The AP13CP02 study is a phase 2, open-label, dose escalation trial to determine how safe and tolerable multiple subcutaneous (SC) injections of APL-9796 are for patients with PH. The study will also assess how effective APL-9796 could be for treating patients with PH and whether the body produces antibodies working against APL-9796. The trial will be conducted in two parts: * Part A: Up to 36 adults with WHO Group 1 Pulmonary arterial hypertension (PAH). * Part B (optional): Up to 12 adults with WHO Group 3 - PH associated with ILD (PH-ILD).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
48
3 Cohorts are planned in Part A.
The decision to initiate optional Part B in WHO Group 3 PH-ILD participants will be made by the Safety Review Committee (SRC).
Hammersmith/Imperial Hospital
London, London, United Kingdom
RECRUITINGRoyal United Hospital Bath
Bath, United Kingdom
RECRUITINGRoyal Brompton Hospital
London, United Kingdom
RECRUITINGSheffield Teaching Hospitals
Sheffield, United Kingdom
RECRUITINGIncidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Number of participants who experience Treatment-Emergent Adverse Events will be reported which also include the frequency, severity (CTCAE V5.0) and relationship (causality) of AEs
Time frame: Through study completion (approximately 2 years)
To assess the Telemetry Parameters
Telemetry parameter is assessed using Total pulmonary resistance (TPR)
Time frame: from baseline (CFB) to Day 169
To assess the Telemetry Parameters
Telemetry parameter is assessed using Pulmonary arterial pressures: systolic, diastolic and mean (mPAP)
Time frame: from baseline (CFB) to Day 169
To assess Six minute walk test / Six minute walk distance (6MWD)
A 6-minute walk test (6MWT) is assessed per the guidelines of the American Thoracic Society 2003
Time frame: from baseline (CFB) to Day 169
To assess NT-proB-type Natriuretic Peptide (NT-proBNP)
The level of NT-proBNP is assessed by blood sample collection and analysis
Time frame: from baseline (CFB) to Day 169
To assess the Quality of life
Quality of life is assessed using SF-36 questionnaire
Time frame: from baseline (CFB) to Day 169
To assess the Quality of life
Quality of life is assessed using emPHasis-10 questionnaire
Time frame: from baseline (CFB) to Day 169
To evaluate the PK of APL-9796 in participants with PH
APL-9796 concentrations in serum
Time frame: at each visit (approximately every 28 days until day 169), then day 254, day 339 and day 508
To evaluate the immunogenicity of APL-9796 in participants with PH.
* Incidence of anti-drug antibody (ADA) against APL-9796 (including titres) * Incidence of neutralising antibody (Nab) against APL-9796 (including titres)
Time frame: at each visit (approximately every 28 days until day 169), then day 254, day 339 and day 508
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.