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Pulmonary Arterial HypertensionPhase 2/3IMPAHCTNCT05036135

A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)

A Phase 2/3 study testing AV-101 in people with Pulmonary Arterial Hypertension, run by Aerovate Therapeutics at 128 sites in 26 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

IMPAHCT: Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial is a Phase 2b/Phase 3 study to evaluate the safety and efficacy of AV-101 (dry powder inhaled imatinib) 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?
AV-101
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
202 participants
Where
128 sitesin 26 countries
Study dates
2 Dec 2021to 9 Aug 2024
Phase
Phase 2/3
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

  • PAH belonging to one of the subgroups:
  • I/HPAH, PAH-CTD,
  • PAH due to drugs and/or toxins/chemicals (having been in the care of the investigator for at least one year with no relapses of drug or toxin/chemical abuse),
  • HIV associated or
  • PAH due to repaired congenital heart disease (at least 1 year since repair)
  • World Health Organization (WHO) Functional Class II, III or IV symptoms
  • Stable concomitant background therapy of at least one PAH approved medications
  • Able to walk a distance of at least 100 m but no more than 475 m during the Screening 6-minute walk tests.

Who cannot join

  • Pulmonary hypertension (PH) belonging to Groups 2 to 5
  • A history of left-sided heart disease
  • Pregnant or breast-feeding females
  • Additional criteria may apply, per protocol

Trial locations and contacts

128 sites in 26 countries.

128 sites in 26 countries

United States39 sites

  • The Unviersity of New Mexico - UNM Hospitals

    Albuquerque, New Mexico, 87131

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Emory University

    Atlanta, Georgia, 30322

  • University of Colorado Denver

    Aurora, Colorado, 80045

  • Tufts Medical Center, Inc.

    Boston, Massachusetts, 02111

  • New York-Presbyterian Brooklyn Methodist Hospital

    Brooklyn, New York, 11215

  • The Lindner Center for Research and Education

    Cincinnati, Ohio, 45219

  • University of Cincinnati

    Cincinnati, Ohio, 45267

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210

  • Baylor University Medical Center

    Dallas, Texas, 75246

  • National Jewish Health

    Denver, Colorado, 80206

  • Duke University Medical Center - Duke South Clinic

    Durham, North Carolina, 27710

  • Houston Methodist Hospital

    Houston, Texas, 77030

  • Indiana University Health

    Indianapolis, Indiana, 46202

  • University of Iowa Hospitals & Clinics

    Iowa City, Iowa, 52242

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • Stat Care Pulmonary Consultants, PLLC

    Knoxville, Tennessee, 37919

  • UCLA/David Geffen School of Medicine-Pulmonary and Criticial Care

    Los Angeles, California, 90024

  • Kaiser Permanente Los Angeles Medical Center

    Los Angeles, California, 90027

  • Cedars-Sinai Medical Center, Pulmonary & Critical Care

    Los Angeles, California, 90048

  • Dept of Veterans Affairs Greater Los Angeles Healthcare System

    Los Angeles, California, 90073

  • University of Minnesota

    Minneapolis, Minnesota, 55455

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • NYU Langone Seaport

    New York, New York, 10016

  • Sentara Medical Group - Sentara Pulmonary & Critical Care Specialists

    Norfolk, Virginia, 23507

  • University of Nebraska Medical Center- Hematology/Oncology Section

    Omaha, Nebraska, 68198

  • Bend Memorial Clinic

    Oregon City, Oregon, 97701

  • Orlando Heart Center

    Orlando, Florida, 32804

  • Hospital of the University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

  • Arizona Pulmonary Specialists, Ltd.

    Phoenix, Arizona, 85012

  • UPMC Presbyterian Hospital

    Pittsburgh, Pennsylvania, 15213

  • UC Davis Medical Center

    Sacramento, California, 95817

  • University of California

    San Francisco, California, 94143

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • LA Biomedical Research Institute Harbor

    Torrance, California, 90509

  • University of Arizona, Department of Medicine

    Tucson, Arizona, 85724

  • George Washington University Medical Faculty Associates, Inc.

    Washington D.C., District of Columbia, 20073

Other trials in United States

Australia9 sites

  • Royal Adelaide Hospital

    Adelaide, 5000

  • Sunshine Coast University Hospital

    Birtinya, Queensland, 4575

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales, 2050

  • The Prince Charles Hospital

    Chermside, Queensland, 4032

  • Concord Repatriation General Hospital

    Concord West, New South Wales, 2138

  • Nepean Hospital

    Kingswood, New South Wales, 2747

  • The Alfred Hospital

    Melbourne, Victoria, 3004

  • Fiona Stanley Hospital

    Murdoch, 6150

  • Westmead Hospital

    Westmead, New South Wales, 2145

Other trials in Australia

China9 sites

  • Peking Union Medical College Hospital

    Beijing, 100730

  • Chongqing Medical University - The First Affiliated Hospital

    Chongqing, 40016

  • Guangdong Provincial People's Hospital

    Guangdong, 510000

  • Central South University - The Second Xiangya Hospital

    Hunan, 410011

  • Yanan Hospital of Kunming City

    Kunming, 650051

  • Gansu Provincial People's Hospital

    Lanzhou, 730000

  • Southeast University (SEU) - Zhongda Hospital

    Nanjing, 210009

  • Shanghai Pulmonary Hospital

    Shanghai, 200437

  • Tianjin Medical University General Hospital

    Tianjin, 300052

Other trials in China

Spain9 sites

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035

  • Hospital Clinic de Barcelona

    Barcelona, 8036

  • Hosp. Universitario Gran Canaria Doctor Negrin

    Las Palmas de Gran Canaria, 35010

  • Hospital 12 de Octubre

    Madrid, 28014

  • Universidad Autonoma de Madrid

    Madrid, 28046

  • Hospital Universitario Virgen de la Victoria

    Málaga, 29071

  • Hospital universtario de Salamanca

    Salamanca, 37007

  • Hosp. Universitario Marques de Valdecilla

    Santander, 39003

  • Hospital Universitario de Toledo

    Toledo, 45007

Other trials in Spain

Argentina7 sites

  • Hospital Britanico de Buenos Aires

    Buenos Aires, 1134

  • Nexo Salud Investigación Clínica

    Buenos Aires, 1425

  • Sanatorio de la Trinidad Mitre

    Buenos Aires, C1039AAO

  • Cardiología Palermo

    CABA, 1425

  • Insituto Medico DAMIC

    Córdoba, 5009

  • Hospital Italiano de Cordoba

    Córdoba, X5004FJE

  • Hospital Dr. Jose Maria Cullen

    Santa Fe, S300EOZ

Other trials in Argentina

Italy7 sites

  • Azienda Ospedaliero Universitaria Ospedali Riuniti - Foggia

    Foggia, 71122

  • Ospedale San Giuseppe - Fatebenefratelli

    Milan, 20123

  • Azienda Socio Sanitaria Territoriale di Monza

    Monza, 20900

  • Azienda Ospedaliera Vincenzo Monaldi

    Naples, 80131

  • Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione

    Palermo, 90127

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100

  • Azienda Ospedaliera Universitaria Policlinico Umberto I

    Rome, 137

Other trials in Italy

France5 sites

  • Hôpital Cardio-Vasculaire et Pneumologique Louis Pradel

    Bron, 69677

  • Groupement Hospitalier Sud - Hôpital Bicêtre

    Le Kremlin-Bicêtre, Cedex, 94275

  • Hôpital Nord - CHU Marseille

    Marseille, 94275

  • Hopital Arnaud de Villeneuve

    Montpellier, 34295

  • Les Hôpitaux Universitaires de Strasbourg

    Strasbourg, 67091

Other trials in France

Canada4 sites

  • The Governors of the University of Calgary

    Calgary, Alberta, T2N 1N4

  • University of Alberta Hospital

    Edmonton, Alberta, T6G 2B7

  • London Health Science Centre

    London, Ontario, N6A 5W9

  • Jewish General Hospital

    Montreal, Quebec, H3T 1E2

Other trials in Canada

Germany4 sites

  • Universitaetsklinikum Carl Gustav Carus TU Dresden

    Dresden, 1307

  • Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen

    Giesen, 35292

  • Universitaetsklinikum Heidelberg

    Heidelberg, 69126

  • München Klinik Bogenhausen

    München, 81925

Other trials in Germany

Mexico4 sites

  • CICUM San Miguel

    Guadalajara, Jalisco, 44160

  • Instituto Nacional de Cardiologia Dr Ignacio Chavez Rivera

    Mexico City, 14080

  • Unidad de Investigacion Clinica en Medicina S.C.

    Monterrey, Nuevo León, 64718

  • Universidad Autonoma de Nuevo Leon, Hospital Universitario

    Monterrey, 64460

Other trials in Mexico

Poland4 sites

  • Krakowski Szpital Specjalistyczny im. Jana Pawla II

    Krakow, 31-202

  • Bieganski Provincial Specialist Hospital

    Lodz, 91-347

  • Szpital Kliniczny Przemienienia Pańskiego UM im. Marcinkowskiego

    Poznan, 61-848

  • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego

    Wroclaw, 50-556

Other trials in Poland

United Kingdom4 sites

  • Golden Jubilee National Hospital

    Glasgow, G81 4DY

  • Imperial College Healthcare NHS Trust

    London, England, W12 0HS

  • Royal Brompton Hospital

    London, SW3 6HP

  • The Newcastle upon Tyne Hospitals NHS Foundation Trust

    Newcastle, NE7 7DN

Other trials in United Kingdom

Greece3 sites

  • University General Hospital Attikon

    Athens, 10676

  • Cardiosurgery Hospital

    Kallithea, 17674

  • AHEPA General Hospital of Thessaloniki

    Thessaloniki, 54637

Other trials in Greece

Israel3 sites

  • The Lady Davis Carmel Medical Center

    Haifa, 34362

  • Hadassah University Hospital - Ein Kerem

    Jerusalem, 91999

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 64239

Other trials in Israel

Portugal3 sites

  • Hospital Garcia de Orta, EPE

    Almada, 2805-267

  • Hospital Pulido Valente

    Lisbon, 1649-035

  • CHUPorto

    Porto, 4099-001

Other trials in Portugal

Belgium2 sites

  • Hopital Erasme - Cliniques Universitaires de Bruxelles

    Brussels, 1070

  • UZ Leuven

    Leuven, 3000

Other trials in Belgium

Brazil2 sites

  • Santa Casa de Misericordia de Porto Alegre

    Porto Alegre, 90035-074

  • Heart Institute - University of Sao Paulo

    São Paulo, 05403

Other trials in Brazil

Singapore2 sites

  • National University Hospital

    Singapore, 119074

  • Tan Tock Seng Hospital

    Singapore, 308433

Other trials in Singapore

Austria1 site

  • AKH - Medizinische Universitat Wien

    Vienna, 1090

Other trials in Austria

Chile1 site

  • Centro de Investigaciones Clinicas de la Universidad Catolica

    Santiago, 8330024

Other trials in Chile

Czechia1 site

  • Vseobecna fakultni nemocnice v Praze

    Prague, 128 21

Other trials in Czechia

Latvia1 site

  • Pauls Stradins Clinical University Hospital

    Riga, 1002

Other trials in Latvia

Netherlands1 site

  • Amsterdam UMC, Locatie VUMC

    Amsterdam, 1081 HV

Other trials in Netherlands

Puerto Rico1 site

  • Cardiopulmonary Research Center

    Guaynabo, 00968

Other trials in Puerto Rico

South Africa1 site

  • Center of Chest Disease Johannesburg

    Johannesburg, 2193

Other trials in South Africa

Sweden1 site

  • Norrlands Universitetssjukhus

    Umeå, 907 37

Other trials in Sweden

About this study

Official title: 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)

IMPAHCT: Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial is a Phase 2b/Phase 3 study to evaluate the safety and efficacy of AV-101 (dry powder inhaled imatinib) in patients with Pulmonary Arterial Hypertension (PAH). The Phase 2b part of the study will assess three doses to establish an optimal dose for the Phase 3 part of the study. The Phase 2b primary endpoint will be the placebo corrected change in pulmonary vascular resistance (PVR). The Phase 3 primary endpoint will be the placebo corrected change in 6-minute walk distance (6MWD) after 24 weeks of treatment.

Being tested
AV-101, Placebo
Keywords
Pulmonary Arterial Hypertension, Lungs, Pulmonary, PAH, AV-101, imatinib, IMPAHCT
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.

  • AV-101Drug

    AV-101 (imatinib) administered via dry powder inhalation

  • PlaceboDrug

    Placebo administered via dry powder inhalation

Study groups in NCT05036135
Study groupRoleWhat they receive
AV-101 10 mgExperimentalNot described
Receives: Drug: AV-101
AV-101 35 mgExperimentalNot described
Receives: Drug: AV-101
AV-101 70 mgExperimentalNot described
Receives: Drug: AV-101
PlaceboPlacebo comparatorNot described
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase 2b: Change from Baseline in Pulmonary Vascular Resistance (PVR)

    Measured: 24 weeks

  2. Phase 3: Change from Baseline in Six Minute Walk Distance (6MWD)

    Measured: 24 weeks

Secondary outcomes (11)

  1. Phase 2b: Change from Baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP)

    Measured: 24 weeks

  2. Phase 2b: Change from Baseline in Six Minute Walk Distance (6MWD)

    Measured: 24 weeks

  3. Phase 2b: Time to Clinical Worsening

    Measured: 24 weeks

  4. Phase 2b: Proportion of Subjects with Improvement in WHO Functional Class

    Measured: 24 weeks

  5. Phase 2b: Change from Baseline in Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Risk Score

    REVEAL Lite 2.0 risk scores range from 1 to 14; a higher score represents higher risk

    Measured: 24 weeks

  6. Phase 2b: Change from Baseline in Health-Related Quality of Life emPHasis-10 Questionnaire Score

    emPHasis-10 questionnaire scores range from 0 - 50; a higher score represents a higher symptom burden

    Measured: 24 weeks

  7. Phase 3: Change from Baseline in NT-proBNP

    Measured: 24 weeks

  8. Phase 3: Time to Clinical Worsening

    Measured: 24 weeks

  9. Phase 3: Proportion of Subjects with Improvement in WHO Functional Class

    Measured: 24 weeks

  10. Phase 3: Change from Baseline in Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Risk Score

    REVEAL Lite 2.0 risk scores range from 1 to 14; a higher score represents higher risk

    Measured: 24 weeks

  11. Phase 3: Change from Baseline in Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire Score

    PAH-SYMPACT is a patient reported outcome instrument developed to assess PAH symptoms and impacts

    Measured: 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Sep 2021
  2. Study start2 Dec 2021
  3. Primary completion9 Aug 2024
  4. Study completion9 Aug 2024

Why it stopped: The IMPAHCT study demonstrated that while inhaled imatinib was well tolerated, it did not prove to be efficacious at any of the doses of AV-101 evaluated in the study.

Resources

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 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
202 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05036135
Sponsor's protocol ID
AV-101-002
Acronym
IMPAHCT
Status last verified
Aug 2024
First submitted
16 Aug 2021
Last updated
23 Aug 2024
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Hill NS, Gillies H, Chakinala MM, Feldman JP, Hoeper MM, Jing ZC, Mclaughlin VV, Rosenkranz S, Escribano-Subias P, Ghio S, Gomberg-Maitland M, Habib N, Kopec G, Langleben D, Lescano A, Ramirez A, Rischard F, Sitbon O, Skride A, Dake BT, Khindri S, Langley J, Niven R, Zhang X, Humbert M. Phase 2b trial of inhaled imatinib for treatment of pulmonary arterial hypertension. Am J Respir Crit Care Med. 2026 Apr 1;212(4):802-812. doi: 10.1093/ajrccm/aamaf128.

    PubMed 41738079

  • Gillies H, Chakinala MM, Dake BT, Feldman JP, Hoeper MM, Humbert M, Jing ZC, Langley J, McLaughlin VV, Niven RW, Rosenkranz S, Zhang X, Hill NS. IMPAHCT: A randomized phase 2b/3 study of inhaled imatinib for pulmonary arterial hypertension. Pulm Circ. 2024 Mar 25;14(1):e12352. doi: 10.1002/pul2.12352. eCollection 2024 Jan.

    PubMed 38532768

  • Gillies H, Niven R, Dake BT, Chakinala MM, Feldman JP, Hill NS, Hoeper MM, Humbert M, McLaughlin VV, Kankam M. AV-101, a novel inhaled dry-powder formulation of imatinib, in healthy adult participants: a phase 1 single and multiple ascending dose study. ERJ Open Res. 2023 Mar 13;9(2):00433-2022. doi: 10.1183/23120541.00433-2022. eCollection 2023 Mar.

    PubMed 36923571

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing AV-101 in people with Pulmonary Arterial Hypertension, run by Aerovate Therapeutics at 128 sites in 26 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 8 inclusion and 4 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 23 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 128 sites in United States, Australia, China, Spain, Argentina and 21 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. 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 combined 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.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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: The IMPAHCT study demonstrated that while inhaled imatinib was well tolerated, it did not prove to be efficacious at any of the doses of AV-101 evaluated in the study.). 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?

Phase 2b: Change from Baseline in Pulmonary Vascular Resistance (PVR) (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05036135, which also lists 3 linked publications.

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 23 Aug 2024.

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