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NCT03043651

Open-label Extension of Oral Treprostinil in Subjects With PH Associated With HFpEF

Terminated Phase 3 Results posted Last updated 22 October 2020
What this trial tests

Phase 3 trial testing Oral treprostinil in Pulmonary Hypertension Associated With HFpEF in 48 participants. Terminated before completion.

Timeline
30 January 2018
Primary endpoint
2 March 2020
2 March 2020

Quick facts

Lead sponsorUnited Therapeutics
PhasePhase 3
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment48
Start date30 January 2018
Primary completion2 March 2020
Estimated completion2 March 2020
Sites82 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

United Therapeutics — full company profile →

Who can join

Adults 18 to 85, any sex, with Pulmonary Hypertension Associated With HFpEF. 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.

Long-term Safety of Oral Treprostinil in Subjects With PH Associated With HFpEF for Subjects Who Completed Study TDE-HF-301 Primary · Baseline through study completion, up to approximately 25 months

The primary objective of this study was to evaluate the long-term safety of oral treprostinil in subjects with PH associated with HFpEF for subjects who completed Study TDE-HF-301. The number of subjects with adverse events during the study is reported as the primary outcome measure, which was the only outcome measurement reported for this Sponsor-terminated study.

GroupValue95% CI
Oral Treprostinil47

Adverse events — posted to ClinicalTrials.gov

Time frame: The study duration was 25 months overall. The study was discontinued by the Sponsor on 14 October 2019 due to slow enrollment in the parent study, TDE-HF-301.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Oral Treprostinil
Serious: 19/48 (40%)
Deaths: 1/48

Serious adverse events (38 terms)

ReactionSystemOral Treprostinil
Acute kidney injuryRenal and urinary disorders
Cardiac failureCardiac disorders
Acute respiratory failureRespiratory, thoracic and mediastinal disorders
Cardiac failure acuteCardiac disorders
Cardiac failure congestiveCardiac disorders
HypokalaemiaMetabolism and nutrition disorders
Pleural effusionRespiratory, thoracic and mediastinal disorders
PneumoniaInfections and infestations
Atrial fibrillationCardiac disorders
AzotaemiaRenal and urinary disorders
Cardio-respiratory arrestCardiac disorders
CardiomyopathyCardiac disorders
Cerebrovascular accidentNervous system disorders
Chest painGeneral disorders
Chronic obstructive pulmonary diseaseRespiratory, thoracic and mediastinal disorders
Confusional statePsychiatric disorders
DeathGeneral disorders
DiarrhoeaGastrointestinal disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
Electrocardiogram QT prolongedInvestigations
EndocarditisInfections and infestations
Fluid overloadMetabolism and nutrition disorders
Gastrointestinal angiectasiaGastrointestinal disorders
HeadacheNervous system disorders
Hip fractureInjury, poisoning and procedural complications
Other adverse events (44 terms — click to expand)

ReactionSystemOral Treprostinil
HeadacheNervous system disorders
DiarrhoeaGastrointestinal disorders
NauseaGastrointestinal disorders
FatigueGeneral disorders
Pain in extremityMusculoskeletal and connective tissue disorders
DizzinessNervous system disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
Oedema peripheralGeneral disorders
Back painMusculoskeletal and connective tissue disorders
CoughRespiratory, thoracic and mediastinal disorders
Pain in jawMusculoskeletal and connective tissue disorders
ArthralgiaMusculoskeletal and connective tissue disorders
DyspepsiaGastrointestinal disorders
Acute kidney injuryRenal and urinary disorders
Blood creatinine increasedInvestigations
Chest painGeneral disorders
FlushingVascular disorders
Muscle spasmsMusculoskeletal and connective tissue disorders
PneumoniaInfections and infestations
Upper respiratory tract infectionInfections and infestations
Abdominal painGastrointestinal disorders
Atrial fibrillationCardiac disorders
ConstipationGastrointestinal disorders
Decreased appetiteMetabolism and nutrition disorders
HypotensionVascular disorders
OedemaGeneral disorders
RashSkin and subcutaneous tissue disorders
SinusitisInfections and infestations
VomitingGastrointestinal disorders
Abdominal distensionGastrointestinal disorders
AnaemiaBlood and lymphatic system disorders
Blood alkaline phosphatase increasedInvestigations
Cardiac failureCardiac disorders
FlatulenceGastrointestinal disorders
Gamma-glutamyltransferase increasedInvestigations
Musculoskeletal painMusculoskeletal and connective tissue disorders
MyalgiaMusculoskeletal and connective tissue disorders
N-terminal prohormone brain natriuretic peptide increasedInvestigations
PalpitationsCardiac disorders
Pleural effusionRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: Acute kidney injury, Cardiac failure, Acute respiratory failure, Cardiac failure acute, Cardiac failure congestive, Hypokalaemia, Pleural effusion, Pneumonia.

Data from ClinicalTrials.gov NCT03043651 adverse events section.

Sponsor's own description

This was an open-label study to evaluate the safety of continued therapy with oral treprostinil in subjects who completed Study TDE-HF-301. This study provided long-term, open-label data regarding the effect of continued long-term oral treprostinil therapy for the treatment of pulmonary hypertension (PH) associated with heart failure with preserved ejection fraction (HFpEF). Subject visits occurred at Baseline, Weeks 6, 12, 18, 24, and every 12 weeks thereafter until either oral treprostinil was commercially available to treat PH associated with HFpEF or the study was discontinued by the Sponsor. The Sponsor terminated Studies TDE-HF-301 and TDE-HF-302 on 14 October 2019 due to slow enrollment. Safety data from the final subject in Study TDE-HF-302 were recorded on 02 March 2020. Due to the lower than expected number of subjects enrolled, the planned secondary efficacy-related endpoints were not analyzed.

Publications & conference data

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

  1. Immunomodulation in Heart Failure with Preserved Ejection Fraction: Current State and Future Perspectives.
    Kessler EL, Oerlemans MIFJ, van den Hoogen P, Yap C, et al · · 2021 · cited 15× · PMID 32444946 · DOI 10.1007/s12265-020-10026-3
  2. A roadmap for therapeutic discovery in pulmonary hypertension associated with left heart failure. A scientific statement of the Heart Failure Association (HFA) of the ESC and the ESC Working Group on Pulmonary Circulation & Right Ventricular Function.
    Ameri P, Mercurio V, Pollesello P, Anker MS, et al · · 2024 · cited 12× · PMID 38639017 · DOI 10.1002/ejhf.3236
  3. Heart Failure with Preserved Ejection Fraction: a Pharmacotherapeutic Update.
    Vaz-Salvador P, Adão R, Vasconcelos I, Leite-Moreira AF, et al · · 2023 · cited 5× · PMID 35098432 · DOI 10.1007/s10557-021-07306-8

Verify or expand the search:

Other trials of Oral treprostinil

Trials testing the same drug.

Other recruiting trials for Pulmonary Hypertension Associated With HFpEF

Currently open trials in the same condition.

Other United Therapeutics trials

Trials by the same sponsor.

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