Clinical trial NCT06649110: A Study to Learn About the Treatment LTP001 in Healthy Participants (Part A) and in Participants With PAH (Part B)
Official title: A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of LTP001 in Healthy Adult Participants (Part A) and to Evaluate the Efficacy and Safety of LTP001 for the Treatment of Participants With Pulmonary Arterial Hypertension (Part B)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Healthy Volunteers, Pulmonary Arterial Hypertension
Keywords: LTP001
pulmonary arterial hypertension
PAH
PVR
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-10-18
Enrollment: 232
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A study to learn about the treatment LTP001 in healthy participants (Part A) and in participants with PAH (Part B)

Detailed description: The CLTP001A12202 study will explore the safety, tolerability, and pharmacokinetics of LTP001 in healthy volunteers (Part A) and will evaluate the safety and efficacy (Part B) followed by safety extension in participants with pulmonary arterial hypertension.

Eligibility criteria: Part A Inclusion Criteria:

* Healthy males and non-child-bearing potential females

Part A Exclusion Criteria:

* Clinically significant ECG or cardiac abnormalities, any surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of drugs, or which may jeopardize the participant in the study Other protocol-defined inclusion/exclusion criteria may apply.

Part B Inclusion Criteria:

-Confirmed diagnosis of PAH, pre-randomization PVR ≥400 dyn.sec.cm-5, treatment with stable doses of standard-of-care PAH therapies, 6-minute walk distance ≥ 150 m and ≤450 m.

Part B Exclusion Criteria:

Any surgical or medical condition which may place the participant at higher risk from his/her participation in the study Women of child-bearing potential unless they are using highly effective methods of contraception Sexually active males unwilling to use a condom during intercourse while taking study treatment and for 24 hours after stopping study treatment.

History of hypersensitivity to any of the study treatments or excipients

Other protocol-defined inclusion / exclusion criteria may apply

Primary outcomes:
measure: Part A- Number of participants with Adverse events (AEs) and Serious Adverse events (SAEs)
timeFrame: Baseline to Day 35
measure: Part B-Treatment Period 1: Change in pulmonary vascular resistance (PVR)
timeFrame: Baseline to Week 24
measure: Part B-Treatment Period 2: Number of participants with Adverse events (AEs) and Serious Adverse events (SAEs)
timeFrame: From Day 1 until Week 106
Secondary outcomes:
measure: Part A- Maximum observed plasma concentrations (Cmax)
timeFrame: Baseline to Day 35
measure: Part A- Time to reach maximum plasma concentration (Tmax)
timeFrame: Baseline to Day 35
measure: Part A- Area under plasma concentration-time curve from time zero to the last measurable concentration sampling time (AUClast)
timeFrame: Baseline to Day 35
measure: Part A- Area under the plasma concentration-time curve from time zero extrapolated to infinity (AUC[0-inf])
timeFrame: Baseline to Day 35
measure: Part A- Terminal elimination half-life (T1/2)
timeFrame: Baseline to Day 35
measure: Part B-Treatment Period 1: Change in the Six-minute Walk Test (6MWT)
timeFrame: Baseline to Week 24
measure: Part B-Treatment Period 1: Change in N-terminal pro-brain natriuretic peptide (NT-proBNP)
timeFrame: Baseline to Week 24
measure: Part B-Treatment Period 1: Change in World Health Organization (WHO) functional class (FC)
timeFrame: Baseline to Week 24
measure: Part B-Treatment Period 2: Change in the Six-minute Walk Test (6MWT)
timeFrame: Baseline to Month 18
measure: Part B-Treatment Period 2: Change in World Health Organization (WHO) functional class (FC)
timeFrame: Baseline to Month 18
Other outcomes:
Locations:
city: Austin
country: United States
facility: PPD Development LP
state: Texas
Study officials:
References: