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NCT03496623: PERFECT

A Phase 3 Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Participants With Pulmonary Hypertension (PH) Due to Chronic Obstructive Pulmonary Disease (COPD)

Terminated Phase 3 Results posted Last updated 24 November 2023
What this trial tests

Phase 3 trial testing Inhaled treprostinil solution in Pulmonary Hypertension in 188 participants. Terminated before completion.

Timeline
8 May 2018
Primary endpoint
13 October 2022
13 October 2022

Quick facts

Lead sponsorUnited Therapeutics
PhasePhase 3
StatusTerminated
Study typeINTERVENTIONAL
Allocationrandomized
Designcrossover
Maskingquadruple
Primary purposetreatment
Enrollment188
Start date8 May 2018
Primary completion13 October 2022
Estimated completion13 October 2022
Sites76 locations across Argentina, Puerto Rico, United States, Israel

Drugs / interventions tested

Conditions studied

Sponsor

United Therapeutics — full company profile →

Who can join

18 and older, any sex, with Pulmonary Hypertension or Chronic Obstructive Pulmonary Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change From Baseline to Week 12 in 6-Minute Walk Distance (6MWD) Primary · Baseline, Week 12

6 MWD was calculated at peak exposure (10 to 60 minutes after dosing). 6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course for 6 minutes (timed) and the distance walked (in meters) was recorded. Statistical analyses were not performed due to lack of appropriate sample size.

Baseline
GroupValue95% CI
Original Crossover Design - Treprostinil225.07± 77.41
Original Crossover Design - Placebo220.47± 77.64
Contingent Parallel Design - Treprostinil231.50± 60.77
Contingent Parallel Design - Placebo283.00± 67.78
Change at Week 12
GroupValue95% CI
Original Crossover Design - Treprostinil-4.47± 39.01
Original Crossover Design - Placebo-5.14± 50.71
Contingent Parallel Design - Treprostinil20.33± 116.99
Contingent Parallel Design - Placebo18.5± 42.49

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 6 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Run-In
Serious: 9/108 (8%)
Deaths: 1/108
Treprostinil
Serious: 17/66 (26%)
Deaths: 5/66
Placebo
Serious: 6/58 (10%)
Deaths: 0/58

Serious adverse events (41 terms)

ReactionSystemRun-InTreprostinilPlacebo
Chronic obstructive pulmonary diseaseRespiratory, thoracic and mediastinal disorders
Acute respiratory failureRespiratory, thoracic and mediastinal disorders
Acute myocardial infarctionCardiac disorders
AnaemiaBlood and lymphatic system disorders
Acute coronary syndromeCardiac disorders
Cardiac arrestCardiac disorders
Cardiac failure congestiveCardiac disorders
Right ventricular dysfunctionCardiac disorders
Right ventricular failureCardiac disorders
Atrial fibrillationCardiac disorders
Cardiac failure acuteCardiac disorders
Cardiogenic shockCardiac disorders
Pulseless electrical activityCardiac disorders
Gastric ulcerGastrointestinal disorders
Large intestine polypGastrointestinal disorders
Ischaemic hepatitisHepatobiliary disorders
Appendicitis perforatedInfections and infestations
COVID-19Infections and infestations
COVID-19 pneumoniaInfections and infestations
PneumoniaInfections and infestations
BronchitisInfections and infestations
Pneumonia aspirationInfections and infestations
Staphylococcal bacteraemiaInfections and infestations
FallInjury, poisoning and procedural complications
Head injuryInjury, poisoning and procedural complications
Other adverse events (13 terms — click to expand)

ReactionSystemRun-InTreprostinilPlacebo
DyspnoeaRespiratory, thoracic and mediastinal disorders
CoughRespiratory, thoracic and mediastinal disorders
HeadacheNervous system disorders
Oropharyngeal painRespiratory, thoracic and mediastinal disorders
FatigueGeneral disorders
Chest discomfortGeneral disorders
Productive coughRespiratory, thoracic and mediastinal disorders
Upper respiratory tract infectionInfections and infestations
DizzinessNervous system disorders
HypoxiaRespiratory, thoracic and mediastinal disorders
ArthralgiaMusculoskeletal and connective tissue disorders
Pain in jawMusculoskeletal and connective tissue disorders
Chronic obstructive pulmonary diseaseRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: Chronic obstructive pulmonary disease, Acute respiratory failure, Acute myocardial infarction, Anaemia, Acute coronary syndrome, Cardiac arrest, Cardiac failure congestive, Right ventricular dysfunction.

Data from ClinicalTrials.gov NCT03496623 adverse events section.

Sponsor's own description

The primary objective of this study is to demonstrate the efficacy of inhaled treprostinil compared to placebo in improving exercise ability as measured by change from baseline in 6-Minute Walk Distance (6MWD) following 12 weeks of active treatment in participants with PH-COPD.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Group 3 Pulmonary Hypertension: From Bench to Bedside.
    Singh N, Dorfmüller P, Shlobin OA, Ventetuolo CE. · · 2022 · cited 55× · PMID 35482836 · DOI 10.1161/circresaha.121.319970
  2. Recent advances in the management of pulmonary hypertension with interstitial lung disease.
    Waxman AB, Elia D, Adir Y, Humbert M, et al · · 2022 · cited 41× · PMID 35831007 · DOI 10.1183/16000617.0220-2021
  3. Inhaled treprostinil in pulmonary hypertension associated with COPD: PERFECT study results.
    Nathan SD, Argula R, Trivieri MG, Aziz S, et al · · 2024 · cited 39× · PMID 38811045 · DOI 10.1183/13993003.00172-2024
  4. New trends in pulmonary hypertension.
    Cullivan S, Gaine S, Sitbon O. · · 2023 · cited 15× · PMID 36813291 · DOI 10.1183/16000617.0211-2022
  5. Pathophysiology of Group 3 Pulmonary Hypertension Associated with Lung Diseases and/or Hypoxia.
    Nakamura K, Akagi S, Ejiri K, Taya S, et al · · 2025 · cited 8× · PMID 39859549 · DOI 10.3390/ijms26020835
  6. Medical Management of Pulmonary Arterial Hypertension: Current Approaches and Investigational Drugs.
    Jin Q, Chen D, Zhang X, Zhang F, et al · · 2023 · cited 8× · PMID 37376028 · DOI 10.3390/pharmaceutics15061579
  7. Management of Pulmonary Hypertension Due to Chronic Lung Disease.
    Sugarman J, Weatherald J. · · 2021 · cited 6× · PMID 34326932 · DOI 10.14797/zkut3813
  8. COPD associated pulmonary hypertension: A post hoc analysis of the PERFECT study.
    Nathan SD, Lacasse V, Bell H, Sista P, et al · · 2024 · cited 4× · PMID 39364449 · DOI 10.1002/pul2.12430

Verify or expand the search:

Other trials of Inhaled treprostinil solution

Trials testing the same drug.

Other recruiting trials for Pulmonary Hypertension

Currently open trials in the same condition.

Other United Therapeutics trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03496623.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing