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

• Register: ClinicalTrials.gov
• Last refreshed on: 9 January 2023
• Main ID: NCT02693093
• Date of registration: 12/02/2016
• Prospective Registration: Yes
• Primary sponsor: Kangen Pharmaceuticals, Inc
• Public title: A Dose Ranging Study Evaluating Efficacy and Safety of NI-03 Tactic
• Scientific title: A Phase 1, Single Dose PK and Safety Study With NI-03 Followed by a Phase 2, Randomized, Double-Blind, Parallel-Group Dose-Ranging Study to Evaluate the Safety and Efficacy of NI-03 When Compared to Placebo in Subjects With Chronic Pancreatitis
• Date of first enrolment: February 24, 2016
• Target sample size: 264
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02693093
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

Russian Federation, Ukraine, United States

Contacts

• Name: Phiip Hart, MD
• Affiliation: OSU

Key inclusion & exclusion criteria

Inclusion Criteria:

To be eligible to participate in this study, subjects must meet all of the following
criteria at Screening:

1. Males and females aged 18 to 85 years, inclusive, at the time of consent

2. Ability to communicate effectively with clinic site staff, ability and willingness to
comply with the study schedule, restrictions, and requirements

3. Institutional Review Board (IRB)-approved written informed consent

4. Diagnosis of chronic pancreatitis

5. 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

6. 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.

Exclusion Criteria:

To be eligible to participate in this study, subjects must not meet any of the following
criteria:

1. Any other clinically significant medical condition

2. 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

3. Major abdominal surgery within 90 days of Day 1

4. History or presence of clinically significant cardiovascular disease

5. History of any cancer, except non-melanoma skin cancer, within 5 years of study
enrollment,

6. History of endoscopic intervention within the previous 3 months or presence of a
pancreatic duct stent

7. History of illicit drug abuse (i.e. use of any 'illegal' drugs within 6 months)

8. Active heavy alcohol use (defined as more than 2 alcoholic drinks per day or 14
alcoholic drinks per week)

9. Inadequate venous access

10. Significant blood loss, donation of =450 mL of blood, or blood or blood product
transfusion within 7 days of Day 1

11. History or presence of hepatitis B (surface antigen positivity), active hepatitis C or
human immunodeficiency virus (HIV) antibody

12. Active infection within 30 days of Day 1

13. Pregnant, planning to become pregnant or breast feeding

14. Positive urine or serum pregnancy test result at Screening or on Day 1

15. Active major psychiatric illness requiring a change in treatment within 3 months that
would confound pain assessments

16. History of seizures within the last 12 months

17. Current use of anticonvulsants, antipsychotics, systemic steroids and,
immunosuppressant therapy. *Use of gabapentin, pregabalin and benzodiazepines as
treatment for chronic pancreatitis pain are allowed.

18. Presence of generalized pain syndrome apart from chronic pancreatitis



Age minimum: 18 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Chronic Pancreatitis

Intervention(s)

Drug: NI-03

Drug: Placebo

Primary Outcome(s)

Phase 1 - Safety and Tolerability - Treatment Emergent Adverse Events (TEAE) via CTCAE v4.0 [Time Frame: through 7 days post-dose]

Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA [Time Frame: pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose]

Phase 1 - Safety and Tolerability - Laboratory test results [Time Frame: through 7 days post-dose]

Phase 2 - Efficacy Analysis - average daily worst pain intensity score [Time Frame: 4 Weeks]

Secondary Outcome(s)

Phase 2 - To determine the pharmacokinetic profile for FOY251 and GBA -area under the curve (AUC) [Time Frame: Days 1 and 29, and at 0.25, 0.5, 1, 2 and 4 hours post-dose]

Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA - apparent clearance (CL/F) [Time Frame: 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) [Time Frame: pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.]

Phase 2 - Efficacy Analysis - Change from baseline in current pain score [Time Frame: 4 Weeks]

Phase 2 - Efficacy Analysis - Change from baseline in quality of life [Time Frame: change from baseline to Week 4]

Phase 2 - To determine the pharmacokinetic profile for FOY251 and GBA - plasma terminal half-life (t1/2) [Time Frame: Days 1 and 29, and at 0.25, 0.5, 1, 2 and 4 hours post-dose]

Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA - time to maximum plasma concentration (tmax) [Time Frame: pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.]

Phase 2 - Efficacy Analysis - Change from baseline in average morphine-equivalent daily opioid daily dose [Time Frame: 4 Weeks]

Phase 2 - To determine the pharmacokinetic profile for FOY251 and GBA - Maximum concentration (Cmax) [Time Frame: 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 volume of distribution (Vz/F) [Time Frame: pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.]

Phase 2 - Efficacy Analysis - Change from baseline in average pain score [Time Frame: 4 Weeks]

Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA - plasma terminal half-life (t1/2) [Time Frame: pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.]

Phase 2 - Efficacy Analysis - Change from baseline in least pain score [Time Frame: change from baseline to Week 4]

Phase 1 - To determine the pharmacokinetic profile for FOY251 and GBA -area under the curve (AUC) [Time Frame: pre-dose and at 0.25, 0.5, 1, 2, 4 and 8 hours post-dose.]

Phase 2 - To determine the pharmacokinetic profile for FOY251 and GBA - apparent clearance (CL/F) [Time Frame: Days 1 and 29, and at 0.25, 0.5, 1, 2 and 4 hours post-dose]

Phase 2 - To determine the pharmacokinetic profile for FOY251 and GBA - apparent volume of distribution (Vz/F) [Time Frame: Days 1 and 29, and at 0.25, 0.5, 1, 2 and 4 hours post-dose]

Phase 2 - Safety and Tolerability - Treatment Emergent Adverse Events (TEAE) via CTCAE v4.0 [Time Frame: Through day 57 (End of Study Visit)]

Phase 2 - To determine the pharmacokinetic profile for FOY251 and GBA - time to maximum plasma concentration (tmax) [Time Frame: Days 1 and 29, and at 0.25, 0.5, 1, 2 and 4 hours post-dose]

Phase 2 - Safety and tolerability - Laboratory Test Results [Time Frame: Through day 57 (End of Study Visit)]

Secondary ID(s)

NI03-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors