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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05036135




Registration number
NCT05036135
Ethics application status
Date submitted
16/08/2021
Date registered
5/09/2021
Date last updated
21/06/2024

Titles & IDs
Public title
A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)
Scientific 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)
Secondary ID [1] 0 0
AV-101-002
Universal Trial Number (UTN)
Trial acronym
IMPAHCT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Arterial Hypertension 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AV-101
Treatment: Drugs - Placebo

Experimental: Phase 2b low dose AV-101 -

Experimental: Phase 2b medium dose AV-101 -

Experimental: Phase 2b high dose AV-101 -

Placebo comparator: Phase 2b Placebo -

Experimental: Phase 3 dose AV-101 (Optimal dose selected in Phase 2b) -

Placebo comparator: Phase 3 Placebo -


Treatment: Drugs: AV-101
AV-101 (imatinib) administered via dry powder inhalation

Treatment: Drugs: Placebo
Placebo administered via dry powder inhalation
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase 2b: Change from Baseline in Pulmonary Vascular Resistance (PVR)
Timepoint [1] 0 0
24 weeks
Primary outcome [2] 0 0
Phase 3: Change from Baseline in Six Minute Walk Distance (6MWD)
Timepoint [2] 0 0
24 weeks
Secondary outcome [1] 0 0
Phase 2b: Change from Baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP)
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Phase 2b: Change from Baseline in Six Minute Walk Distance (6MWD)
Timepoint [2] 0 0
24 weeks
Secondary outcome [3] 0 0
Phase 2b: Time to Clinical Worsening
Timepoint [3] 0 0
24 weeks
Secondary outcome [4] 0 0
Phase 2b: Proportion of Subjects with Improvement in WHO Functional Class
Timepoint [4] 0 0
24 weeks
Secondary outcome [5] 0 0
Phase 2b: Change from Baseline in Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Risk Score
Timepoint [5] 0 0
24 weeks
Secondary outcome [6] 0 0
Phase 2b: Change from Baseline in Health-Related Quality of Life emPHasis-10 Questionnaire Score
Timepoint [6] 0 0
24 weeks
Secondary outcome [7] 0 0
Phase 3: Change from Baseline in NT-proBNP
Timepoint [7] 0 0
24 weeks
Secondary outcome [8] 0 0
Phase 3: Time to Clinical Worsening
Timepoint [8] 0 0
24 weeks
Secondary outcome [9] 0 0
Phase 3: Proportion of Subjects with Improvement in WHO Functional Class
Timepoint [9] 0 0
24 weeks
Secondary outcome [10] 0 0
Phase 3: Change from Baseline in Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Risk Score
Timepoint [10] 0 0
24 weeks
Secondary outcome [11] 0 0
Phase 3: Change from Baseline in Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire Score
Timepoint [11] 0 0
24 weeks

Eligibility
Key inclusion criteria
Key Inclusion Criteria

* PAH belonging to one of the subgroups:

1. I/HPAH, PAH-CTD,
2. 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),
3. HIV associated or
4. 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.

Key
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/12/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/01/2025
Actual
Sample size
Target
462
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Concord Repatriation General Hospital - Concord West
Recruitment hospital [3] 0 0
Nepean Hospital - Kingswood
Recruitment hospital [4] 0 0
Westmead Hospital - Westmead
Recruitment hospital [5] 0 0
Sunshine Coast University Hospital - Birtinya
Recruitment hospital [6] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [7] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [8] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [9] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2138 - Concord West
Recruitment postcode(s) [3] 0 0
2747 - Kingswood
Recruitment postcode(s) [4] 0 0
2145 - Westmead
Recruitment postcode(s) [5] 0 0
4575 - Birtinya
Recruitment postcode(s) [6] 0 0
4032 - Chermside
Recruitment postcode(s) [7] 0 0
3004 - Melbourne
Recruitment postcode(s) [8] 0 0
5000 - Adelaide
Recruitment postcode(s) [9] 0 0
6150 - Murdoch
Recruitment outside Australia
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Arizona
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Kansas
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Newcastle

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Aerovate Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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). 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.
Trial website
https://clinicaltrials.gov/study/NCT05036135
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Toll Free Number
Address 0 0
Country 0 0
Phone 0 0
(888) 373-8110
Fax 0 0
Email 0 0
clinicaltrials@aerovatetx.com
Contact person for scientific queries