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

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

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

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: OP-101

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45227
      • Medpace Clinical Pharmacology Unit

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Treatment Emergent Adverse Events Graded as Assessed by CTCAE Version 4.0	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.	Screening to Day 15.

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Orpheris, Inc.
• Investigators: Study Chair: Stephen Huhn, MD, Orpheris, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 30, 2018
• Primary Completion (Actual): July 20, 2018
• Study Completion (Actual): July 20, 2018

Study Registration Dates

• First Submitted: March 27, 2018
• First Submitted That Met QC Criteria: April 16, 2018
• First Posted (Actual): April 18, 2018

Study Record Updates

• Last Update Posted (Actual): September 17, 2018
• Last Update Submitted That Met QC Criteria: September 14, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Keywords: Adrenoleukodystrophy (ALD); Childhood Cerebral Adrenoleukodystrophy (ccALD); OP-101; Dendrimer; N-Acetyl-Cysteine (NAC)
• Other Study ID Numbers: OP-101-002

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No