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The primary objectives of this study are to assess the safety and efficacy of MS1819 in enteric capsules vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). The exploratory objective of the extension phase (EP) is to find a dose of MS1819 in immediate release capsules that is safe and results in CFA values in a therapeutic range.
This is a Phase 2, open-label, multicenter, 2, 2x2 crossover study assessing the safety and efficacy of MS1819, 2240 mg/day vs porcine PERT, and 4480 mg/day vs porcine PERT given at the same dose and dosing regimen that was being administered during the pre-study period. MS1819 will be administered in enteric capsules.
MS1819 will be assessed in a 2x2 crossover including approximately 30 patients completing both periods. Fifteen patients will be randomized to the MS1819 2240 mg/day vs PERT arm, and 15 patients will be randomized to the MS1819 4480 mg/day vs PERT arm. Patients in each arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.The coefficient of fat absorption (CFA) will be measured at the end of each 3 week study period. Patients enrolled into the extension phase (EP) will be composed of patients who have completed the crossover phase of OPTION 2.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MS1819 2240 mg/day vs PERT arm, | Active Comparator | Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks. |
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| MS1819 4480 mg/day vs PERT arm | Active Comparator | Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MS1819 | Drug | MS1819 in enteric capsules. It contains is a yeast-derived (non-porcine) lipase pancreatic enzyme replacement. |
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| Measure | Description | Time Frame |
|---|---|---|
| Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events | Number of subjects reporting 1 or more adverse events | Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) |
| Efficacy of MS1819: Coefficient of Fat Absorption (CFA) | The primary efficacy endpoint is the CFA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods. | 6 weeks for crossover phase (to complete both first and second intervention) and 2 weeks for extension phase. |
| Measure | Description | Time Frame |
|---|---|---|
| Stool Weights | The relative efficacy of MS1819 compared to porcine PERT will be assessed using stool weights. | Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) |
| Coefficient of Nitrogen Absorption (CNA) |
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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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Patients in extension arms only included patients who were already part of the initial phase of the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | MS1819 (2240 mg/Day)-PERT Treatment Sequence | Patients were randomized to MS1819 (2240 mg/day) first and then PERT. |
| FG001 | PERT-MS1819 (2240 mg/Day) Sequence | Patients were randomized to PERT first and then MS1819 (2240 mg/day). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Crossover First and Second Interventions |
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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 | Nov 3, 2020 | May 20, 2022 |
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2, 2x2 Crossover
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| Porcine PERT | Drug | Porcine PERT is being used a comparator to MS1819 as a second drug/intervention. |
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CNA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods. CNAs for MS1819 will be compared to the CNAs of PERT using descriptive methods.
| Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) |
| 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 205 | Bialystok | Poland |
| Investigator Site 203 | Karpacz | Poland |
| Investigator Site 206 | Katowice | Poland |
| Investigator Site 202 | Rabka-Zdrój | Poland |
| Investigator Site 204 | Sopot | Poland |
| FG002 | MS1819 (4480 mg/Day)-PERT Treatment Sequence | Patients were first randomized to MS1819 (4480 mg/day) and then PERT. |
| FG003 | PERT-MS1819 (4480 mg/Day) Treatment Sequence | Patients were first randomized to PERT and then MS1819 (4480 mg/day) |
| FG004 | Extension MS1819 IR (4.4 g/Day) | Regardless of previous treatment sequence in study patients received MS1819 IR 4.4 g/day |
| FG005 | Extension MS1819 (6.7 g/Day) | Regardless of previous treatment sequence in study patients received MS1819 IR 6.7 g/day |
| COMPLETED |
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| NOT COMPLETED |
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| Extension Phase |
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Modified intent to treat population.
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| ID | Title | Description |
|---|---|---|
| BG000 | MS1819 (2240 mg/Day)-PERT Treatment Sequence | Patients were randomized to MS1819 (2240 mg/day) first and then PERT. |
| BG001 | PERT-MS1819 (2240 mg/Day) Sequence | Patients were randomized to PERT first and then MS1819 (2240 mg/day). |
| BG002 | MS1819 (4480 mg/Day)-PERT Treatment Sequence | Patients were first randomized to MS1819 (4480 mg/day) and then PERT. |
| BG003 | PERT-MS1819 (4480 mg/Day) Treatment Sequence | Patients were first randomized to PERT and then MS1819 (4480 mg/day) |
| BG004 | 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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| 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 | Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events | Number of subjects reporting 1 or more adverse events | Safety Population | Posted | Count of Participants | Participants | Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) |
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| Primary | Efficacy of MS1819: Coefficient of Fat Absorption (CFA) | The primary efficacy endpoint is the CFA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods. | Modified intent to treat | Posted | Mean | Standard Deviation | percentage of fat absorption | 6 weeks for crossover phase (to complete both first and second intervention) and 2 weeks for extension phase. |
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| Secondary | Stool Weights | The relative efficacy of MS1819 compared to porcine PERT will be assessed using stool weights. | modified intent to treat | Posted | Mean | Standard Deviation | grams | Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) |
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| Secondary | Coefficient of Nitrogen Absorption (CNA) | CNA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods. CNAs for MS1819 will be compared to the CNAs of PERT using descriptive methods. | modified intent to treat | Posted | Mean | Standard Deviation | percentage of nitrogen absorption | Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period) |
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Up to 15 weeks. The maximum overall duration for patients who participated in the crossover phase and extension phase, including screening and end of study visit was 15 weeks. The maximum overall duration for patients who participated in the crossover phase but did not participate in the extension phase, including screening and end of study visit was 11 weeks.
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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 (2240 mg/Day) | Patients on MS1819 in MS1819 (2240 mg/day) - PERT or PERT-MS1819 (2240 mg/day) treatment sequences. | 0 | 14 | 0 | 14 | 1 | 14 |
| EG001 | PERT (MS1819 2240 mg/Day Crossover Group) | Patients on PERT in MS1819 (2240 mg/day) - PERT or PERT-MS1819 (2240 mg/day) treatment sequences. | 0 | 13 | 0 | 13 | 0 | 13 |
| EG002 | MS1819 (4480 mg/Day) | Patients on MS1819 in MS1819 (4480 mg/day) - PERT or PERT-MS1819 (4480 mg/day) treatment sequences. | 0 | 13 | 0 | 13 | 2 | 13 |
| EG003 | PERT (MS1819 4480 mg/Day Crossover Group | Patients on PERT in MS1819 (4480 mg/day) - PERT or PERT-MS1819 (4480 mg/day) treatment sequences. | 0 | 12 | 0 | 12 | 0 | 12 |
| EG004 | Extension MS1819 IR (4.4 g/Day) | Patients enrolled in crossover phase | 0 | 9 | 0 | 9 | 2 | 9 |
| EG005 | Extension MS1819 IR (6.7g/Day) | Patients enrolled in crossover phase | 0 | 8 | 0 | 8 | 2 | 8 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Viral Infection | Infections and infestations | MedDRA (23.0) | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (23.0) | Systematic Assessment |
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| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (23.0) | Systematic Assessment |
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| Pollakiuria | Renal and urinary disorders | MedDRA (23.0) | Systematic Assessment |
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| Urinary hesitation | Renal and urinary disorders | MedDRA (23.0) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (23.0) | Systematic Assessment |
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| Thirst | General disorders | MedDRA (23.0) | Systematic Assessment |
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| Bone Pain | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (23.0) | 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 | May 21, 2021 | May 20, 2022 | SAP_001.pdf |
| ICF | No | No | Yes | Informed Consent Form | Apr 23, 2020 | May 20, 2022 | ICF_002.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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| Between 18 and 65 years |
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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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Patients on PERT in MS1819 (4480 mg/day) - PERT or PERT-MS1819 (4480 mg/day) treatment sequences.
| OG004 | Extension MS1819 IR (4.4 g/Day) | Patients enrolled in crossover phase |
| OG005 | Extension MS1819 IR (6.7g/Day) | Patients enrolled in crossover phase |
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| OG004 |
| Extension MS1819 IR (4.4 g/Day) |
Patients enrolled in crossover phase |
| OG005 | Extension MS1819 IR (6.7g/Day) | Patients enrolled in crossover phase |
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Patients on PERT in MS1819 (4480 mg/day) - PERT or PERT-MS1819 (4480 mg/day) treatment sequences. |
| OG004 | Extension MS1819 IR (4.4 g/Day) | Patients enrolled in crossover phase |
| OG005 | Extension MS1819 IR (6.7g/Day) | Patients enrolled in crossover phase |
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