Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ended
Participants planned
462
Countries
14
Sites
46
Pulmonary Arterial Hypertension (PAH)
The primary objective of the study is to establish the long-term safety of AV-101. The long-term effects of AV-101 on efficacy measures (e.g., 6MWD, NT-proBNP, cardiac echo) will also be assessed.
Key facts
- Sponsor
- Aerovate Therapeutics Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Cardiovascular Diseases [C14]
- Trial duration
- 5 May 2023 → 8 Aug 2024
- Decision date (initial)
- 2024-04-08
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Aerovate Therapeutics Inc
External identifiers
- EU CT number
- 2023-508735-31-00
- EudraCT number
- 2021-006864-25
- ClinicalTrials.gov
- NCT05557942
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
The primary objective of the study is to establish the long-term safety of AV-101. The long-term effects of AV-101 on efficacy measures (e.g., 6MWD, NT-proBNP, cardiac echo) will also be assessed.
Conditions and MedDRA coding
Pulmonary Arterial Hypertension (PAH)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | LLT | 10077740 | Pulmonary arterial hypertension WHO functional class II | 10038738 |
| 20.0 | LLT | 10077730 | Pulmonary arterial hypertension WHO functional class IV | 10038738 |
| 21.1 | PT | 10064911 | Pulmonary arterial hypertension | 100000004855 |
| 20.0 | LLT | 10077729 | Pulmonary arterial hypertension WHO functional class III | 10038738 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Long Term Extension Long-term safety
|
Randomised Controlled | Double | [{"id":46366,"code":1,"name":"Subject"},{"id":46367,"code":5,"name":"Carer"},{"id":46368,"code":2,"name":"Investigator"}] | Long Term Extension: Participants will receive the same active doses received on the parent study (AV-101-002) until the optimal dose is selected. |
Regulatory references
- Scientific advice from competent authorities
- European Medicines Agency
| EU CT number | Title | Sponsor |
|---|---|---|
| 2023-508734-34-00 | IMPAHCT: A Phase 2b/3, Randomized, Double-Blind, Placebo-Controlled, 24-Week Dose Ranging and Confirmatory Study to Evaluate the Safety and Efficacy of AV-101 in Patients with Pulmonary Arterial Hypertension (PAH) | Aerovate Therapeutics Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Consented to participate in the LTE and has successfully completed the placebo-controlled 24-week Study AV-101-002.
- Female subjects of childbearing potential who have agreed to continue to use a highly effective form of contraception during the LTE and for at least 30 days after completing or discontinuing study treatment.
Exclusion criteria 2
- The Investigator believes that it would not be in the best interest of the subject to be included in the LTE e.g., for clinical or social reasons.
- Subjects who were not compliant with study medication in AV-101-002 as assessed by the Investigator.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 5
- Safety and tolerability of AV-101
- Change from baseline in the 6MWD
- Change from baseline in NT-proBNP
- Time to Clinical Worsening events
- Change from baseline in Transthoracic Echo parameters of right ventricular (RV) function
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 3
PRD10968960 · Product
- Active substance
- Imatinib
- Pharmaceutical form
- CAPSULE
- Route of administration
- INHALATION
- Max daily dose
- 70 mg milligram(s)
- Max total dose
- 80850 mg milligram(s)
- Max treatment duration
- 165 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- AEROVATE THERAPEUTICS, INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2449
PRD10968961 · Product
- Active substance
- Imatinib
- Pharmaceutical form
- CAPSULE
- Route of administration
- INHALATION
- Max daily dose
- 140 mg milligram(s)
- Max total dose
- 161700 mg milligram(s)
- Max treatment duration
- 165 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- AEROVATE THERAPEUTICS, INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2449
PRD10968959 · Product
- Active substance
- Imatinib
- Pharmaceutical form
- CAPSULE
- Route of administration
- INHALATION
- Max daily dose
- 20 mg milligram(s)
- Max total dose
- 23100 mg milligram(s)
- Max treatment duration
- 165 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- AEROVATE THERAPEUTICS, INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2449
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Aerovate Therapeutics Inc.
- Sponsor organisation
- Aerovate Therapeutics Inc.
- Address
- 200 Berkeley Street Fl 18
- City
- Boston
- Postcode
- 02116-5035
- Country
- United States
Scientific contact point
- Organisation
- Aerovate Therapeutics Inc.
- Contact name
- Medical Monitor
Public contact point
- Organisation
- Aerovate Therapeutics Inc.
- Contact name
- Medical Monitor
Third parties 2
| Organisation | City, country | Duties |
|---|---|---|
| IQVIA RDS Hellas Single Member S.A. ORG-100048380
|
Chalandri, Greece | On site monitoring, Other, Code 8 |
| Iqvia Biotech LLC ORG-100008704
|
Durham, United States | Code 5 |
Locations
14 EU/EEA countries · 46 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ended | 9 | 1 |
| Belgium | Ended | 9 | 2 |
| Czechia | Ended | 9 | 1 |
| France | Ended | 29 | 5 |
| Germany | Ended | 15 | 5 |
| Greece | Ended | 18 | 3 |
| Ireland | Ended | 5 | 1 |
| Italy | Ended | 24 | 7 |
| Latvia | Ended | 12 | 1 |
| Netherlands | Ended | 2 | 1 |
| Poland | Ended | 13 | 4 |
| Portugal | Ended | 13 | 4 |
| Spain | Ended | 34 | 10 |
| Sweden | Ended | 13 | 1 |
| Rest of world
Turkey, China, Mexico, Puerto Rico, Australia, Argentina, Israel, Singapore, United Kingdom, Chile, South Africa, United States, Canada, Colombia, Brazil
|
— | 257 | — |
Investigational sites
Medical University Of Vienna
Department Internal Medicine II, Cardiology, Waehringer Guertel 18-20, Alsergrund, Vienna
UZ Leuven
Pulmonology, Herestraat 49, 3000, Leuven
Hopital Erasme
Cardiology, Lennikse Baan 808, 1070, Anderlecht
Vseobecna Fakultni Nemocnice V Praze
II. interní klinika kardiologie a angiologie, Centrum pro plicní hypertenzi, U Nemocnice 499/2, Nove Mesto, Prague
Assistance Publique Hopitaux De Paris
Pneumology, 78 Rue Du General Leclerc, 94270, Le Kremlin-Bicetre
Hospices Civils De Lyon
Pneumology, 59 Boulevard Pinel, 69500, Bron
Centre Hospitalier Regional De Marseille
Pneumology, 265 Chemin Des Bourrely, 13015, Marseille
Les Hopitaux Universitaires De Strasbourg
Pneumology, 1 Place De L Hopital, Cs 80426, Strasbourg Cedex
Centre Hospitalier Universitaire De Montpellier
Pneumology, 191 Avenue Du Doyen Gaston Giraud, 34295, Montpellier Cedex 5
Muenchen Klinik gGmbH
Klinik für Pneumologie und Pneumologische Onkologie, Englschalkinger Strasse 77, Bogenhausen, Munich
Justus-Liebig-Universitaet Giessen
Standort Gießen, Klinikstrasse 33, 35392, Giessen
Technische Universitaet Dresden
Medizinische Klinik und Poliklinik I, Fetscherstrasse 74, Johannstadt-Nord, Dresden
University Hospital Cologne AöR
Pneumologie, Angiologie und internistische Intensivmedizin, Kerpener Strasse 62, Lindenthal, Cologne
Thoraxklinik Heidelberg gGmbH
Thoraxklinik Heidelberg gGmbH, Roentgenstrasse 1, Rohrbach, Heidelberg
University General Hospital Of Thessaloniki Ahepa
1st Cardiology Clinic, 1st St Kiriakidis Str, 546 36, Thessaloniki
Evangelismos S.A.
1st Intensive Care Clinic, Ipsiladou 45-47, 106 76, Athens
Onassis Cardiac Surgery Center
Hemodynamic Studies and Invasive Cardiology, Leoforos Andrea Siggrou 356, 176 74, Kallithea
Mater Misericordiae University Hospital
Respiratory Department, Eccles Street, D07 R2WY, Dublin 7
Istituto Mediterraneo Per I Trapianti E Terapie Ad Alta Specializzazione S.r.l. I.S.M.E.T.T. S.r.L
Centro per la Cura dell'lpertensione Polmonare, U.O. di Pneumologia, Via Ernesto Tricomi 5, 90127, Palermo
Multimedica S.p.A.
U.O. di Pneumologia, Via San Vittore 12, 20123, Milan
Fondazione IRCCS San Gerardo Dei Tintori
U.O. Pneumologia, Via Giovanni Battista Pergolesi 33, 20900, Monza
Azienda Ospedaliero-Universitaria Policlinico Umberto I
U.O.D. Malattie Circolo Polmonare, Viale Del Policlinico 155, 00161, Rome
Fondazione IRCCS Policlinico San Matteo
Divisione di Cardiologia, Viale Camillo Golgi 19, 27100, Pavia
Azienda Ospedaliero Universitaria Ospedali Riuniti
Cardiologia Universitaria - UTIC -Riabilitazione Cardiologica, Viale Luigi Pinto 1, 71122, Foggia
Azienda Ospedaliera Dei Colli
U.O.C. Cardiologia “Vanvitelli”, Via Leonardo Bianchi, 80131, Naples
Pauls Stradins Clinical University Hospital
Centre of Cardiology, Pilsonu Iela 13, 1002, Riga
Amsterdam UMC Stichting
Oncology / pneumology, De Boelelaan 1117, 1081 HV, Amsterdam
Wojewodzki Specjalistyczny Szpital Im Dr Wl Bieganskiego
Cardiology, Ul. Gen. Karola Kniaziewicza 1/5, 91-347, Lodz
Uniwersytecki Szpital Kliniczny W Poznaniu
Cardiology, Ul. Dluga 1/2, 61-848, Poznan
Krakowski Szpital Specjalistyczny Im. Sw. Jana Pawla II
Cardiology, Ul. Pradnicka 80, 31-202, Cracow
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Cardiology, Ul. Borowska 213, 50-556, Wroclaw
Centro Hospitalar Universitario De Santo Antonio E.P.E.
Cardiology, Largo Professor Abel Salazar, 4050-011, Porto
Hospital Garcia De Orta E.P.E.
Cardiology, Avenida Torrado Da Silva, 2801-951, Almada
Unidade Local De Saude De Coimbra E.P.E.
Cardiology, Praceta Professor Mota Pinto, 3004-561, Coimbra
Centro Hospitalar Universitario De Lisboa Norte E.P.E.
Cardiology, Alameda Das Linhas De Torres No 117, 1769-001, Lisbon
Hospital Clinic De Barcelona
Pulmonology, Calle Villarroel 170, 08036, Barcelona
Hospital Universitario La Paz
Pulmonology Service, Paseo Castellana 261, 28046, Madrid
Hospital Universitario De Toledo
Pulmorany Arterial Hypertension Unit, Avenue Del Rio Guadiana Sn, 45007, Toledo
Hospital Universitario Ramon Y Cajal
Pediatric Cardiology and Congenital heart Disease department, Carretera Del Colmenar Viejo Km 9 100, Por El Pardo, Madrid
El Hospital Universitario De Gran Canaria Dr. Negrin
Respiratoy Medicine, Barranco De La Ballena S N, 35010, Las Palmas De Gran Canaria
Hospital Universitario 12 De Octubre
Cardiology, Bloque D, Avenida De Cordoba Sn, Madrid
Hospital Universitario Virgen De La Victoria
Cardiology, Calle Del Arroyo Teatinos Sn, 29010, Malaga
Hospital Universitari Vall D Hebron
Respiratory Department, Edificio Materno-Infantil, Passeig De La Vall D'hebron 119-129, Barcelona
Hospital Universitario Marques De Valdecilla
Pneumology, Avenida Valdecilla Sn, 39008, Santander
Hospital Universitario De Salamanca
Pulmonology, Paseo De San Vicente 58-182, 37007, Salamanca
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Belgium | 2023-06-02 | 2023-06-06 | 2024-06-18 | ||
| Czechia | 2024-04-10 | 2024-04-17 | 2024-06-18 | ||
| France | 2023-05-17 | 2023-05-17 | 2024-06-18 | ||
| Germany | 2024-02-28 | 2024-03-06 | 2024-06-18 | ||
| Greece | 2024-05-13 | 2024-05-16 | 2024-06-18 | ||
| Italy | 2023-05-05 | 2023-05-24 | 2024-06-18 | ||
| Latvia | 2023-12-15 | 2023-12-28 | 2024-06-18 | ||
| Poland | 2023-07-04 | 2023-07-04 | 2024-06-18 | ||
| Portugal | 2024-01-18 | 2024-01-22 | 2024-06-18 | ||
| Spain | 2023-08-18 | 2023-08-23 | 2024-06-18 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| AV-101-003_Summary of Results_2023-508735-31-00 SUM-46233
|
2024-09-13T23:54:53 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| AV-101-003_Layperson Summary of Results_2023-508735-31-00 | 2024-09-13T23:56:55 | Submitted | Laypersons Summary of Results |
Documents 2 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | AV-101-003_Layperson Summary of Results_2023-508735-31-00 | 1 |
| Summary of results (for publication) | AV-101-003_Summary of Results_2023-508735-31-00 | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-02-10 | Latvia | Acceptable 2024-03-12
|
2024-03-12 |