Trial Outcomes & Findings for OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis (NCT NCT03746483)

Last Updated: 2022-06-06

Results Overview

The Coefficient of Fat Absorption (CFA%) is defined as: \[72-hour fat intake (g) - 72-hour fat excretion (g)/72-hour fat intake(g)\] x 100 = CFA% The threshold for CFA results (\>80%) is considered clinically significant for treatment effectiveness by the FDA.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

41 participants

Primary outcome timeframe

3 weeks

Results posted on

2022-06-06

Participant Flow

Participant milestones

Participant milestones
Measure	MS1819 2240 mg/Day (3 Weeks) Then PERT Pre-study Dose (3 Weeks) Participants were first randomized to receive MS1819 first and PERT during the crossover phase	PERT Pre-study Dose (3 Weeks) Then MS1819 2240 mg/Day (3 Weeks) Participants were first randomized to receive PERT first and MS1819 during the crossover phase
Overall Study STARTED	21	20
Overall Study COMPLETED	15	17
Overall Study NOT COMPLETED	6	3

Reasons for withdrawal

Reasons for withdrawal
Measure	MS1819 2240 mg/Day (3 Weeks) Then PERT Pre-study Dose (3 Weeks) Participants were first randomized to receive MS1819 first and PERT during the crossover phase	PERT Pre-study Dose (3 Weeks) Then MS1819 2240 mg/Day (3 Weeks) Participants were first randomized to receive PERT first and MS1819 during the crossover phase
Overall Study Adverse Event	0	1
Overall Study Lack of Efficacy	3	2
Overall Study Lost to Follow-up	1	0
Overall Study Protocol Violation	2	0

Baseline Characteristics

OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	MS1819 - PERT Sequence n=17 Participants Participants were first randomized to receive MS1819 first and PERT during the crossover phase	PERT - MS1819 Sequence n=19 Participants Participants were first randomized to receive PERT first and MS1819 during the crossover phase	Total n=36 Participants Total of all reporting groups
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	28.5 years STANDARD_DEVIATION 7.60 • n=5 Participants	29.0 years STANDARD_DEVIATION 7.97 • n=7 Participants	28.8 years STANDARD_DEVIATION 7.69 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	11 Participants n=7 Participants	15 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants	8 Participants n=7 Participants	21 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	16 Participants n=5 Participants	19 Participants n=7 Participants	35 Participants n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	17 Participants n=5 Participants	19 Participants n=7 Participants	36 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	0 Participants n=7 Participants	1 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
Region of Enrollment United States	13 participants n=5 Participants	13 participants n=7 Participants	26 participants n=5 Participants
Region of Enrollment Poland	4 participants n=5 Participants	6 participants n=7 Participants	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 3 weeks

Population: modified Intent To Treat (mITT)

Outcome measures

Outcome measures
Measure	MS1819 n=33 Participants Measured while participants on MS1819	PERT n=35 Participants Measured while participants on PERT
Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)	55.6 % CFA Standard Deviation 21.44	86.2 % CFA Standard Deviation 7.39

PRIMARY outcome

Timeframe: 6 weeks

Population: Safety Population

Number of participants reporting 1 or more adverse events

Outcome measures

Outcome measures
Measure	MS1819 n=40 Participants Measured while participants on MS1819	PERT n=38 Participants Measured while participants on PERT
Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE) Number of subjects with any TEAEs	13 Participants	6 Participants
Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE) Number of subjects with TEAEs related to	2 Participants	0 Participants

PRIMARY outcome

Timeframe: 6 weeks

Population: Safety Population

Number of Treatment emergent adverse events

Outcome measures

Outcome measures
Measure	MS1819 n=40 Participants Measured while participants on MS1819	PERT n=38 Participants Measured while participants on PERT
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs) Number of Treatment-emergent adverse event (TEAEs)	19 Number of TEAEs reported	8 Number of TEAEs reported
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs) Number of serious TEAEs reported	0 Number of TEAEs reported	0 Number of TEAEs reported

SECONDARY outcome

Timeframe: 6 weeks

Population: mITT. Samples from 4 patients were not available for analysis due to discontinuation from the study. Three patients discontinued in the MS1819 group while 1 patient discontinued in the PERT group.

The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights

Outcome measures

Outcome measures
Measure	MS1819 n=33 Participants Measured while participants on MS1819	PERT n=35 Participants Measured while participants on PERT
Stool Weights	1394.0 grams Standard Deviation 590.63	727.3 grams Standard Deviation 334.29

SECONDARY outcome

Timeframe: 3 weeks

Population: Data presented at 3 weeks post first dose.

The relative efficacy of MS1819 compared to porcine PERT will be assessed using signs and symptoms of malabsorption. Abdominal pain, bloating, flatulence, increased stool quantity, and worsening of overall bowel habit were graded as 0 = none, 1 = mild, 2 = moderate, or 3 = severe.

Outcome measures

Outcome measures
Measure	MS1819 n=36 Participants Measured while participants on MS1819	PERT n=36 Participants Measured while participants on PERT
Signs and Symptoms of Malabsorption Grade 0	15 Participants	32 Participants
Signs and Symptoms of Malabsorption Grade 1	12 Participants	3 Participants
Signs and Symptoms of Malabsorption Grade 2	6 Participants	1 Participants
Signs and Symptoms of Malabsorption Grade 3	1 Participants	0 Participants
Signs and Symptoms of Malabsorption Unknown	2 Participants	0 Participants

SECONDARY outcome

Timeframe: 3 weeks per group.

CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.

Outcome measures

Outcome measures
Measure	MS1819 n=33 Participants Measured while participants on MS1819	PERT n=35 Participants Measured while participants on PERT
Coefficient of Nitrogen Absorption (CNA)	93.0 % CNA Standard Deviation 2.54	97.2 % CNA Standard Deviation 1.21

Adverse Events

MS1819

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

PERT

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Chief Medical Officer

First Wave BioPharma Inc.

Phone: (561) 589-7020

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	MS1819 n=40 participants at risk AE reported during treatment with MS1819	PERT n=38 participants at risk AE reported during treatment with PERT
Infections and infestations Infective pulmonary exacerbation of cystic fibrosis	5.0% 2/40 • Number of events 2 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	2.6% 1/38 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Infections and infestations Herpes Zoster	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Infections and infestations Sinusitis	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	2.6% 1/38 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Infections and infestations Upper respiratory tract infection	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Infections and infestations Vulvovaginal mycotic infection	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	2.6% 1/38 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Gastrointestinal disorders Constipation	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Gastrointestinal disorders Nausea	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Gastrointestinal disorders Oral pain	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Gastrointestinal disorders Abdominal pain	0.00% 0/40 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	2.6% 1/38 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
General disorders Chest discomfort	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
General disorders Fatigue	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Investigations Blood glucose decreased	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Investigations Blood triglycerides increased	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Investigations Blood creatine increased	0.00% 0/40 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	2.6% 1/38 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Injury, poisoning and procedural complications Rib fracture	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Injury, poisoning and procedural complications Contusion	0.00% 0/40 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	2.6% 1/38 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Injury, poisoning and procedural complications Road traffic accident	0.00% 0/40 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	2.6% 1/38 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Musculoskeletal and connective tissue disorders Myalgia	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	2.6% 1/38 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Nervous system disorders Sinus Headache	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Renal and urinary disorders Nephrolithiasia	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Respiratory, thoracic and mediastinal disorders Epistaxis	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)
Respiratory, thoracic and mediastinal disorders Lower Respiratory Tract Congestion	2.5% 1/40 • Number of events 1 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)	0.00% 0/38 • ~11 weeks Number of participants reporting 1 or more adverse events (Affected)