L606 Inhalation Suspension for Pulmonary Arterial Hypertension

Not currently recruiting at 4 trial locations
PK
SC
MC
Overseen ByMarisa C. Law
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: Liquidia Technologies, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new inhalation treatment called L606 for individuals with pulmonary arterial hypertension (PAH) or pulmonary hypertension with interstitial lung disease (PH-ILD). The trial aims to assess the safety and tolerability of L606 in both the short and long term, comparing it to an existing treatment, Tyvaso. Researchers will also evaluate how L606 affects exercise ability, quality of life, and treatment satisfaction. Individuals diagnosed with PAH or PH-ILD for at least a year and who experience limited physical activity due to their condition might be suitable candidates for this trial. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that L606 is likely to be safe for humans?

Research shows that L606 is undergoing testing to determine its safety for individuals with pulmonary arterial hypertension (PAH) or pulmonary hypertension with interstitial lung disease (PH-ILD). This trial is in a late stage, indicating that earlier studies found the treatment generally safe and well-tolerated. In these late-stage trials, researchers typically test treatments on more participants to confirm safety and effectiveness. Reaching this stage usually suggests that any side effects observed earlier were manageable. However, joining a trial might still involve risks, and the study team will closely monitor any possible side effects.12345

Why do researchers think this study treatment might be promising?

L606 is unique because it is an inhalation suspension specifically designed for Pulmonary Arterial Hypertension (PAH). Unlike standard treatments, which are often oral or injectable medications like endothelin receptor antagonists or phosphodiesterase-5 inhibitors, L606 is inhaled directly into the lungs. This delivery method allows for targeted action, potentially reducing systemic side effects and improving patient comfort and adherence. Researchers are excited about L606 because it offers a new way to manage PAH, potentially leading to quicker relief and better quality of life for patients.

What evidence suggests that L606 might be an effective treatment for pulmonary arterial hypertension?

Research has shown that L606, which participants in this trial will receive, could help treat pulmonary arterial hypertension (PAH). Patients who switched from a similar treatment, nebulized treprostinil, to L606 did not encounter any major safety problems, suggesting that L606 might be a safe alternative. Early studies also indicate that using inhaled treprostinil, like L606, alongside current oral treatments, may improve symptoms. This could enhance exercise ability and quality of life for patients.56789

Who Is on the Research Team?

Jeremy Feldman - Arizona Pulmonary ...

Jeremy P Feldman, MD

Principal Investigator

Arizona Pulmonary Specialists

EG

Elizabeth Gay, MD

Principal Investigator

Brigham and Women's Hospital

MG

Michael G Risbano, MD

Principal Investigator

UPMC Montefiore

Are You a Good Fit for This Trial?

This trial is for adults aged 18-80 with Pulmonary Arterial Hypertension (PAH) or PH due to Lung Disease (PH-ILD), who can walk at least 150 meters and have a certain level of heart and lung function. They must not have severe heart disease, uncontrolled blood pressure, recent serious illness, liver dysfunction, or kidney failure requiring dialysis.

Inclusion Criteria

I have some level of difficulty with physical activity due to heart problems.
Able to understand and complete study requirements and provide written informed consent
I have been diagnosed with a specific type of lung hypertension for at least a year.
See 2 more

Exclusion Criteria

Your heart's electrical activity, as shown on a test called an electrocardiogram (ECG), is abnormal.
My liver enzymes are not more than three times the normal limit and I don't have severe liver disease.
My kidney function is very low or I am on dialysis.
See 5 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Main Study Period (MSP)

Participants receive L606 to evaluate short-term safety and tolerability

2 weeks for Cohort A, 12 weeks for Cohort B

Open-label Extension Period (OEP)

Participants continue L606 dosing to evaluate long-term safety and tolerability

46 weeks for Cohort A, 36 weeks for Cohort B

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • L606
Trial Overview The study tests the safety and tolerability of L606 inhalation suspension in PAH or PH-ILD patients. It compares its effects on exercise ability, quality of life, and treatment satisfaction against Tyvaso over short-term and long-term use.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: L606Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Liquidia Technologies, Inc.

Lead Sponsor

Trials
7
Recruited
500+

Pharmosa Biopharm Inc.

Lead Sponsor

Trials
4
Recruited
200+

PPD DEVELOPMENT, LP

Industry Sponsor

Trials
167
Recruited
38,000+
David Simmons profile image

David Simmons

PPD DEVELOPMENT, LP

Chief Executive Officer since 2012

BSc in Applied Science from Georgia Institute of Technology

Martina Flammer profile image

Martina Flammer

PPD DEVELOPMENT, LP

Chief Medical Officer since 2024

MD

PPD Development, LP

Industry Sponsor

PPD

Industry Sponsor

Trials
162
Recruited
36,600+
Dr. Austin Smith profile image

Dr. Austin Smith

PPD

Chief Medical Officer since 2020

Doctor of Medicine from the Royal College of Surgeons in Ireland

David Simmons profile image

David Simmons

PPD

Chief Executive Officer since 2012

Bachelor’s degree in Applied Mathematics and Industrial Management from Carnegie Mellon University

Published Research Related to This Trial

Recent clinical trials have shifted the treatment approach for pulmonary arterial hypertension (PAH) from a clinical-based strategy to an evidence-based therapy, allowing for a more systematic treatment algorithm based on the efficacy and side-effect profiles of various approved drugs.
The proposed treatment algorithm is specifically designed for patients in NYHA functional class III or IV, recommending oral anticoagulation for all, and suggesting specific therapies like endothelin receptor antagonists or prostanoids for nonresponders to acute vasoreactivity testing, with continuous intravenous epoprostenol as a rescue option for the most severe cases.
Comparative analysis of clinical trials and evidence-based treatment algorithm in pulmonary arterial hypertension.Galiè, N., Seeger, W., Naeije, R., et al.[2021]
Early recognition and referral of patients with pulmonary arterial hypertension (PAH) to specialized centers is crucial for improving outcomes, as novel treatments and rehabilitation programs can enhance exercise capacity and quality of life.
Current medical treatments, including prostanoids and endothelin antagonists, not only promote pulmonary vasodilation but may also address the underlying vascular abnormalities in PAH, with emerging evidence suggesting that combination therapy may be more effective than single-agent treatments.
Current treatment approaches to pulmonary arterial hypertension.Provencher, S., Granton, JT.[2022]

Citations

A Phase 3, 2-part, Open-label, Multicenter Study to ...CONCLUSIONS: Patients receiving stable doses of nebulized treprostinil successfully transitioned to L606 with no significant safety concerns and the addition of ...
NCT04691154 | A Phase 3 Study to Evaluate the Safety ...This Phase 3, 2-part, open-label, multicenter study aims to demonstrate the safety and tolerability of repeated doses of L606 in patients with PAH or PH-ILD.
An Open-Label Safety Study of L606 (Liposomal ...Results from the continuation of this study will be pivotal in establishing the long-term safety and efficacy of L606 in patients with PAH and PH-ILD. Table 1.
Navigating options for inhaled treprostinil therapy in ...Addition of inhaled treprostinil to oral therapy for pulmonary arterial hypertension: a randomized controlled clinical trial ... efficacy ...
Future Perspectives of Pulmonary Arterial HypertensionThis review aims to provide an overview of novel drugs or formulations and new drug indications for pulmonary arterial hypertension
NCT04691154 | A Phase 3 Study to Evaluate the Safety ...This Phase 3, 2-part, open-label, multicenter study aims to demonstrate the safety and tolerability of repeated doses of L606 in patients with PAH or PH-ILD.
Press releaseThe Phase 3, 2-part, open-label, multicenter study aims to demonstrate the safety and tolerability of L606 in patients with PAH or PH-ILD in the short-term and ...
L606 Inhalation Suspension for Pulmonary Arterial ...Trial Overview The study tests the safety and tolerability of L606 inhalation suspension in PAH or PH-ILD patients. It compares its effects on exercise ability, ...
ProtocolThe principal aim of this study is to obtain safety and tolerability data when L606 is administered as an oral inhalation as single doses to healthy. Page 15 ...
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