A Dose Ranging Study Evaluating Efficacy and Safety of NI-03

NCT02693093 | Completed Phase 1

• 1 other identifier: NI03-001
• Study Type: interventional
• Target: 264
• Locations: 3 countries
• Sites: 48

Brief Summary

The purpose of this study is to determine the safety and efficacy of NI-03.

Conditions

Chronic Pancreatitis

Outcome Measures

Primary Outcomes (4)

• Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA

  Pharmacokinetic (PK) parameters such as Maximum concentration (Cmax), time to maximum concentration (Tmax), minimum concentration(Cmin), area under the curve (AUC), half-life (t1/2), apparent clearance (CL/F), and apparent volume of distribution (Vz/F) are assessed.

  pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose

• Phase 1 - Safety and Tolerability - Treatment Emergent Adverse Events (TEAE) via CTCAE v4.0

  Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  through 7 days post-dose

• Phase 1 - Safety and Tolerability - Laboratory test results

  Laboratory test results will be graded and summarized based on CTCAE v4.03. and by shifts in results before and after dosing

  through 7 days post-dose

• Phase 2 - Efficacy Analysis - average daily worst pain intensity score

  4 Weeks

Secondary Outcomes (19)

• Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA -area under the curve (AUC)

  pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.

• Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA - Maximum concentration (Cmax)

  pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.

• Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA - time to maximum plasma concentration (tmax)

  pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.

• Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA - apparent clearance (CL/F)

  pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.

• Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA - plasma terminal half-life (t1/2)

  pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.

Study Arms (4)

placebo

PLACEBO COMPARATOR

TID Day for 28 Days

Drug: Placebo

100 mg NI-03

EXPERIMENTAL

TID Day for 28 Days

Drug: NI-03

200 mg NI-03

EXPERIMENTAL

TID Day for 28 Days

Drug: NI-03

300 mg NI-03

EXPERIMENTAL

TID Day for 28 Days

Drug: NI-03

Interventions

NI-03 DRUG

100 mg NI-03; 200 mg NI-03; 300 mg NI-03

Placebo DRUG

placebo

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• To be eligible to participate in this study, subjects must meet all of the following criteria at Screening:
• Males and females aged 18 to 85 years, inclusive, at the time of consent
• Ability to communicate effectively with clinic site staff, ability and willingness to comply with the study schedule, restrictions, and requirements
• Institutional Review Board (IRB)-approved written informed consent
• Diagnosis of chronic pancreatitis
• Baseline average daily worst pain score must be a minimum of 4 using the Numeric Rating Scale (NRS) during the 7-day run-in period
• Patients on a non-opioid analgesic regimen that is expected to remain stable during the study period, or an opioid regimen with a morphine-equivalent dose not more than 100 mg daily.

You may not qualify if:

• To be eligible to participate in this study, subjects must not meet any of the following criteria:
• Any other clinically significant medical condition
• Treatment with any investigational product within 14 days of Day 1 (or 5 drug half-lives if 5 drug half-lives are expected to exceed 14 days) of Day -7
• Major abdominal surgery within 90 days of Day 1
• History or presence of clinically significant cardiovascular disease
• History of any cancer, except non-melanoma skin cancer, within 5 years of study enrollment,
• History of endoscopic intervention within the previous 3 months or presence of a pancreatic duct stent
• History of illicit drug abuse (i.e. use of any 'illegal' drugs within 6 months)
• Active heavy alcohol use (defined as more than 2 alcoholic drinks per day or 14 alcoholic drinks per week)
• Inadequate venous access
• Significant blood loss, donation of ≥450 mL of blood, or blood or blood product transfusion within 7 days of Day 1
• History or presence of hepatitis B (surface antigen positivity), active hepatitis C or human immunodeficiency virus (HIV) antibody
• Active infection within 30 days of Day 1
• Pregnant, planning to become pregnant or breast feeding
• Positive urine or serum pregnancy test result at Screening or on Day 1

Sponsors & Collaborators

• Kangen Pharmaceuticals, Inc: lead

Study Sites (48)

University of Arkansas

Little Rock, Arkansas, 72204, United States

Location

Kaiser Permanente Medical Group

Los Angeles, California, 90027, United States

Location

University of Southern California, Keck School of Medicine

Los Angeles, California, 90033, United States

Location

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

University of Colorado-Div of Gastroenterology and Hepatology

Aurora, Colorado, 80045, United States

Location

Yale School of Medicine

New Haven, Connecticut, 06519, United States

Location

University of Florida - Division of Gastroenterology

Gainesville, Florida, 32608, United States

Location

Borland-Groover Clinic

Jacksonville, Florida, 32256, United States

Location

Gastroenterology Group of Naples

Naples, Florida, 34102, United States

Location

The Carle Foundation Hospital

Urbana, Illinois, 61801, United States

Location

Indiana University - Indiana University Hospital

Indianapolis, Indiana, 46202-5149, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

UMass Memorial Medical Center

Worcester, Massachusetts, 01605, United States

Location

Clinical Research Institute of Michigan, LLC

Chesterfield, Michigan, 48047, United States

Location

Gastroenterology Associates of Western Michigan

Wyoming, Michigan, 49519, United States

Location

Kansas City Research Institute

Kansas City, Missouri, 64131, United States

Location

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Columbia University School of Medicine

New York, New York, 10027, United States

Location

PMG Research of Winston-Salem

Winston-Salem, North Carolina, 27103, United States

Location

MetroHealth Medical Center

Cleveland, Ohio, 44109, United States

Location

Ohio State University (OSU) - Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

UPMC Presbyterian

Pittsburgh, Pennsylvania, 15213-2536, United States

Location

Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425, United States

Location

Texas Clinical Research Institute

Arlington, Texas, 76012, United States

Location

Texas Tech University Health Sciences Center

El Paso, Texas, 79905, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Virginia Mason

Seattle, Washington, 98101, United States

Location

Wisconsin Center for Advanced Research, a division of GI Associates LLC

Milwaukee, Wisconsin, 53215, United States

Location

Federal Research Centre Institute of Cytology and Genetics

Novosibirsk, 630089, Russia

Location

Military Medical Academu, Dep of Therapy of Adv. Training

Saint Petersburg, 191015, Russia

Location

Military Medical Academy, Department of Hospital Therapy

Saint Petersburg, 191124, Russia

Location

1st Saint Petersburg State Medical University

Saint Petersburg, 197022, Russia

Location

City Hospital #40

Saint Petersburg, 197706, Russia

Location

City Polyclinic #4

Saint Petersburg, 199178, Russia

Location

Medical University "Reaviz"

Samara, 443011, Russia

Location

Tomsk Regional Clinical Hospital

Tomsk, 634063, Russia

Location

Institute of Gastroenterology

Dnipro, 49074, Ukraine

Location

Central city Clinical Hospital, Therapeutic Department #2

Ivano-Frankivsk, 76018, Ukraine

Location

Malaya Therapy National Institute

Kharkiv, 61039, Ukraine

Location

City Policlinic #9

Kharkiv, 61172, Ukraine

Location

City Hospital Named After Tropins, Therapy Department #1

Kherson, 73000, Ukraine

Location

OK Clinic

Kyiv, 02091, Ukraine

Location

Medical Center "Consilium Medical"

Kyiv, 04050, Ukraine

Location

Emergency Care Hospital, #1 Therapeutic Department

Lviv, 79059, Ukraine

Location

Regional Hospital, Department of General Surgery

Odesa, 65025, Ukraine

Location

1st City Clinical Hospital, Therapeutic Department

Poltava, 36038, Ukraine

Location

City Clinical Hospital #1, Gastroenterology Department

Vinnytsia, 21029, Ukraine

Location

Medical Centre Diaservis

Zaporizhia, 69076, Ukraine

Location

Related Publications (2)

• Hart PA, Osypchuk Y, Hovbakh I, Shah RJ, Nieto J, Cote GA, Avgaitis S, Kremzer O, Buxbaum J, Inamdar S, Fass R, Phillips RW, Yadav D, Ladd AM, Al-Assi MT, Gardner T, Conwell DL, Irani S, Sheikh A, Nuttall J; TACTIC Study Investigators. A Randomized Controlled Phase 2 Dose-Finding Trial to Evaluate the Efficacy and Safety of Camostat in the Treatment of Painful Chronic Pancreatitis: The TACTIC Study. Gastroenterology. 2024 Apr;166(4):658-666.e6. doi: 10.1053/j.gastro.2023.12.008. Epub 2023 Dec 15.

  PMID: 38103842 DERIVED

• Ramsey ML, Nuttall J, Hart PA; TACTIC Investigative Team. A phase 1/2 trial to evaluate the pharmacokinetics, safety, and efficacy of NI-03 in patients with chronic pancreatitis: study protocol for a randomized controlled trial on the assessment of camostat treatment in chronic pancreatitis (TACTIC). Trials. 2019 Aug 14;20(1):501. doi: 10.1186/s13063-019-3606-y.

  PMID: 31412955 DERIVED

MeSH Terms

Conditions

Pancreatitis, Chronic

Condition Hierarchy (Ancestors)

Pancreatitis; Pancreatic Diseases; Digestive System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Phiip Hart, MD

  OSU

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 12, 2016
• First Posted: February 26, 2016
• Study Start: February 24, 2016
• Primary Completion: September 30, 2021
• Study Completion: December 30, 2021
• Last Updated: December 16, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (28); RU (8); UA (12)