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

NCT03500627 | Completed Phase 1 FDA Drug

• 1 other identifier: OP-101-002
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Healthy

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Pharmacokinetic (PK) Profile Analysis

  Days 1, 2, 3, 4, 8, and 15.

Study Arms (3)

Cohort 1

EXPERIMENTAL

20 mg/kg OP-101 administered intravenously for over 1 hour.

Drug: OP-101

Cohort 2

EXPERIMENTAL

40 mg/kg OP-101 administered intravenously for over 1 hour.

Drug: OP-101

Cohort 3 (optional)

EXPERIMENTAL

80 mg/kg OP-101 administered intravenously for over 1 hour.

Drug: OP-101

Interventions

OP-101 DRUG

Intravenous Injection of OP-101 in healthy volunteers.

Cohort 1; Cohort 2; Cohort 3 (optional)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Orpheris, Inc.: lead

Study Sites (1)

Medpace Clinical Pharmacology Unit

Cincinnati, Ohio, 45227, United States

Location

MeSH Terms

Conditions

Adrenoleukodystrophy

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Study Officials

• Stephen Huhn, MD

  Orpheris, Inc.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 27, 2018
• First Posted: April 18, 2018
• Study Start: March 30, 2018
• Primary Completion: July 20, 2018
• Study Completion: July 20, 2018
• Last Updated: September 17, 2018

Record last verified: 2018-09

Locations

US (1)