HealthyPhase 1NCT03500627
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers
A Phase 1 study testing OP-101 in people with Healthy, run by Orpheris, Inc. at 1 site in United States.
Trial summary
A clinical study to measure the effect of OP-101 after being administered intravenously in healthy volunteers. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy
- What is being tested?
- OP-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Mar 2018to 20 Jul 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Medpace Clinical Pharmacology Unit
Cincinnati, Ohio, 45227
About this study
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
A clinical study to measure the effect of OP-101 after being administered intravenously in healthy volunteers.
- Conditions
- Healthy
- Being tested
- OP-101
- Keywords
- Adrenoleukodystrophy (ALD), Childhood Cerebral Adrenoleukodystrophy (ccALD), OP-101, Dendrimer, N-Acetyl-Cysteine (NAC)
- Medical subject headings
- Adrenoleukodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OP-101Drug
Intravenous Injection of OP-101 in healthy volunteers.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 20 mg/kg OP-101 administered intravenously for over 1 hour. Receives: Drug: OP-101 |
| Cohort 2 | Experimental | 40 mg/kg OP-101 administered intravenously for over 1 hour. Receives: Drug: OP-101 |
| Cohort 3 (optional) | Experimental | 80 mg/kg OP-101 administered intravenously for over 1 hour. Receives: Drug: OP-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Screening to Day 15.
Secondary outcomes (1)
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.
Measured: Days 1, 2, 3, 4, 8, and 15.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2018
- Study start30 Mar 2018
- Primary completion20 Jul 2018
- Study completion20 Jul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HealthyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Orpheris, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stephen Huhn, MD, Study chair, Orpheris, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03500627
- Sponsor's protocol ID
- OP-101-002
- Status last verified
- Sep 2018
- First submitted
- 27 Mar 2018
- Last updated
- 17 Sep 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03500627
- Acronym / sponsor ID
- OP-101-002
- Conditions
- Healthy
- Keywords
- Adrenoleukodystrophy (ALD), Childhood Cerebral Adrenoleukodystrophy (ccALD), OP-101, Dendrimer, N-Acetyl-Cysteine (NAC)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Orpheris, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Mar 2018 (actual)
- Primary completion
- 20 Jul 2018 (actual)
- Study completion
- 20 Jul 2018 (actual)
- First posted
- 18 Apr 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A clinical study to measure the effect of OP-101 after being administered intravenously in healthy volunteers.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: OP-10120 mg/kg OP-101 administered intravenously for over 1 hour. |
| Cohort 2 | Experimental | Drug: OP-10140 mg/kg OP-101 administered intravenously for over 1 hour. |
| Cohort 3 (optional) | Experimental | Drug: OP-10180 mg/kg OP-101 administered intravenously for over 1 hour. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OP-101Intravenous Injection of OP-101 in healthy volunteers. | Drug | Cohort 1, Cohort 2, Cohort 3 (optional) |
Primary outcome
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.
1 secondary outcome
Pharmacokinetic (PK) Profile Analysis
Time frame: Days 1, 2, 3, 4, 8, and 15.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stephen Huhn, MD study chair
Orpheris, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing OP-101 in people with Healthy, run by Orpheris, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Treatment Emergent Adverse Events Graded as Assessed by CTCAE Version 4.0 (time frame: Screening to Day 15.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2018; AzTrials last refreshed its copy on 6 Nov 2026.