 1

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Cohort 2

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1 n=4 participants at risk MDGN-002 1.0 mg/kg	Cohort 2 n=4 participants at risk MDGN-002 3.0 mg/kg
Blood and lymphatic system disorders Microcytic anaemia	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Gastrointestinal disorders Abdominal distension	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Gastrointestinal disorders Abdominal pain lower	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Gastrointestinal disorders Crohn's disease	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Gastrointestinal disorders Diarrhoea	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Gastrointestinal disorders Abdominal pain	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Metabolism and nutrition disorders Dehydration	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Metabolism and nutrition disorders Hypokalaemia	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Metabolism and nutrition disorders Malnutrition	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.
Nervous system disorders Syncope	0.00% 0/4 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.	25.0% 1/4 • Number of events 1 • All adverse events were collected from the time of the informed consent until the final safety follow-up visit (Day 84 or 28 days after Early Termination). This included events occurring during the screening phase of the study, regardless of whether investigational product was administered. Therefore all serious and non-serious events are included regardless of meeting or not meeting the study definition of treatment emergent.

Other adverse events

Additional Information

Garry Neil, MD

Avalo Therapeutics, Inc.

Phone: 908-720-2691

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Cohort 1 n=4 Participants MDGN-002 1.0 mg/kg	Cohort 2 n=4 Participants MDGN-002 3.0 mg/kg	Total n=8 Participants Total of all reporting groups
Age, Continuous	45.3 years STANDARD_DEVIATION 14.66 • n=4 Participants	26.5 years STANDARD_DEVIATION 5.07 • n=4 Participants	35.9 years STANDARD_DEVIATION 14.27 • n=8 Participants
Sex: Female, Male Female	3 Participants n=4 Participants	2 Participants n=4 Participants	5 Participants n=8 Participants
Sex: Female, Male Male	1 Participants n=4 Participants	2 Participants n=4 Participants	3 Participants n=8 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=4 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=4 Participants	4 Participants n=4 Participants	8 Participants n=8 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=4 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=4 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Race (NIH/OMB) Asian	0 Participants n=4 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=4 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Race (NIH/OMB) Black or African American	0 Participants n=4 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Race (NIH/OMB) White	4 Participants n=4 Participants	4 Participants n=4 Participants	8 Participants n=8 Participants
Race (NIH/OMB) More than one race	0 Participants n=4 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=4 Participants	0 Participants n=4 Participants	0 Participants n=8 Participants
Region of Enrollment United States	4 participants n=4 Participants	4 participants n=4 Participants	8 participants n=8 Participants
Body Mass Index (BMI)	27.37 kg/m2 STANDARD_DEVIATION 10.192 • n=4 Participants • BMI not available for all subjects	15.84 kg/m2 STANDARD_DEVIATION 4.428 • n=3 Participants • BMI not available for all subjects	22.43 kg/m2 STANDARD_DEVIATION 9.820 • n=7 Participants • BMI not available for all subjects
Time Since Diagnosis (Years)	25.2 years STANDARD_DEVIATION 6.17 • n=4 Participants	6.2 years STANDARD_DEVIATION 2.18 • n=4 Participants	15.7 years STANDARD_DEVIATION 11.00 • n=8 Participants