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This study is a Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-103. The approximately 393-days study consists of a Screening Period (28 days), Treatment Period (90 days), and Post-Dose Evaluations (275 days).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adult Cohort 1 (100 mg CNP-103) | Experimental | Three (3) IV administrations of CNP-103 (100 mg) on Days 1, 8, and 90 |
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| Adult Cohort 2 (300 mg CNP-103) | Experimental | Three (3) IV administrations of CNP-103 (300 mg) on Days 1, 8, and 90 |
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| Adult Cohort 3 (600 mg CNP-103) | Experimental | Three (3) IV administrations of CNP-103 (600 mg) on Days 1, 8, and 90 |
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| Adolescent Cohort 1 (100 mg CNP-103) | Experimental | Three (3) IV administrations of CNP-103 (100 mg) on Days 1, 8, and 90 |
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| Adolescent Cohort 2 (300 mg CNP-103) | Experimental | Three (3) IV administrations of CNP-103 (300 mg) on Days 1, 8, and 90 |
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| Adolescent Cohort 3 (600 mg CNP-103) |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CNP-103 | Drug | CNP-103 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety | Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs), MedDRA 28.1 (CTCAE v. 5.0) or current | Day 1 Through Day 365 |
| Immune Safety | Serum Cytokines: IL-1β, TNF-α, IL-6, MCP-1, MIP-1α, IFN-γ, IL-4, IL-10 | Day 1 Through Day 365 |
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Inclusion Criteria:
Participants who are willing and able to provide Institutional Review Board (IRB) approved written informed consent and privacy language as per national regulations.
Men and non-pregnant, non-breast-feeding women ages 12-35 years inclusive.
Documented diagnosis of Stage 3 T1D within 180 days prior to study enrollment according to American Diabetes Association (ADA) criteria.
Participants must be on standard of care diabetes management including insulin therapy as a routine and also consisting of a nutrition plan, regular exercise, or other relevant specialty care as required on a patient-by-patient basis.
Participants with a peak stimulated C-peptide of >0.2 nmol/L measured from a screening mixed meal tolerance test (MMTT).
Participants with an episode of diabetic ketoacidosis (DKA) must have a MMTT performed no sooner than 2 weeks after resolution of the DKA event to have a qualifying C-peptide reading.
Participants on systemic corticosteroids or any medication used to treat the symptoms of T1D (other than insulin) must undergo a washout period of at least two weeks prior to enrollment and must agree to use a non-steroid alternative throughout the trial, if necessary, for any disorder requiring corticosteroids. In addition, participants must be on a stable dose of any other medications, other than insulin, for a minimum of 1 month prior to enrollment and must agree not to increase their dose from the Screening Visit through the End of Study Visit unless reviewed and approved by the medical monitor and the principal investigator.
Female participants of non-childbearing potential (e.g., surgical sterilization, no menses for a year).
Women of childbearing potential (WOCBP) who have agreed not to become pregnant during the study, have a negative pregnancy test at Screening Visit, and agree to use 1 highly effective form of birth control starting at initial screening and continuing throughout the entire study to Day 365.
Female participants who agree to not breastfeed starting at initial Screening and throughout the entire study to Day 365.
Female participants who agree to not donate ova, including autologous, starting at initial Screening and throughout the entire study to Day 365.
Male participant and with a spouse or partner of childbearing potential, who themselves and their spouse or partner agree to practice an effective form of birth control as discussed with the study doctor or study staff starting at Screening and throughout the entire study to Day 365.
Participants must weigh >35 kg at Screening for Cohort 1 (100 mg) and Cohort 2 (300 mg); participants must weigh >50 kg at Screening for Cohort 3 (600 mg).
Body mass index (BMI):
Exclusion Criteria:
Participants unable to comply with prohibited medication outlined in the protocol.
Exclusion of additional immunomodulation will be at the discretion of the Medical Monitor and study site Investigator.
Participants with a history of tuberculosis or positive Quantiferon test.
Participants who received vaccinations in the following time frame:
Known or suspected acute infection, including COVID-19 at the time of Screening or within 2 weeks prior to Screening. After confirmed recent COVID-19 infection, a minimum of 2 weeks of recovery post-acute infection is required.
Participants with Screening laboratory test results that are outside the normal limits and considered by the Investigator to be clinically significant.
Participants with positive test results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV) antigen/antibody as determined at Screening.
Participants with a history of or currently active immune disorders other than T1D (including autoimmune disease) unless the condition, after discussion with the Medical Monitor, has been deemed to be acceptable for the participant's participation in this study.
Participants with a clinical history of significant cardiovascular disease in the past 12 months.
Participants with a complication or medical history of malignant tumor, other than basal cell or squamous cell carcinomas of the skin.
Participants who, in the Investigator's opinion, will be unable to adhere to study visits and procedures.
Participants who have received investigational therapy other than CNP-103 within 28 days or 5 half-lives, whichever is longer, prior to Screening.
Participants with any known active condition which, in the Investigator's opinion, makes the participant unsuitable for study participation.
Known sensitivity to any components of CNP-103.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Stephanie Slaughter | Contact | 317-727-2551 | sslaughter@courpharma.com | |
| Cristina Varela | Contact | 901-517-2602 | cvarela@courpharma.copm |
| Name | Affiliation | Role |
|---|---|---|
| Paul Peloso, MD | COUR Pharma | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Wake Research - Tucson | Recruiting | Tucson | Arizona | 85710 | United States |
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The study will enroll up to three cohorts across two age groups, adults (18-35 years) and adolescents (12-17 years), with six subjects per cohort (approximately 18 subjects per age group, 36 subjects total) at multiple ascending dose levels during the Escalation Phase. Enrollment of each adolescent cohort will be staggered, beginning only after the corresponding adult cohort has completed the Day 15 visit and undergone DMC review. Subjects will be randomized 2:1 to receive either CNP-103 or placebo (0.9% Sodium Chloride Injection, USP) as a 200 mL intravenous infusion on Day 1, Day 8, and Day 90.
The Expansion Phase will follow, enrolling approximately 36 subjects at the safe and tolerated dose(s) identified during the Escalation Phase. Subjects in this phase will be randomized 3:1 to receive either CNP-103 or placebo (0.9% Sodium Chloride Injection, USP).
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| Experimental |
Three (3) IV administrations of CNP-103 (600 mg) on Days 1, 8, and 90 |
|
| Expansion Cohort | Experimental | Dosing for the Expansion Cohort will be determined from Escalation Phase results |
|
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| Placebo | Drug | 0.9% sodium chloride for injection |
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| Liver Institute PLLC | Recruiting | Tucson | Arizona | 85716 | United States |
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| Rady Children's Hospital San Diego | Recruiting | San Diego | California | 92123 | United States |
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| Diablo Clinical Research | Recruiting | Walnut Creek | California | 94598 | United States |
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| Barbara Davis Center for Childhood Diabetes | Recruiting | Aurora | Colorado | 80045 | United States |
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| University of Florida | Recruiting | Gainesville | Florida | 32610 | United States |
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| Evolution Research Center | Recruiting | Hialeah | Florida | 33012 | United States |
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| University of Miami | Recruiting | Miami | Florida | 33136 | United States |
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| DY Professional Research Center | Recruiting | Miami | Florida | 33176 | United States |
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| University of South Florida | Recruiting | Tampa | Florida | 33612 | United States |
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| Atlanta Diabetes Association | Recruiting | Atlanta | Georgia | 30318 | United States |
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| Emory University | Recruiting | Atlanta | Georgia | 30322 | United States |
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| Southern Illinois University School of Medicine | Recruiting | Springfield | Illinois | 62702 | United States |
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| Riley Hospital for Children- Indiana University | Recruiting | Indianapolis | Indiana | 46202 | United States |
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| Barry J. Reiner, MD, LLC | Recruiting | Baltimore | Maryland | 21229 | United States |
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| Joslin Diabetes Center | Recruiting | Boston | Massachusetts | 02215 | United States |
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| Embassy Research Network | Recruiting | Bloomfield Township | Michigan | 48302 | United States |
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| University of Minnesota | Recruiting | Minneapolis | Minnesota | 55454 | United States |
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| Children's Mercy Hospital | Recruiting | Kansas City | Missouri | 64108 | United States |
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| Washington University School of Medicine | Recruiting | St Louis | Missouri | 63110 | United States |
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| UBMD Pediatrics - University of Buffalo | Recruiting | Buffalo | New York | 14203 | United States |
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| MainStreet Health | Recruiting | Syosset | New York | 11791 | United States |
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| Duke University | Recruiting | Durham | North Carolina | 27710 | United States |
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| Physicians East, PA | Recruiting | Greenville | North Carolina | 27834 | United States |
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| Wake Research - Raleigh | Recruiting | Raleigh | North Carolina | 27612 | United States |
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| Superior Clinical Research | Recruiting | Smithfield | North Carolina | 27577 | United States |
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| University Diabetes & Endocrine Consultants | Recruiting | Chattanooga | Tennessee | 37411 | United States |
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| Texas Diabetes & Endocrinology | Recruiting | Austin | Texas | 78749 | United States |
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| M3 Wake Research - Dallas | Recruiting | Dallas | Texas | 75246 | United States |
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| Accurate Clinical Research, Inc | Recruiting | Humble | Texas | 77346 | United States |
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| Diabetes & Glandular Disease (DGD) Clinic, P.A. | Recruiting | San Antonio | Texas | 78229 | United States |
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| TopLine Clinical, Inc | Recruiting | Waxahachie | Texas | 75165 | United States |
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| University of Washington Diabetes Institute | Recruiting | Seattle | Washington | 98109 | United States |
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| ID | Term |
|---|---|
| D003922 | Diabetes Mellitus, Type 1 |
| D003920 | Diabetes Mellitus |
| ID | Term |
|---|---|
| D044882 | Glucose Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D004700 | Endocrine System Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D013812 | Therapeutics |
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