PAHPhase 1NCT02149095
Dose Escalation, MTD, Safety and PK Study of a Single Dose SC Injection of TransCon PEG Treprostinil in Healthy Male Volunteers
A Phase 1 study testing TransCon PEG treprostinil in people with PAH, run by United Therapeutics at 1 site in United States.
Trial summary
TransCon PEG treprostinil is a novel prodrug form of treprostinil, in which treprostinil is reversibly conjugated via a four-arm branched polyethylene glycol (PEG) molecule. Reversible coupling of treprostinil to PEG should allow for a modified extended pharmacokinetic profile to achieve sustained plasma… Read more about this study
- Who is it for?
- Ages 18 to 50 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- PAH
- What is being tested?
- TransCon PEG treprostinil
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2014to Nov 2014
- 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 50 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Subject gives voluntary written informed consent to participate in the study
- Subject is a healthy male between the ages of 18 and 50 years, inclusive, at Screening
- Subjects must weigh between 60 and 120 kg, inclusive, with a BMI between 19.0-32.0 kg/m2, inclusive at Screening
- Subject has a medical history, physical examination, vital signs, ECG and clinical laboratory results within normal limits or considered not clinically significant by the Investigator at Screening
- Subject agrees to abstain from taking any prescription medication for 14 days prior to check-in and to abstain from taking any non-prescription medications (except multivitamins) or herbal supplements for 7 days prior to check-in Baseline until discharge from the study (unless prescribed by the Investigator to treat an AE)
- Subject agrees to abstain from consuming alcohol from three days prior to check-in and until discharge from the study
- Subject agrees to refrain from strenuous exercise from check-in and until discharge from the study
- Subject is able to communicate effectively with study personnel and be considered reliable, willing and cooperative in terms of compliance with the protocol requirements
Who cannot join
- Subject has any clinically relevant abnormality identified during the screening physical examination, 12-lead ECG, or laboratory examinations
- Subject has a history of anaphylaxis, a previous documented hypersensitivity reaction, or a clinically significant idiosyncratic reaction to any drug
- Subject has a clinically significant history of neurological, cardiovascular, respiratory, endocrine, hematological, hepatic, renal, gastrointestinal, genitourinary, pulmonary, and/or musculoskeletal disease; glaucoma; a psychiatric disorder, or any other chronic disease, whether controlled by medication or not
- Subject has a history of postural hypotension, or unexplained syncope
- Subject has a blood pressure that is less than 85 mmHg systolic or 50 mmHg diastolic at Screening or Baseline
- Subject has a pulse rate that is greater than 90 bpm after sitting at rest for 5 minutes at Screening or Baseline
Show all 16Show fewer
- Subject has a history of hypertension
- Subject has a blood pressure that is greater than 150 mmHg systolic or 90 mmHg diastolic at Screening or Baseline
- Subject has a predisposing condition that could interfere with the absorption, distribution, metabolism, or excretion of drugs
- Subject has tested positive at the screening visit for HIV infection, HBsAg, or the HCV antibody
- Subject currently uses tobacco products or has a history of tobacco use within six months prior to Baseline
- Subject has a history of alcohol abuse or a history of or current impairment of organ function reasonably related to alcohol abuse
- Subject has a history of or current evidence of abuse of licit or illicit drugs or a positive urine screen for drugs of abuse
- Subject has a history of abnormal bleeding tendencies
- Subject has donated blood or plasma or has lost a significant volume of blood (greater than 450 mL) within four weeks prior to Baseline
- Subject has participated in any investigational drug study within 30 days prior to Screening
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I, Open-label, Dose Escalation Study to Determine the Maximum Tolerated Dose and Evaluate the Safety, Tolerability and Pharmacokinetics of Single Doses of TransCon PEG Treprostinil Administered as a Subcutaneous Injection in Healthy Adult Male Volunteers
TransCon PEG treprostinil is a novel prodrug form of treprostinil, in which treprostinil is reversibly conjugated via a four-arm branched polyethylene glycol (PEG) molecule. Reversible coupling of treprostinil to PEG should allow for a modified extended pharmacokinetic profile to achieve sustained plasma concentrations of treprostinil.
This will be the first investigation of TransCon PEG treprostinil in humans. This study aims to determine the maximum tolerated dose (MTD) and assess the safety, tolerability and pharmacokinetics of escalating single doses of a subcutaneous injection of TransCon PEG treprostinil.
- Conditions
- PAH
- Being tested
- TransCon PEG treprostinil
- Keywords
- treprostinil
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TransCon PEG treprostinilDrug
| Study group | Role | What they receive |
|---|---|---|
| TransCon PEG treprostinil | Experimental | Dosing will begin at 0.116 mg/kg TransCon PEG treprostinil subcutaneous injection and the dose escalated in subsequent cohorts to MTD. Receives: Drug: TransCon PEG treprostinil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent AEs
Measured: Day 43
Maximum tolerated dose
Measured: 5 days
Treatment-emergent changes in clinical laboratory results
Measured: 43 days
Treatment-emergent changes in vital signs
Measured: 43 days
Secondary outcomes (3)
Area under the concentration versus time curve: (AUC)
Measured: 42 days
Maximum observed plasma concentration: Cmax
Measured: 42 days
Time to maximum observed plasma concentration: Tmax
Measured: 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 May 2014 (estimated)
- Study startJul 2014
- Primary completionNov 2014 (estimated)
Why it stopped: Study will not be conducted
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PAHStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- United Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02149095
- Sponsor's protocol ID
- TCP-PH-101
- Status last verified
- Aug 2014
- First submitted
- 20 May 2014
- Last updated
- 7 Aug 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-label, Dose Escalation Study to Determine the Maximum Tolerated Dose and Evaluate the Safety, Tolerability and Pharmacokinetics of Single Doses of TransCon PEG Treprostinil Administered as a Subcutaneous Injection in Healthy Adult Male Volunteers
- ClinicalTrials.gov ID
- NCT02149095
- Acronym / sponsor ID
- TCP-PH-101
- Conditions
- PAH
- Keywords
- treprostinil
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study will not be conducted
- Enrolment
- 0 participants (actual)
- Lead sponsor
- United TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Jul 2014
- Primary completion
- Nov 2014 (estimated)
- First posted
- 29 May 2014 (estimated)
- 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.
TransCon PEG treprostinil is a novel prodrug form of treprostinil, in which treprostinil is reversibly conjugated via a four-arm branched polyethylene glycol (PEG) molecule. Reversible coupling of treprostinil to PEG should allow for a modified extended pharmacokinetic profile to achieve sustained plasma concentrations of treprostinil.
This will be the first investigation of TransCon PEG treprostinil in humans. This study aims to determine the maximum tolerated dose (MTD) and assess the safety, tolerability and pharmacokinetics of escalating single doses of a subcutaneous injection of TransCon PEG treprostinil.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TransCon PEG treprostinil | Experimental | Drug: TransCon PEG treprostinilDosing will begin at 0.116 mg/kg TransCon PEG treprostinil subcutaneous injection and the dose escalated in subsequent cohorts to MTD. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Subject gives voluntary written informed consent to participate in the study
- Subject is a healthy male between the ages of 18 and 50 years, inclusive, at Screening
- Subjects must weigh between 60 and 120 kg, inclusive, with a BMI between 19.0-32.0 kg/m2, inclusive at Screening
- Subject has a medical history, physical examination, vital signs, ECG and clinical laboratory results within normal limits or considered not clinically significant by the Investigator at Screening
- Subject agrees to abstain from taking any prescription medication for 14 days prior to check-in and to abstain from taking any non-prescription medications (except multivitamins) or herbal supplements for 7 days prior to check-in Baseline until discharge from the study (unless prescribed by the Investigator to treat an AE)
Key exclusion criteria (16 listed)
- Subject has any clinically relevant abnormality identified during the screening physical examination, 12-lead ECG, or laboratory examinations
- Subject has a history of anaphylaxis, a previous documented hypersensitivity reaction, or a clinically significant idiosyncratic reaction to any drug
- Subject has a clinically significant history of neurological, cardiovascular, respiratory, endocrine, hematological, hepatic, renal, gastrointestinal, genitourinary, pulmonary, and/or musculoskeletal disease; glaucoma; a psychiatric disorder, or any other chronic disease, whether controlled by medication or not
- Subject has a history of postural hypotension, or unexplained syncope
- Subject has a blood pressure that is less than 85 mmHg systolic or 50 mmHg diastolic at Screening or Baseline
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 |
|---|---|---|
| TransCon PEG treprostinil | Drug | TransCon PEG treprostinil |
Primary outcome
Incidence of treatment-emergent AEs
Time frame: Day 43
Maximum tolerated dose
Time frame: 5 days
Treatment-emergent changes in clinical laboratory results
Time frame: 43 days
Treatment-emergent changes in vital signs
Time frame: 43 days
3 secondary outcomes
Area under the concentration versus time curve: (AUC)
Time frame: 42 days
Maximum observed plasma concentration: Cmax
Time frame: 42 days
Time to maximum observed plasma concentration: Tmax
Time frame: 42 days
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2014.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 TransCon PEG treprostinil in people with PAH, run by United Therapeutics 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 8 inclusion and 16 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: withdrawn, as of the sponsor's last update on 7 Aug 2014. 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 was withdrawn before enrolling participants, so there are no results.
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?
Incidence of treatment-emergent AEs (time frame: Day 43). The study lists 4 primary outcomes in all. 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 7 Aug 2014.