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The primary objectives of this study are to assess the safety and efficacy of MS1819-SD vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).
This is a Phase 2, open-label, multi-center, 2x2 crossover study assessing the safety and efficacy of MS1819-SD (spray dried) vs porcine PERT given at the same dose that was being administered during the pre-study period.
MS1819-SD will be assessed in a 2x2 crossover including at least 30 patients completing both periods.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks) | Experimental | Patients in arm will further be randomized to receive either the sequence consisting of MS1819 2240 mg/day for 3 weeks followed by PERT for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose. |
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| PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks) | Experimental | Patients in arm will be randomized to receive PERT for 3 weeks followed by MS1819 2240 mg/day for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MS1819 | Drug | MS1819, an oral, non-systemic, that is non-enterically-coated. It is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA) | 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. | 3 weeks |
| Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE) | Number of participants reporting 1 or more adverse events | 6 weeks |
| Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs) | Number of Treatment emergent adverse events | 6 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Stool Weights | The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights | 6 weeks |
| Signs and Symptoms of Malabsorption | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigator Site 105 | Long Beach | California | 90806 | United States | ||
| Investigator Site 102 |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 |
| FG001 | PERT Pre-study Dose (3 Weeks) Then MS1819 2240 mg/Day (3 Weeks) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 30, 2018 | Mar 4, 2022 |
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2x2 Crossover
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Unblinded
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| Porcine PERT | Drug | Porcine PERT is being used as a comparator to MS1819 as a second drug/intervention |
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| 3 weeks |
| Coefficient of Nitrogen Absorption (CNA) | CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet. | 3 weeks per group. |
| Altamonte Springs |
| Florida |
| 32701 |
| United States |
| Investigator Site 107 | Miami | Florida | 33136 | United States |
| Investigator Site 101 | Glenview | Illinois | 60025 | United States |
| Investigator Site 111 | Wichita | Kansas | 67214 | United States |
| Investigator Site 108 | Portland | Maine | 04102 | United States |
| Investigator Site 103 | Las Vegas | Nevada | 89109 | United States |
| Investigator Site 110 | Cleveland | Ohio | 44106 | United States |
| Investigator Site 104 | Toledo | Ohio | 43606 | United States |
| Investigator Site 106 | Hershey | Pennsylvania | 17033 | United States |
| Investigator Site 109 | Dallas | Texas | 75235 | United States |
| Investigator Site 203 | Karpacz | Poland |
| Investigator Site 202 | Rabka-Zdrój | Poland |
| Investigator Site 204 | Sopot | Poland |
Participants were first randomized to receive PERT first and MS1819 during the crossover phase
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| NOT COMPLETED |
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modified intent-to-treat (mITT)
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| ID | Title | Description |
|---|---|---|
| BG000 | MS1819 - PERT Sequence | Participants were first randomized to receive MS1819 first and PERT during the crossover phase |
| BG001 | PERT - MS1819 Sequence | Participants were first randomized to receive PERT first and MS1819 during the crossover phase |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA) | 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. | modified Intent To Treat (mITT) | Posted | Mean | Standard Deviation | % CFA | 3 weeks |
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| Primary | Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE) | Number of participants reporting 1 or more adverse events | Safety Population | Posted | Count of Participants | Participants | 6 weeks |
|
| ||||||||||||||||||||||||||||||
| Primary | Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs) | Number of Treatment emergent adverse events | Safety Population | Posted | Number | Number of TEAEs reported | 6 weeks |
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| Secondary | Stool Weights | The relative efficacy of MS1819-SD compared to porcine PERT will be assessed using stool weights | 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. | Posted | Mean | Standard Deviation | grams | 6 weeks |
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| Secondary | Signs and Symptoms of Malabsorption | 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. | Data presented at 3 weeks post first dose. | Posted | Count of Participants | Participants | 3 weeks |
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| Secondary | Coefficient of Nitrogen Absorption (CNA) | CNA at the end of each treatment period was expressed as the percentage of nitrogen (protein) absorbed from the subjects diet. | 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. | Posted | Mean | Standard Deviation | % CNA | 3 weeks per group. |
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|
~11 weeks
Number of participants reporting 1 or more adverse events (Affected)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | MS1819 | AE reported during treatment with MS1819 | 0 | 40 | 0 | 40 | 13 | 40 |
| EG001 | PERT | AE reported during treatment with PERT | 0 | 38 | 0 | 38 | 6 | 38 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA Version 21 | Systematic Assessment |
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| Herpes Zoster | Infections and infestations | MedDRA Version 21 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA Version 21 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA Version 21 | Systematic Assessment |
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| Vulvovaginal mycotic infection | Infections and infestations | MedDRA Version 21 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA Version 21 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA Version 21 | Systematic Assessment |
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| Oral pain | Gastrointestinal disorders | MedDRA Version 21 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA Version 21 | Systematic Assessment |
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| Chest discomfort | General disorders | MedDRA Version 21 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA Version 21 | Systematic Assessment |
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| Blood glucose decreased | Investigations | MedDRA Version 21 | Systematic Assessment |
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| Blood triglycerides increased | Investigations | MedDRA Version 21 | Systematic Assessment |
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| Blood creatine increased | Investigations | MedDRA Version 21 | Systematic Assessment |
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| Rib fracture | Injury, poisoning and procedural complications | MedDRA Version 21 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA Version 21 | Systematic Assessment |
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| Road traffic accident | Injury, poisoning and procedural complications | MedDRA Version 21 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA Version 21 | Systematic Assessment |
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| Sinus Headache | Nervous system disorders | MedDRA Version 21 | Systematic Assessment |
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| Nephrolithiasia | Renal and urinary disorders | MedDRA Version 21 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21 | Systematic Assessment |
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| Lower Respiratory Tract Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA Version 21 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | First Wave BioPharma Inc. | (561) 589-7020 | info@firstwavebio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 4, 2019 | Mar 4, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D010188 | Exocrine Pancreatic Insufficiency |
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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| >=65 years |
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| Male |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Poland |
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