Pulmonary Arterial HypertensionPhase 2NCT03924154
A Study of RVT-1201 in Patients With Pulmonary Arterial Hypertension (ELEVATE 1)
A Phase 2 study testing RVT-1201 in people with Pulmonary Arterial Hypertension, run by Altavant Sciences GmbH at 23 sites in 2 countries.
Trial summary
This is an exploratory Phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of RVT-1201 in patients with pulmonary arterial hypertension (PAH). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- RVT-1201
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 3 participants
- Where
- 23 sitesin 2 countries
- Study dates
- 1 Aug 2019to 24 Feb 2020
- Phase
- Phase 2
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Symptomatic PAH belonging to one of the following types:
- Idiopathic
- Heritable
- Drug- or toxin- induced
- Associated with one of the following: connective tissue disease or congenital heart disease
- World Health Organization (WHO) Functional Class (FC) II or III
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- PAH diagnosed by right heart cardiac catheterization prior to Screening
- Receiving standard of care treatment for PAH with oral monotherapy or dual therapy for at least 12 weeks prior to Screening at a dose which has been stable for at least 8 weeks prior to Screening
- If on a diuretic, dose must be stable for at least 4 weeks prior to Screening, with no changes anticipated during study participation
- 6-Minute Walk Distance (6MWD) between 150 and 500 meters at Screening and Baseline visits
- Plasma N-terminal pro B-type natriuretic peptide (NT-proBNP) level ≥ 300 pg/mL at Screening
- Ability and willingness to give written informed consent and to comply with the requirements of the study
Who cannot join
- PAH associated with human immunodeficiency virus (HIV) infection, portal hypertension or schistosomiasis
- Other types of pulmonary hypertension (PH):
- Pulmonary hypertension due to left heart disease (WHO PH Group 2)
- Pulmonary hypertension due to lung diseases and/or hypoxia (WHO PH Group 3)
- Chronic thromboembolic pulmonary hypertension (WHO PH Group 4)
- Pulmonary hypertension with unclear multifactorial mechanisms (WHO PH Group 5)
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- Hospitalization for pulmonary hypertension within 12 weeks of screening
- Cardiopulmonary rehabilitation program based on exercise (planned, or started ≤ 12 weeks prior to Screening)
- Prostanoid or prostacyclin receptor agonist therapy within 12 weeks of screening
- Evidence of left-sided heart disease
- If Pulmonary function tests were done prior to screening, Pulmonary function tests demonstrate obstructive or restrictive lung disease
- Use of telotristat (Xermelo®) within the last 6 months
- Use of any investigational drug within 30 days or five half-lives (whichever is longer) prior to Screening, or 90 days if an investigational drug for PAH
- Have uncontrolled atrial fibrillation (AFib) or other uncontrolled arrhythmias
- Body mass index (BMI) >45 kg/m2
- Women of childbearing potential who are pregnant, planning to become pregnant, or lactating or female/male patients unwilling to use effective contraception
Trial locations and contacts
23 sites in 2 countries.
23 sites in 2 countries
United States20 sites
University of Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
University of Chicago Medical Center
Chicago, Illinois, 60637
Cleveland Clinic
Cleveland, Ohio, 44195
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Duke University Medical Center
Durham, North Carolina, 27710
Pulmonary Research Institute of Southeast Michigan
Farmington Hills, Michigan, 48336
University of Florida
Gainesville, Florida, 32610
University of Texas Health Science Center at Houston, McGovern Medical School
Houston, Texas, 77030
Kentuckiana Pulmonary Research Center
Louisville, Kentucky, 40202
San Marcus Research Clinic, Inc.
Miami Lakes, Florida, 33014
Louisiana State University Health Sciences Center
New Orleans, Louisiana, 70112
Central Florida Pulmonary Group, P.A.
Orlando, Florida, 32803
Pulmonary Associates, PA
Phoenix, Arizona, 85006
Oregon Health & Science University
Portland, Oregon, 97239
University of California Davis Medical Center
Sacramento, California, 95817
SBPA Research LLC
Santa Barbara, California, 93105
Baystate Medical Center
Springfield, Massachusetts, 01199
Washington University School of Medicine
St Louis, Missouri, 63110
George Washington Medical Faculty Associates - Pulmonary Hypertension Program
Washington D.C., District of Columbia, 20037
Canada3 sites
University of Calgary
Calgary, Alberta, T1Y6J4
University of Alberta
Edmonton, Alberta, T6G 2B7
London Health Sciences Centre
London, Ontario, N6A 5W9
About this study
Official title: A Phase 2a, Double-Blind, Placebo-Controlled Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of RVT-1201 in Patients With Pulmonary Arterial Hypertension
This is an exploratory Phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of RVT-1201 in patients with pulmonary arterial hypertension (PAH).
Read the detailed description
This is an exploratory Phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of RVT-1201 in patients with pulmonary arterial hypertension (PAH).
Study participation for each patient will last approximately 3 months and will consist of a screening period (up to 28 days in duration), a baseline period (day 1, pre-dose), a 6-week treatment period, and a 2-week follow-up period.
The study will enroll approximately 36 patients at approximately 20 centers across the United States and Canada.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- RVT-1201, Placebo
- Keywords
- Pulmonary arterial hypertension (PAH), Pulmonary hypertension (PH), RVT-1201, Rodatristat ethyl, Tryptophan hydroxylase (TPH), Serotonin reduction, ELEVATE 1, KAR5417
- Medical subject headings
- Pulmonary Arterial Hypertension, Hypertension, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RVT-1201Drug
Also called rodatristat ethyl
RVT-1201 600 mg immediate-release tablet
PlaceboDrug
Also called Placebo (for RVT-1201)
Inactive pill manufactured to mimic RVT-1201 600 mg immediate-release tablet
| Study group | Role | What they receive |
|---|---|---|
| RVT-1201 | Experimental | RVT-1201 600 mg immediate-release tablet, administered orally twice daily with food for 6 weeks, in addition to the patient's current standard of care medication(s) for PAH (n=24 [Anticipated]) Receives: Drug: RVT-1201 |
| Placebo | Placebo comparator | Matching placebo tablet, administered orally twice daily with food for 6 weeks, in addition to the patient's current standard of care medication(s) for PAH (n=12 [Anticipated]) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events (AEs) and discontinuations due to AEs
Incidence of treatment-emergent adverse events (TEAEs), drug-related adverse events (AEs), and discontinuations due to AEs
Measured: 8 weeks
Secondary outcomes (6)
Concentration of biomarkers of serotonin biosynthesis in plasma
Absolute concentrations and percent change from baseline in plasma 5-hydroxyindoleacetic acid (5-HIAA) and plasma 5-hydroxytryptamine (5-HT, also known as serotonin) concentrations
Measured: 8 weeks
Concentration of biomarkers of serotonin biosynthesis in urine
Concentration of urine 5-hydroxyindoleacetic acid (5-HIAA) will be normalized against urine creatinine concentration to determine absolute ratio and percent change from baseline in urine 5-HIAA:creatinine ratio
Measured: 8 weeks
Study drug (RVT-1201) and active metabolite (KAR5417) plasma concentrations
Measured RVT-1201 and KAR5417 plasma concentrations from sparse sampling
Measured: 6 weeks
Area under the plasma concentration versus time curve (AUC) of KAR5417 (the active metabolite of RVT-1201)
Measured KAR5417 plasma concentrations from sparse sampling will be used to assess the pharmacokinetic (PK) parameter AUC of KAR5417 administered twice daily in patients with PAH, by means of population PK (PopPK) analysis
Measured: 6 weeks
Relationship between KAR5417 exposure and percent change from baseline in plasma concentrations of the serotonin-related biomarkers
Evaluate the relationship between exposure (area under the plasma concentration versus time curve [AUC]) of KAR5417 (the active metabolite of RVT-1201) and percent change from baseline in plasma concentrations of the serotonin-related biomarkers (5-HIAA and 5-HT)
Measured: 6 weeks
Relationship between KAR5417 exposure and percent change from baseline in urine concentrations of the serotonin-related biomarkers
Evaluate the relationship between exposure (area under the plasma concentration versus time curve [AUC]) of KAR5417 (the active metabolite of RVT-1201) and percent change from baseline in urine 5-HIAA:creatinine concentration ratio
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2019
- Study start1 Aug 2019
- Primary completion24 Feb 2020
- Study completion24 Feb 2020
Why it stopped: Inability to enroll
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial HypertensionStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Following screening assessments, PAH patients who meet all entrance criteria will be randomly assigned to receive one of the following treatments in a ratio of 2:1: * Arm 1 (n=24) - RVT-1201 Treatment: RVT-1201 immediate-release tablets will be administered orally, at a dose of 600 mg twice daily (BID), for a total of 6 weeks in addition to the patient's current standard of care (SOC) medication(s) for PAH. * Arm 2 (n=12) - Placebo Treatment: Matching placebo tablets will be administered orally, at a dose of 600 mg twice daily (BID), for a total of 6 weeks in addition to the patient's current SOC medication(s) for PAH. Participants will be followed in face-to-face visits with trial personnel every 2 weeks for 8 weeks (6 weeks of treatment plus a 2-week follow-up), with an additional phone call at Week 1, to assess drug effects and monitor safety during their treatments.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Altavant Sciences GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PPD Development, LP
- Responsible party
- Sponsor
- Investigators
- Ed Parsley, DO, Study director, Altavant Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03924154
- Sponsor's protocol ID
- RVT-1201-2001
- Status last verified
- Aug 2019
- First submitted
- 5 Apr 2019
- Last updated
- 9 Mar 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Double-Blind, Placebo-Controlled Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of RVT-1201 in Patients With Pulmonary Arterial Hypertension
- ClinicalTrials.gov ID
- NCT03924154
- Acronym / sponsor ID
- RVT-1201-2001
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- Pulmonary arterial hypertension (PAH), Pulmonary hypertension (PH), RVT-1201, Rodatristat ethyl, Tryptophan hydroxylase (TPH), Serotonin reduction, ELEVATE 1, KAR5417
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Inability to enroll
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Altavant Sciences GmbHIndustry (pharma, biotech, devices)
- Collaborators
- PPD Development, LP
- Study start
- 1 Aug 2019 (actual)
- Primary completion
- 24 Feb 2020 (actual)
- Study completion
- 24 Feb 2020 (actual)
- First posted
- 23 Apr 2019 (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.
This is an exploratory Phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of RVT-1201 in patients with pulmonary arterial hypertension (PAH).
Detailed description
This is an exploratory Phase 2a, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of RVT-1201 in patients with pulmonary arterial hypertension (PAH).
Study participation for each patient will last approximately 3 months and will consist of a screening period (up to 28 days in duration), a baseline period (day 1, pre-dose), a 6-week treatment period, and a 2-week follow-up period.
The study will enroll approximately 36 patients at approximately 20 centers across the United States and Canada.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Following screening assessments, PAH patients who meet all entrance criteria will be randomly assigned to receive one of the following treatments in a ratio of 2:1: * Arm 1 (n=24) - RVT-1201 Treatment: RVT-1201 immediate-release tablets will be administered orally, at a dose of 600 mg twice daily (BID), for a total of 6 weeks in addition to the patient's current standard of care (SOC) medication(s) for PAH. * Arm 2 (n=12) - Placebo Treatment: Matching placebo tablets will be administered orally, at a dose of 600 mg twice daily (BID), for a total of 6 weeks in addition to the patient's current SOC medication(s) for PAH. Participants will be followed in face-to-face visits with trial personnel every 2 weeks for 8 weeks (6 weeks of treatment plus a 2-week follow-up), with an additional phone call at Week 1, to assess drug effects and monitor safety during their treatments.
| Arm | Type | Receives |
|---|---|---|
| RVT-1201 | Experimental | Drug: RVT-1201RVT-1201 600 mg immediate-release tablet, administered orally twice daily with food for 6 weeks, in addition to the patient's current standard of care medication(s) for PAH (n=24 [Anticipated]) |
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo tablet, administered orally twice daily with food for 6 weeks, in addition to the patient's current standard of care medication(s) for PAH (n=12 [Anticipated]) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Symptomatic PAH belonging to one of the following types:
- World Health Organization (WHO) Functional Class (FC) II or III
- PAH diagnosed by right heart cardiac catheterization prior to Screening
- Receiving standard of care treatment for PAH with oral monotherapy or dual therapy for at least 12 weeks prior to Screening at a dose which has been stable for at least 8 weeks prior to Screening
- If on a diuretic, dose must be stable for at least 4 weeks prior to Screening, with no changes anticipated during study participation
Key exclusion criteria (16 listed)
- PAH associated with human immunodeficiency virus (HIV) infection, portal hypertension or schistosomiasis
- Other types of pulmonary hypertension (PH):
- Hospitalization for pulmonary hypertension within 12 weeks of screening
- Cardiopulmonary rehabilitation program based on exercise (planned, or started ≤ 12 weeks prior to Screening)
- Prostanoid or prostacyclin receptor agonist therapy within 12 weeks of screening
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 |
|---|---|---|
| RVT-1201Also: rodatristat ethylRVT-1201 600 mg immediate-release tablet | Drug | RVT-1201 |
| PlaceboAlso: Placebo (for RVT-1201)Inactive pill manufactured to mimic RVT-1201 600 mg immediate-release tablet | Drug | Placebo |
Primary outcome
Adverse events (AEs) and discontinuations due to AEs
Time frame: 8 weeks
Incidence of treatment-emergent adverse events (TEAEs), drug-related adverse events (AEs), and discontinuations due to AEs
6 secondary outcomes
Concentration of biomarkers of serotonin biosynthesis in plasma
Time frame: 8 weeks
Concentration of biomarkers of serotonin biosynthesis in urine
Time frame: 8 weeks
Study drug (RVT-1201) and active metabolite (KAR5417) plasma concentrations
Time frame: 6 weeks
Area under the plasma concentration versus time curve (AUC) of KAR5417 (the active metabolite of RVT-1201)
Time frame: 6 weeks
Relationship between KAR5417 exposure and percent change from baseline in plasma concentrations of the serotonin-related biomarkers
Time frame: 6 weeks
Relationship between KAR5417 exposure and percent change from baseline in urine concentrations of the serotonin-related biomarkers
Time frame: 6 weeks
Results and safety
The study was stopped early (reason given: Inability to enroll). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2020.
- Individual participant dataSharing planned: No
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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.
Ed Parsley, DO study director
Altavant Sciences
23 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RVT-1201 in people with Pulmonary Arterial Hypertension, run by Altavant Sciences GmbH at 23 sites in 2 countries.
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 12 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: stopped early, as of the sponsor's last update on 9 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, Canada. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 stopped early (reason given: Inability to enroll). Results are not available in the source record.
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?
Adverse events (AEs) and discontinuations due to AEs (time frame: 8 weeks). 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 9 Mar 2020.