A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers

• ClinicalTrials.gov Identifier: NCT03500627
• Recruitment Status: Completed
• First Posted: April 18, 2018
• Last Update Posted: September 17, 2018
• Sponsor: Orpheris, Inc.
• Information provided by (Responsible Party): Orpheris, Inc.

Study Description

Brief Summary:

A clinical study to measure the effect of OP-101 after being administered intravenously in healthy volunteers.

Condition or disease	Intervention/treatment	Phase
Healthy	Drug: OP-101	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 8 participants
• Allocation: Non-Randomized
• Intervention Model: Sequential Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 1 Open-Label Single-Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 (Dendrimer N-Acetyl-Cysteine) After Intravenous Administration in Healthy Volunteers
• Actual Study Start Date: March 30, 2018
• Actual Primary Completion Date: July 20, 2018
• Actual Study Completion Date: July 20, 2018

Arms and Interventions

Arm	Intervention/treatment
Experimental: Cohort 1; 20 mg/kg OP-101 administered intravenously for over 1 hour.	Drug: OP-101; Intravenous Injection of OP-101 in healthy volunteers.
Experimental: Cohort 2; 40 mg/kg OP-101 administered intravenously for over 1 hour.	Drug: OP-101; Intravenous Injection of OP-101 in healthy volunteers.
Experimental: Cohort 3 (optional); 80 mg/kg OP-101 administered intravenously for over 1 hour.	Drug: OP-101; Intravenous Injection of OP-101 in healthy volunteers.

Outcome Measures

Primary Outcome Measures :

1. Number of Participants with Treatment Emergent Adverse Events Graded as Assessed by CTCAE Version 4.0 [ Time Frame: Screening to Day 15. ]
   Evaluate the safety and tolerability of OP-101 after single IV doses in healthy subjects by monitoring and documenting all adverse events, which include laboratory test variables.

Secondary Outcome Measures :

1. Pharmacokinetic (PK) Profile Analysis [ Time Frame: Days 1, 2, 3, 4, 8, and 15. ]
   Determine the PK profile of OP-101 after single IV doses in healthy subjects as determined by plasma concentrations using the PK Concentration Population.

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 65 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Criteria

Key Inclusion Criteria:

• Body Mass Index (BMI) between 18 and 32 kg/m^2.
• Is in general good health, based upon the results of a medical history assessment, physical examination, vital signs, laboratory profile, and 12-lead electrocardiogram (ECG), as judged by the investigator.
• Female subjects may not be pregnant, lactating, or breastfeeding.
• Subjects must have a negative urine test for drugs of abuse, cotinine, and breath alcohol test at screening and Check-in.

Key Exclusion Criteria:

• Evidence of clinically significant hematologic, renal, endocrine, pulmonary, cardiac, gastrointestinal, hepatic, psychiatric, neurologic, immunologic, allergic disease, or any other condition that, in the opinion of the Investigator, might significantly interfere with the absorption, distribution, metabolism, or excretion of study drug, or place the subject at an unacceptable risk as a participant in this study.
• History of malignancy (other than successfully treated basal cell or squamous cell skin cancer).
• History or presence of an abnormal ECG that, in the opinion of the Investigator, is clinically significant.
• Has used any product containing nicotine within 90 days prior to screening or intends to use any product containing nicotine during the course of the study.
• Has used medications that affect gastrointestinal motility or gastric emptying; such as metoclopramide, proton pump inhibitors, and H2 blockers; within 30 days prior to Day 1.
• Has used prescription or over-the-counter medication, vitamins/herbal supplements (with the exception of hormonal contraceptives) within 14 days prior to Day 1.

Contacts and Locations

Locations

United States, Ohio

Medpace Clinical Pharmacology Unit

Cincinnati, Ohio, United States, 45227

Sponsors and Collaborators

Orpheris, Inc.

Investigators

• Study Chair: Stephen Huhn, MD; Orpheris, Inc.

More Information

• Responsible Party: Orpheris, Inc.
• ClinicalTrials.gov Identifier: NCT03500627
• Other Study ID Numbers: OP-101-002
• First Posted: April 18, 2018
• Last Update Posted: September 17, 2018
• Last Verified: September 2018

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Orpheris, Inc.:

Adrenoleukodystrophy (ALD); Childhood Cerebral Adrenoleukodystrophy (ccALD); OP-101; Dendrimer; N-Acetyl-Cysteine (NAC)