Pulmonary Arterial HypertensionPhase 2NCT04316143
Pharmacokinetics, Safety and Efficacy of BIA 5-1058 in PAH (Zamicastat)
A Phase 2 study testing Oral zamicastat in people with Pulmonary Arterial Hypertension, run by Bial - Portela C S.A. at 13 sites in 7 countries.
Trial summary
This Study evaluates the pharmacokinetic (PK) profile of different zamicastat doses in Pulmonary arterial hypertension (PAH) patients to find the most promising therapeutic dosage range for the treatment of PAH disease Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- Oral zamicastat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 13 sitesin 7 countries
- Study dates
- 3 Jun 2019to 20 Oct 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients aged 18 to 70 years.
- Able to comprehend and willing to sign an informed consent form.
- Diagnosis of PAH (pulmonary arterial hypertension WHO Group 1), documented by right heart catheterisation with a mean pulmonary artery pressure (mPAP) ≥ 25 mmHg, a pulmonary artery wedge pressure (PAWP) ≤ 15 mmHg and a pulmonary vascular resistance (PVR) > 3 wood unit (WU):
- Idiopathic, in non-vasoreactive patients
- Heritable: Bone morphogenetic protein receptor type II (BMPR2) mutation and other mutations, in non-vasoreactive patients
- Drugs and toxin induced, in non-vasoreactive patients
Show all 10Show fewer
- Associated with connective tissue disease
- Associated with simple congenital defects (atrial septal defect and/or ventricular septal defect) if closed > 12 months before inclusion.
- World Health Organization (WHO) functional class II or III as judged by the investigator.
- Stable treatment with at least one of the following approved PAH therapies for at least 90 days prior to V1: Ambrisentan, Bosentan, Macitentan, Riociguat, Selexipag, Sildenafil, Tadalafil, Epoprostenol intravenous, Iloprost inhaled or Treprostinil intravenous or subcutaneous.
Who cannot join
- Contraindication to zamicastat, i.e. known hypersensitivity to ingredients of zamicastat formulation.
- Two or more consecutive measurements of systolic blood pressure (SBP) < 95 mmHg or diastolic blood pressure (DBP) < 50 mmHg.
- Uncontrolled diabetes mellitus with HbA1c ≥ 8.5% within the last three months or at screening.
- PAH WHO Group 1 due to portal hypertension, human immunodeficiency virus (HIV) infection and schistosomiasis.
- Any disease known to cause pulmonary hypertension other than PAH WHO Group 1.
- Obstructive lung disease: Forced Expiratory Volume in 1 second/Forced Vital Capacity (FEV1/FVC) < 60% and FEV1 < 60% of predicted value after bronchodilator administration.
Show all 16Show fewer
- Restrictive lung disease: Total Lung Capacity (TLC) < 70% of predicted value.
- History of moderate to severe hepatic impairment (Child-Pugh B and C).
- Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2 (measured at V1).
- Use of the following prohibited medication or treatments during study participation: calcium channel blockers (CCBs) if used for the treatment of PAH in vasoreactive patients; drugs containing a catechol group that is metabolised by dopamine ß-hydroxylase (DβH) e.g. rimiterole, isoprenaline, dopamine, dopexamine or dobutamide or α- and/or β-blockers.
- Current or previous (within the past year) alcohol or substance abuse excluding caffeine or nicotine.
- Presence of any other significant or progressive/unstable medical condition that, in the opinion of the investigator, would compromise evaluation of the study treatment or may jeopardise the patient's safety, compliance or adherence to protocol requirements.
- For women: Pregnancy or breast-feeding. Women of childbearing potential unable or unwilling to undergo pregnancy tests and practice acceptable contraceptive measures from the time of informed consent until 30 days after last investigational medicinal product (IMP) intake. Acceptable methods for women are surgical intervention (e.g. bilateral tubal occlusion), non-hormonal implantable intrauterine device, double-barrier methods, true sexual abstinence (i.e. when this is in line with the preferred and usual lifestyle of the patient) and vasectomised partner (provided that the partner is the sole sexual partner of the patient and the partner has received medical assessment of the surgical success). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods), hormonal contraceptives and withdrawal are not acceptable methods of contraception.
- For men: Male patients who are sexually active with a partner of childbearing potential must use, with their partner, a condom plus an approved acceptable contraceptive measure from the time of informed consent until 90 days after the last IMP intake. The following methods are acceptable methods of contraception: partner's use of combined (oestrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal); partner's use of progestogen-only hormonal contraception (oral, injectable/implantable, intrauterine hormone-releasing system); partner's use of implantable intrauterine device; surgical sterilisation (for example, vasectomy or bilateral tubal occlusion).
- Previous participation in any other drug investigational study within the past 30 days (or five half-lives of IMP whichever is longer) prior to V1.
- Vulnerable patients according to Section 1.61 of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guideline for Good Clinical Practice E6.
Trial locations and contacts
13 sites in 7 countries.
13 sites in 7 countries
Spain4 sites
Hospital Clinic de Barcelona Calle Villarroel, 170
Barcelona, 08036
Hospital Universitario "12 de Octubre" Avda. de Córdoba, s/n
Madrid, 28041
Complejo Asistencial Universitario de Salamanca Pº. San Vicente, 58
Salamanca, 37007
Hospital Universitario Marques de Valdecilla Avenida Valdecilla, 25
Santander, 39008
Italy3 sites
Ospedale Generale Regionale Miulli-Cardiologia e UTIC Strada Prov. 127 Acquaviva - Santeramo Km. 4,100
Acquaviva delle Fonti, 70021
ASST di Monza-Ospedale San Gerardo -Dipartimento di Pneumologia via Pergolesi 33
Monza, 20900
AOU di Roma-Policlinico Umberto I-Unità Dipartimentale Malattie del Circolo Polmonare Viale del Policlinico 155
Roma, 00161
United Kingdom2 sites
Golden Jubilee National Hospital Golden Jubilee National Hospital Agamemnon St, Scottish Pulmonary Vascular Unit Golden Jubilee National Hospital
Clydebank, G81 4DY
Royal Free Hospital Pond Street
London, NW3 2QG
Austria1 site
Ordensklinikum Linz Elisabethinen, Interne 2 - Kardiologie, Angiologie & Interne Intensivmedizin Fadingerstraße 1
Linz, 4020
Germany1 site
Universitätsklinikum Carl Gustav Carus Dresden, Medizinische Klinik und Poliklinik I, Pneumologie Fetscherstraße 74
Dresden, 01307
Portugal1 site
Centro Hospitalar Lisboa Norte, E.P.E. - Hospital Pulido Valente Consulta Externa de Hipertensão Pulmonar Alameda das Linhas de Torres, 117
Lisbon, 1769-001
Ukraine1 site
State Institution ""National Scientific Centre "M.D. Strazhesko Institute of Cardiology" of NAMS of Ukraine", Department of symptomatic arterial hypertensions Narodnogo opolcheniya 5
Kyiv, 03151
About this study
Official title: An Open-label, Multicentre Study to Evaluate Pharmacokinetics, Safety and Efficacy of Zamicastat as Adjunctive Therapy in Pulmonary Arterial Hypertension (PAH)
This Study evaluates the pharmacokinetic (PK) profile of different zamicastat doses in Pulmonary arterial hypertension (PAH) patients to find the most promising therapeutic dosage range for the treatment of PAH disease
Read the detailed description
This is an open-label, multi-centre study in patients with PAH who are currently on stable treatment with at least one PAH medication. It is planned to evaluate the PK profile (24 hour profile and trough levels) and the safety, tolerability and efficacy of four different zamicastat doses. Each patient will start treatment with the lowest dose (50 mg zamicastat once daily) and the dose will be up-titrated to the individual highest tolerated dose (HTD) i.e. up to 200 mg zamicastat once daily.
A data safety monitoring board (DSMB) will periodically review the safety data and will issue a recommendation if the doses can be used as planned.
This study will consist of:
- A screening period, 5 to 12 days: visit V1
- Up to four dose finding periods, 14 days each:
- Dose A (50 mg zamicastat once daily): visits A1, A2 and A3
- Dose B (100 mg zamicastat once daily): visits B2 and B3
- Dose C (150 mg zamicastat once daily): visits C2 and C3
- Dose D (200 mg zamicastat once daily): visits D2 and D3
- Maintenance period, 42 days: maintenance period visit (MPV)1, MPV2 and MPV3
- Follow-up (FU) period, 14 to 28 days: visits FU (down-titration) and FU
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Oral zamicastat
- Keywords
- PAH, BIA 5-1058, Zamicastat, Bial, Bial - Portela & Ca, S.A., dopamine ß-hydroxylase (DßH) inhibitor, vascular obstruction, pulmonary arteries, right-heart catheterisation (RHC), idiopathic, rare lung disease
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oral zamicastatDrug
Also called BIA 5-1058
Tablets for oral administration under fed conditions containing 100 mg of zamicastat
| Study group | Role | What they receive |
|---|---|---|
| 50 mg zamicastat | Experimental | 50 mg zamicastat once daily Receives: Drug: Oral zamicastat |
| 100 mg zamicastat once daily | Experimental | 100 mg zamicastat once daily Receives: Drug: Oral zamicastat |
| 150 mg zamicastat once daily | Experimental | 150 mg zamicastat once daily Receives: Drug: Oral zamicastat |
| 200 mg zamicastat once daily | Experimental | 200 mg zamicastat once daily Receives: Drug: Oral zamicastat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area Under the Curve 0-24h (AUC0-24h) (ng.h/mL) - 50 mg
This PK parameters (24-hour profile) for zamicastat and its metabolites will be derived after a single dose of 50 mg zamicastat
Measured: Day 1 (0 hours and then 1, 2, 4, 8, 16 and 24 hours after investigational medicinal product (IMP) intake)
Area Under the Curve 0-24h (AUC0-24h) (ng.h/mL/mg) - HTD
This PK parameter (24-hour profile) for zamicastat and its metabolites will be derived at steady-state at the individual highest tolerated dose (HTD)
Measured: 1, 2, 4, 8, 16 and 24 hours after IMP intake
Maximum Plasma Concentration (Cmax) (ng/mL) - 50 mg
This PK parameters (24-hour profile) for zamicastat and its metabolites will be derived after a single dose of 50 mg zamicastat
Measured: Day 1 (0 hours and then 1, 2, 4, 8, 16 and 24 hours after IMP intake)
Maximum Plasma Concentration (Cmax) (ng/mL/mg) - HTD
This PK parameter (24-hour profile) for zamicastat and its metabolites will be derived at steady-state at the individual highest tolerated dose (HTD)
Measured: 1, 2, 4, 8, 16 and 24 hours after IMP intake
Time Until Cmax (Tmax) (h) - 50 mg
This PK parameters (24-hour profile) for zamicastat and its metabolites will be derived after a single dose of 50 mg zamicastat
Measured: Day 1 (0 hours and then 1, 2, 4, 8, 16 and 24 hours after IMP intake)
Time Until Cmax (Tmax) (h) - HTD
This PK parameter (24-hour profile) for zamicastat and its metabolites will be derived at steady-state at the individual highest tolerated dose (HTD)
Measured: 1, 2, 4, 8, 16 and 24 hours after IMP intake
Minimum Plasma Concentration at the End of the Dosing Interval (Cmin,SS) (ng/mL/mg) - HTD
This PK parameter (24-hour profile) for zamicastat and its metabolites will be derived at steady-state at the individual highest tolerated dose (HTD). Following multiple administrations of the HTD of zamicastat to PAH patients at MPV3, PK concentrations and parameter summaries for only the 50 mg, 100 mg and 200 mg dose levels are presented and discussed, as the only subject receiving the 150 mg HTD level had a major protocol deviation documented, impacting the reliability of their PK data.
Measured: 1, 2, 4, 8, 16 and 24 hours after IMP intake
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2020
- Study start3 Jun 2019
- Primary completion20 Oct 2021
- Study completion20 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bial - Portela C S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04316143
- Sponsor's protocol ID
- BIA-51058-201
- Other IDs
- 2018-002448-10, 2018
- Status last verified
- Sep 2024
- First submitted
- 18 Mar 2020
- Last updated
- 15 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multicentre Study to Evaluate Pharmacokinetics, Safety and Efficacy of Zamicastat as Adjunctive Therapy in Pulmonary Arterial Hypertension (PAH)
- ClinicalTrials.gov ID
- NCT04316143
- Acronym / sponsor ID
- BIA-51058-201
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- PAH, BIA 5-1058, Zamicastat, Bial, Bial - Portela & Ca, S.A., dopamine ß-hydroxylase (DßH) inhibitor, vascular obstruction, pulmonary arteries
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Bial - Portela C S.A.Industry (pharma, biotech, devices)
- Study start
- 3 Jun 2019 (actual)
- Primary completion
- 20 Oct 2021 (actual)
- Study completion
- 20 Oct 2021 (actual)
- First posted
- 20 Mar 2020 (actual)
- Results
- Posted 15 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Study evaluates the pharmacokinetic (PK) profile of different zamicastat doses in Pulmonary arterial hypertension (PAH) patients to find the most promising therapeutic dosage range for the treatment of PAH disease
Detailed description
This is an open-label, multi-centre study in patients with PAH who are currently on stable treatment with at least one PAH medication. It is planned to evaluate the PK profile (24 hour profile and trough levels) and the safety, tolerability and efficacy of four different zamicastat doses. Each patient will start treatment with the lowest dose (50 mg zamicastat once daily) and the dose will be up-titrated to the individual highest tolerated dose (HTD) i.e. up to 200 mg zamicastat once daily.
A data safety monitoring board (DSMB) will periodically review the safety data and will issue a recommendation if the doses can be used as planned.
This study will consist of:
- A screening period, 5 to 12 days: visit V1
- Up to four dose finding periods, 14 days each:
- Dose A (50 mg zamicastat once daily): visits A1, A2 and A3
- Dose B (100 mg zamicastat once daily): visits B2 and B3
- Dose C (150 mg zamicastat once daily): visits C2 and C3
- Dose D (200 mg zamicastat once daily): visits D2 and D3
- Maintenance period, 42 days: maintenance period visit (MPV)1, MPV2 and MPV3
- Follow-up (FU) period, 14 to 28 days: visits FU (down-titration) and FU
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 50 mg zamicastat | Experimental | Drug: Oral zamicastat50 mg zamicastat once daily |
| 100 mg zamicastat once daily | Experimental | Drug: Oral zamicastat100 mg zamicastat once daily |
| 150 mg zamicastat once daily | Experimental | Drug: Oral zamicastat150 mg zamicastat once daily |
| 200 mg zamicastat once daily | Experimental | Drug: Oral zamicastat200 mg zamicastat once daily |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male or female patients aged 18 to 70 years.
- Able to comprehend and willing to sign an informed consent form.
- Diagnosis of PAH (pulmonary arterial hypertension WHO Group 1), documented by right heart catheterisation with a mean pulmonary artery pressure (mPAP) ≥ 25 mmHg, a pulmonary artery wedge pressure (PAWP) ≤ 15 mmHg and a pulmonary vascular resistance (PVR) > 3 wood unit (WU):
- World Health Organization (WHO) functional class II or III as judged by the investigator.
- Stable treatment with at least one of the following approved PAH therapies for at least 90 days prior to V1: Ambrisentan, Bosentan, Macitentan, Riociguat, Selexipag, Sildenafil, Tadalafil, Epoprostenol intravenous, Iloprost inhaled or Treprostinil intravenous or subcutaneous.
Key exclusion criteria (16 listed)
- Contraindication to zamicastat, i.e. known hypersensitivity to ingredients of zamicastat formulation.
- Two or more consecutive measurements of systolic blood pressure (SBP) < 95 mmHg or diastolic blood pressure (DBP) < 50 mmHg.
- Uncontrolled diabetes mellitus with HbA1c ≥ 8.5% within the last three months or at screening.
- PAH WHO Group 1 due to portal hypertension, human immunodeficiency virus (HIV) infection and schistosomiasis.
- Any disease known to cause pulmonary hypertension other than PAH WHO Group 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 |
|---|---|---|
| Oral zamicastatAlso: BIA 5-1058Tablets for oral administration under fed conditions containing 100 mg of zamicastat | Drug | 100 mg zamicastat once daily, 150 mg zamicastat once daily, 200 mg zamicastat once daily, 50 mg zamicastat |
Primary outcome
Area Under the Curve 0-24h (AUC0-24h) (ng.h/mL) - 50 mg
Time frame: Day 1 (0 hours and then 1, 2, 4, 8, 16 and 24 hours after investigational medicinal product (IMP) intake)
This PK parameters (24-hour profile) for zamicastat and its metabolites will be derived after a single dose of 50 mg zamicastat
Area Under the Curve 0-24h (AUC0-24h) (ng.h/mL/mg) - HTD
Time frame: 1, 2, 4, 8, 16 and 24 hours after IMP intake
This PK parameter (24-hour profile) for zamicastat and its metabolites will be derived at steady-state at the individual highest tolerated dose (HTD)
Maximum Plasma Concentration (Cmax) (ng/mL) - 50 mg
Time frame: Day 1 (0 hours and then 1, 2, 4, 8, 16 and 24 hours after IMP intake)
This PK parameters (24-hour profile) for zamicastat and its metabolites will be derived after a single dose of 50 mg zamicastat
Maximum Plasma Concentration (Cmax) (ng/mL/mg) - HTD
Time frame: 1, 2, 4, 8, 16 and 24 hours after IMP intake
This PK parameter (24-hour profile) for zamicastat and its metabolites will be derived at steady-state at the individual highest tolerated dose (HTD)
Time Until Cmax (Tmax) (h) - 50 mg
Time frame: Day 1 (0 hours and then 1, 2, 4, 8, 16 and 24 hours after IMP intake)
This PK parameters (24-hour profile) for zamicastat and its metabolites will be derived after a single dose of 50 mg zamicastat
Time Until Cmax (Tmax) (h) - HTD
Time frame: 1, 2, 4, 8, 16 and 24 hours after IMP intake
This PK parameter (24-hour profile) for zamicastat and its metabolites will be derived at steady-state at the individual highest tolerated dose (HTD)
Minimum Plasma Concentration at the End of the Dosing Interval (Cmin,SS) (ng/mL/mg) - HTD
Time frame: 1, 2, 4, 8, 16 and 24 hours after IMP intake
This PK parameter (24-hour profile) for zamicastat and its metabolites will be derived at steady-state at the individual highest tolerated dose (HTD). Following multiple administrations of the HTD of zamicastat to PAH patients at MPV3, PK concentrations and parameter summaries for only the 50 mg, 100 mg and 200 mg dose levels are presented and discussed, as the only subject receiving the 150 mg HTD level had a major protocol deviation documented, impacting the reliability of their PK data.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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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.
13 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Oral zamicastat in people with Pulmonary Arterial Hypertension, run by Bial - Portela C S.A. at 13 sites in 7 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 10 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: finished, as of the sponsor's last update on 15 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Spain, Italy, United Kingdom, Austria, Germany and 2 more countries. 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 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?
Yes. Results are posted in the registry (first posted 15 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Area Under the Curve 0-24h (AUC0-24h) (ng.h/mL) - 50 mg (time frame: Day 1 (0 hours and then 1, 2, 4, 8, 16 and 24 hours after investigational medicinal product (IMP) intake)). The study lists 7 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 15 Oct 2024.