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

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ClinicalTrials.gov Identifier: NCT03746483
Recruitment Status : Completed
First Posted : November 19, 2018
Results First Posted : June 6, 2022
Last Update Posted : June 6, 2022
Sponsor:
Information provided by (Responsible Party):
First Wave Bio, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Exocrine Pancreatic Insufficiency (EPI)
Cystic Fibrosis (CF)
Interventions Drug: MS1819
Drug: Porcine PERT
Enrollment 41
Recruitment Details  
Pre-assignment Details  
Arm/Group Title MS1819 2240 mg/Day (3 Weeks) Then PERT Pre-study Dose (3 Weeks) PERT Pre-study Dose (3 Weeks) Then MS1819 2240 mg/Day (3 Weeks)
Hide Arm/Group Description Participants were first randomized to receive MS1819 first and PERT during the crossover phase Participants were first randomized to receive PERT first and MS1819 during the crossover phase
Period Title: Overall Study
Started 21 20
Completed 15 17
Not Completed 6 3
Reason Not Completed
Adverse Event             0             1
Lack of Efficacy             3             2
Lost to Follow-up             1             0
Protocol Violation             2             0
Arm/Group Title MS1819 - PERT Sequence PERT - MS1819 Sequence Total
Hide Arm/Group Description Participants were first randomized to receive MS1819 first and PERT during the crossover phase Participants were first randomized to receive PERT first and MS1819 during the crossover phase Total of all reporting groups
Overall Number of Baseline Participants 17 19 36
Hide Baseline Analysis Population Description
modified intent-to-treat (mITT)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 19 participants 36 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
17
 100.0%
19
 100.0%
36
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 17 participants 19 participants 36 participants
28.5  (7.60) 29.0  (7.97) 28.8  (7.69)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 19 participants 36 participants
Female
4
  23.5%
11
  57.9%
15
  41.7%
Male
13
  76.5%
8
  42.1%
21
  58.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 19 participants 36 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
16
  94.1%
19
 100.0%
35
  97.2%
More than one race
1
   5.9%
0
   0.0%
1
   2.8%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 17 participants 19 participants 36 participants
United States 13 13 26
Poland 4 6 10
1.Primary Outcome
Title Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)
Hide Description

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.

Time Frame 3 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
modified Intent To Treat (mITT)
Arm/Group Title MS1819 PERT
Hide Arm/Group Description:
Measured while participants on MS1819
Measured while participants on PERT
Overall Number of Participants Analyzed 33 35
Mean (Standard Deviation)
Unit of Measure: % CFA
55.6  (21.44) 86.2  (7.39)
2.Primary Outcome
Title Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)
Hide Description Number of participants reporting 1 or more adverse events
Time Frame 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title MS1819 PERT
Hide Arm/Group Description:
AE reported during treatment with MS1819
AE reported during treatment with PERT
Overall Number of Participants Analyzed 40 38
Measure Type: Count of Participants
Unit of Measure: Participants
Number of subjects with any TEAEs
13
  32.5%
6
  15.8%
Number of subjects with TEAEs related to
2
   5.0%
0
   0.0%
3.Primary Outcome
Title Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
Hide Description Number of Treatment emergent adverse events
Time Frame 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title MS1819 PERT
Hide Arm/Group Description:
AE reported during treatment with MS1819
AE reported during treatment with PERT
Overall Number of Participants Analyzed 40 38
Measure Type: Number
Unit of Measure: Number of TEAEs reported
Number of Treatment-emergent adverse event (TEAEs) 19 8
Number of serious TEAEs reported 0 0
4.Secondary Outcome
Title Stool Weights
Hide Description The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights
Time Frame 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MS1819 PERT
Hide Arm/Group Description:
Measured while participants on MS1819
Measured while participants on PERT
Overall Number of Participants Analyzed 33 35
Mean (Standard Deviation)
Unit of Measure: grams
1394.0  (590.63) 727.3  (334.29)
5.Secondary Outcome
Title Signs and Symptoms of Malabsorption
Hide Description 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.
Time Frame 3 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Data presented at 3 weeks post first dose.
Arm/Group Title MS1819 PERT
Hide Arm/Group Description:
Measured while participants on MS1819
Measured while participants on PERT
Overall Number of Participants Analyzed 36 36
Measure Type: Count of Participants
Unit of Measure: Participants
Grade 0
15
  41.7%
32
  88.9%
Grade 1
12
  33.3%
3
   8.3%
Grade 2
6
  16.7%
1
   2.8%
Grade 3
1
   2.8%
0
   0.0%
Unknown
2
   5.6%
0
   0.0%
6.Secondary Outcome
Title Coefficient of Nitrogen Absorption (CNA)
Hide Description CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet.
Time Frame 3 weeks per group.
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title MS1819 PERT
Hide Arm/Group Description:
Measured while participants on MS1819
Measured while participants on PERT
Overall Number of Participants Analyzed 33 35
Mean (Standard Deviation)
Unit of Measure: % CNA
93.0  (2.54) 97.2  (1.21)
Time Frame ~11 weeks
Adverse Event Reporting Description Number of participants reporting 1 or more adverse events (Affected)
 
Arm/Group Title MS1819 PERT
Hide Arm/Group Description AE reported during treatment with MS1819 AE reported during treatment with PERT
All-Cause Mortality
MS1819 PERT
Affected / at Risk (%) Affected / at Risk (%)
Total   0/40 (0.00%)      0/38 (0.00%)    
Hide Serious Adverse Events
MS1819 PERT
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/40 (0.00%)      0/38 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
MS1819 PERT
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   13/40 (32.50%)      6/38 (15.79%)    
Gastrointestinal disorders     
Constipation  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Nausea  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Oral pain  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Abdominal pain  1  0/40 (0.00%)  0 1/38 (2.63%)  1
General disorders     
Chest discomfort  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Fatigue  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Infections and infestations     
Infective pulmonary exacerbation of cystic fibrosis  1  2/40 (5.00%)  2 1/38 (2.63%)  1
Herpes Zoster  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Sinusitis  1  1/40 (2.50%)  1 1/38 (2.63%)  1
Upper respiratory tract infection  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Vulvovaginal mycotic infection  1  1/40 (2.50%)  1 1/38 (2.63%)  1
Injury, poisoning and procedural complications     
Rib fracture  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Contusion  1  0/40 (0.00%)  0 1/38 (2.63%)  1
Road traffic accident  1  0/40 (0.00%)  0 1/38 (2.63%)  1
Investigations     
Blood glucose decreased  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Blood triglycerides increased  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Blood creatine increased  1  0/40 (0.00%)  0 1/38 (2.63%)  1
Musculoskeletal and connective tissue disorders     
Myalgia  1  1/40 (2.50%)  1 1/38 (2.63%)  1
Nervous system disorders     
Sinus Headache  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Renal and urinary disorders     
Nephrolithiasia  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Epistaxis  1  1/40 (2.50%)  1 0/38 (0.00%)  0
Lower Respiratory Tract Congestion  1  1/40 (2.50%)  1 0/38 (0.00%)  0
1
Term from vocabulary, MedDRA Version 21
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Chief Medical Officer
Organization: First Wave BioPharma Inc.
Phone: (561) 589-7020
EMail: info@firstwavebio.com
Layout table for additonal information
Responsible Party: First Wave Bio, Inc.
ClinicalTrials.gov Identifier: NCT03746483    
Other Study ID Numbers: AZ-CF2001
First Submitted: November 15, 2018
First Posted: November 19, 2018
Results First Submitted: March 4, 2022
Results First Posted: June 6, 2022
Last Update Posted: June 6, 2022