Inhaled Treprostinil for Hypertension, Pulmonary

1
Effectiveness
2
Safety
University of Florida, Division of Pulmonary and Critical Care Medicine, Gainesville, FL
+6 More
Inhaled Treprostinil - Drug
Eligibility
18+
All Sexes
Eligible conditions
Hypertension, Pulmonary

Study Summary

This study is evaluating whether inhaled treprostinil may help improve lung function for individuals with sarcoidosis.

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Eligible Conditions

  • Hypertension, Pulmonary
  • Lung Diseases
  • Sarcoidosis
  • Hypertension
  • Pneumonia, Interstitial
  • Interstitial Lung Diseases (ILD)
  • Precapillary Pulmonary Hypertension
  • Lung Diseases, Interstitial

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Inhaled Treprostinil will improve 2 primary outcomes and 5 secondary outcomes in patients with Hypertension, Pulmonary. Measurement will happen over the course of Baseline, Week 16.

Baseline, Week 16
Change in Brain Natriuretic Peptide (BNP)
Change in Cardiac MRI parameters
Change in Pulmonary Function Testing
PVR by Right heart catheterization (RHC)
mPAP by Right heart catheterization (RHC)
Week 16
Change in 6-Minute Walk Test (6MWT)
Change in WHO Functional Class (WHO FC)

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Side Effects for

Active Inhaled Treprostinil
Cough
44%
Headache
28%
Dyspnoea
25%
Dizziness
18%
Nausea
15%
Fatigue
14%
Diarrhea
13%
Throat irritation
12%
Oropharyngeal pain
11%
Chest pain
9%
Oedema peripheral
8%
Upper respiratory tract infection
7%
N-terminal prohormone brain natriuretic peptide increased
6%
Epistaxis
6%
Chest discomfort
5%
Fall
5%
Rhinorrhoea
5%
Decreased appetite
4%
Acute respiratory failure
2%
Death
2%
Interstitial lung disease
2%
Cellulitis
1%
Cerebral haemorrhage
1%
Chronic respiratory failure
1%
Pneumonia
1%
Pneumothorax
1%
Arrythmia
1%
Hypoxia
1%
Sepsis
1%
Bradycardia
1%
Cor pulmonale
1%
Haemoptysis
1%
Respiratory failure
1%
Left ventricular failure
1%
Pulmonary hypertension
1%
Cardiopulmonary failure
1%
Idiopathic pulmonary fibrosis
1%
Cardiac failure congestive
1%
Bronchitis
1%
Combined pulmonary fibrosis and emphysema
1%
Pain in extremity
1%
Influenza
1%
Bronchopulmonary aspergillosis
1%
Acute right ventricular failure
1%
Hyperglycaemia
1%
Tachycardia
1%
Acute myocardial infarction
1%
Rhinovirus infetion
1%
Cardiac arrest
1%
Right ventricular failure
1%
B-cell lymphoma
1%
Hypervolaemia
1%
Syncope
1%
Chronic obstructive pulmonary disease
1%
Urosepsis
0%
Aspiration
0%
Fluid overload
0%
Acute kidney injury
0%
Pulmonary oedema
0%
Lumbar vertebral fracture
0%
Respiratory distress
0%
Cor pulmonale acute
0%
Haematochezia
0%
Hypertension
0%
Pulmonary congestion
0%
Cardiogenic shock
0%
Atrial fibrillation
0%
Cardiac failure
0%
Metabolic encephalopathy
0%
Disease progression
0%
Coagulopathy
0%
Pneumonia influenzal
0%
Scleroderma
0%
Presyncope
0%
Chronic right ventricular failure
0%
Coronary artery disease
0%
Cardiac failure acute
0%
Abdominal pain
0%
Pain
0%
Transplant dysfunction
0%
Post procedural infection
0%
This histogram enumerates side effects from a completed 2019 Phase 2 & 3 trial (NCT02630316) in the Active Inhaled Treprostinil ARM group. Side effects include: Cough with 44%, Headache with 28%, Dyspnoea with 25%, Dizziness with 18%, Nausea with 15%.

Trial Design

2 Treatment Groups

Control
Study Group

This trial requires 10 total participants across 2 different treatment groups

This trial involves 2 different treatments. Inhaled Treprostinil is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Study Group
Drug
Sarcoidosis patients with interstitial lung disease and precapillary pulmonary hypertension based on right heart catheterization (RHC). All subjects will initiate inhaled treprostinil at a dose of 3 breaths (18 mcg) four times daily. Study drug doses escalations (additional one breath four times daily) can occur every three days with a maximum dosing regimen of up to 12 breaths (72 mcg) four times daily, as clinically tolerated.
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Treprostinil
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, week 8, week 16
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline, week 8, week 16 for reporting.

Closest Location

University of Florida, Division of Pulmonary and Critical Care Medicine - Gainesville, FL

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Sarcoidosis is a rare, chronic, multisystem inflammatory disorder that may affect any organ system show original
(CT) is an indication for transplant If a person has Stage IV chest radiograph or extensive fibrosis on chest computed tomography (CT), this means they have interstitial lung disease and they should get a transplant. show original
Patients who have been treated for sarcoidosis for at least three months prior to screening are eligible for the study. show original
The subject had a right heart catheterization within six months of their baseline visit and their precapillary pulmonary hypertension was found to be greater than 25 mmHg, their PCWP was less than 15 mmHg, and their PVR was greater than 3 WU. show original
The study participant is willing and able to provide informed consent. show original
This means that the women taking part in the study did not have a positive pregnancy test at the beginning of the study. show original
If patients have been taking an oral PAH therapy (such as a PDE5 inhibitor or an endothelin receptor antagonist) for at least three months, their dose should be stable before they are screened. show original
is predictive of a successful CRT candidacy If someone can walk more than 100 meters within three months of their screening visit, they are likely to be a successful candidate for a CRT. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are common treatments for sarcoidosis?

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Immunosuppression and corticosteroids are the standard therapy for sarcoidosis. Immunosuppression is used either as a single agent or in combination with other medications. Corticosteroids (and sometimes an immunosuppressive drug such as cyclosporine or azathioprine) are used as monotherapy and in combination with immunosuppressive therapy. There are no randomized controlled trials for the treatment of sarcoidosis.

Unverified Answer

Can sarcoidosis be cured?

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Sarcoidosis can be cured. This is possible if the treatment is started early in the process and continued for many years. Sarcoidosis can be effectively treated and managed with good quality of life as good as that obtained in unaffected relatives.

Unverified Answer

What are the signs of sarcoidosis?

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Sarcoidosis is probably diagnosed at an early stage. A high index of suspicion is necessary when patients present with tuberculosis, pulmonary edema, respiratory disturbances, unexplained fever lasting for longer than 2 weeks, unexplained weight loss, adenopathy, or pulmonary function impairment. An occupational history of tuberculosis, exposure to hard sand, and abnormal chest X-ray have a high sensitivity for detecting sarcoidosis. The chest X-ray must be reviewed and a history taken by the doctor who makes the diagnosis. The most common symptom of sarcoidosis is a lung examination in which lung nodules may be present and in some cases, they may be bilateral.

Unverified Answer

What causes sarcoidosis?

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Exposure to certain environmental substances, such as mycobacterium, might be a factor influencing susceptibility to Sarcoidosis. A more in-depth knowledge of the pathogenesis of Sarcoidosis is expected to allow a better understanding of the complex etiology of the disease.

Unverified Answer

What is sarcoidosis?

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Sarcoidosis is a disease of unknown cause with multiple etiologies; the most common form in Caucasian adults is systemic sclerosis, secondary infection and exposure to inhaled xenobiotics being the most frequent predisposing factors. Sarcoidosis can present with various non-specific clinical manifestations including pulmonary nodules and hilar adenopathy in which the condition can also be confused with tuberculosis. The diagnosis is often confirmed by pulmonary biopsy. Non-specific findings are frequently encountered in sarcoidosis and should not lead to diagnosis of the disease.

Unverified Answer

How many people get sarcoidosis a year in the United States?

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Although sarcoidosis is fairly common—it occurs in approximately one person in every 1,600—the number of people with the condition who ever receive medical attention for sarcoidosis is low. If sarcoidosis was to be treated on the basis of current practices and if physicians and patients were more appropriately warned about the limitations of available treatments, many people with sarcoidosis would be misdiagnosed. The use of a high throughput screening approach to sarcoidosis, however, is warranted.

Unverified Answer

Is inhaled treprostinil typically used in combination with any other treatments?

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The majority of patients taking inhaled treprostinil, reported no additional benefits for other treatment options. A minority reported worsening of their symptoms. These preliminary data suggest the use of inhaled treprostinil is not always combined with other therapy; the efficacy of treprostinil on pulmonary disease remains questionable, however, and warrants further evaluation.

Unverified Answer

Have there been other clinical trials involving inhaled treprostinil?

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The study demonstrates that treprostinil was effective in treating and improving symptoms of severe respiratory failure and hypoxia. The drug is well tolerated and warrants further investigation as a treatment for other forms of severe respiratory disease.

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Does sarcoidosis run in families?

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Results from a recent paper indicate that sarcoidosis is associated with a high prevalence of familial inheritance. A large multicenter study is needed to confirm these findings and determine the clinical relevance of sarcoidosis in a multi-ethnic population.

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What are the latest developments in inhaled treprostinil for therapeutic use?

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Inhaled treprostinil is a novel drug which may have a significant impact on the management of pulmonary arterial hypertension and other cardiovascular diseases associated with sarcoidosis.

Unverified Answer

What is the average age someone gets sarcoidosis?

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Symptoms may be present as early as age 15 years or as late as 44 years. In the US, sarcoidosis is most common between the ages of 34-41. It occurs more commonly in women in all age groups: women are 4 to 6 times more likely to get sarcoidosis compared to men and in adults, women are more likely to get the disease. Older patients are more likely to present with a lower lung function and radiographic disease. There must be a biological explanation for this association and why women are so predisposed to sarcoidosis answer: answer: Sarcoidosis in Chinese, especially female, occurs earlier in life and has a late development interval.

Unverified Answer

Has inhaled treprostinil proven to be more effective than a placebo?

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This randomized clinical trial demonstrated superiority of inhaled treprostinil over placebo. We conclude that this novel prostanoid treatment should not merely be regarded as a salvage therapy but as a new therapeutic approach, with the potential for improving pulmonary function.

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