LAM-001 for Pulmonary Hypertension

Not currently recruiting at 6 trial locations

Overseen By OrphAI Therapeutics

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: OrphAI Therapeutics

Must be taking: PAH therapy

Must not be taking: IV inotropes, mTOR inhibitors

Disqualifiers: Uncontrolled hypertension, Heart failure, Coronary disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called LAM-001 to determine its effectiveness for people with pulmonary hypertension, a condition where high blood pressure affects the lungs' arteries. The goal is to assess the safety and efficacy of LAM-001 when used alongside other treatments. Suitable participants have pulmonary hypertension that causes noticeable symptoms during everyday activities. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants the opportunity to contribute to significant advancements in pulmonary hypertension treatment.

Will I have to stop taking my current medications?

The trial requires that you be on a stable dose of your current pulmonary hypertension medication for at least 30 days before starting. If you are on any other medications, the protocol does not specify if you need to stop them, but it's best to discuss with the trial team.

Is there any evidence suggesting that LAM-001 is likely to be safe for humans?

Research has shown that LAM-001 has been tested for safety and tolerability. Earlier studies indicate that most people can use LAM-001 without serious side effects. However, side effects can occur with any treatment. Current research aims to ensure the treatment is safe and effective for individuals with pulmonary hypertension, which is high blood pressure in the lungs. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for pulmonary hypertension, which often include medications like endothelin receptor antagonists, phosphodiesterase-5 inhibitors, and prostacyclin analogs, LAM-001 works differently. Researchers are excited about LAM-001 because it targets a unique pathway that could potentially improve blood flow in the lungs more effectively than current options. This novel mechanism of action offers hope for better management of symptoms and quality of life for patients with pulmonary hypertension.

What evidence suggests that LAM-001 might be an effective treatment for pulmonary hypertension?

Research shows that LAM-001, which participants in this trial will receive, is being tested as an additional treatment for pulmonary hypertension, a condition characterized by high blood pressure in the lungs. Early studies suggest that LAM-001 might improve symptoms by targeting pathways that relax blood vessels in the lungs, potentially lowering blood pressure. Although research in humans continues, focusing on these pathways gives researchers hope that LAM-001 could benefit patients. More data from current studies will help confirm these early findings. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

Adults diagnosed with pulmonary hypertension who can walk a certain distance and are already on stable PAH therapy may join. They must not have had major surgery or other clinical trials recently, no active cancer in the past 5 years, no severe heart issues, and their liver and kidney functions should be within specific limits. Women of childbearing age must use contraception; men must agree to use condoms.

Inclusion Criteria

Ability to understand and provide written informed consent

I am using approved birth control methods if I can become pregnant.

I agree to use a condom and not donate sperm during the study.

Exclusion Criteria

I have been diagnosed with a serious heart valve condition.

Pregnant or breastfeeding females

Your heart's pumping ability is less than 45% on a special heart test.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive standard of care plus LAM-001 once daily for 24 weeks

24 weeks

Extension

Participants with a favorable benefit-risk profile continue receiving LAM-001 for up to 12 months

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• LAM-001

Trial Overview The trial is testing LAM-001 as an additional treatment for those with pulmonary hypertension. It aims to see if adding LAM-001 to existing standard care improves patient outcomes compared to what's currently available without this new drug.

How Is the Trial Designed?

Group I: LAM-001 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

OrphAI Therapeutics

Lead Sponsor

Trials

7

Recruited

280+

AI Therapeutics, Inc.

Lead Sponsor

Trials

7

Recruited

280+

Published Research Related to This Trial

The development of new treatments for pulmonary arterial hypertension (PAH) has seen both successes and failures, highlighting the need for careful selection of drug targets and early evaluation of how different doses affect patients.

Utilizing advanced technologies like 'omics' and clinical imaging can help improve patient selection and understanding of treatment responses, potentially making drug development more efficient and cost-effective for PAH therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Why drugs fail in clinical trials in pulmonary arterial hypertension, and strategies to succeed in the future. Lythgoe, MP., Rhodes, CJ., Ghataorhe, P., et al.[2022]

In a study of 11 children and young adults with pulmonary arterial hypertension, selexipag showed significant improvements in key hemodynamic parameters, such as mean pulmonary arterial pressure and cardiac index, indicating its potential efficacy as a treatment.

The treatment was generally well-tolerated, with no clinical worsening observed in patients, although one young adult discontinued due to severe headache, highlighting the need to monitor for side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical efficacy and safety of selexipag in children and young adults with idiopathic and heritable pulmonary arterial hypertension. Takatsuki, S., Nakayama, T., Shimizu, Y., et al.[2023]

Pulmonary arterial hypertension (PAH) has seen significant drug development, with successful treatments like endothelin receptor antagonists and prostacyclin receptor agonists emerging from translational research focused on the pulmonary vasculature and right ventricle.

Despite some failures in drug development, ongoing investigations into new drugs and gene therapy hold promise for future advancements in treating this orphan disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of pulmonary hypertension: bench to bedside. Springer, J., Lainscak, M., Salobir, B., et al.[2021]

Recent advancements in pulmonary hypertension (PH) treatment have introduced new agents and reinforced the efficacy of existing drugs, aiming to improve patient outcomes based on PH type and symptom severity.

While there have been improvements in patient outcomes, particularly in types I and IV PH, the benefits in hemodynamics and symptoms are generally modest and vary among individuals, highlighting the need for personalized treatment approaches.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Advanced therapeutic options in pulmonary hypertension: who, what and when? Singh, M., Mishra, A., Rudzinski, W., et al.[2016]

In a 24-week trial involving 106 adults with pulmonary arterial hypertension, sotatercept significantly reduced pulmonary vascular resistance compared to placebo, indicating its efficacy in improving this condition.

Sotatercept also improved exercise capacity, as shown by a greater increase in 6-minute walk distance in the treatment groups, although some hematologic adverse events were noted, including thrombocytopenia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sotatercept for the Treatment of Pulmonary Arterial Hypertension. Humbert, M., McLaughlin, V., Gibbs, JSR., et al.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05798923

NCT05798923 | A Phase 2a Study of LAM-001 for the ... This is a clinical trial to assess the efficacy and safety of LAM-001 as an add-on therapy for the treatment pulmonary hypertension. Detailed Description. This ...

2. orphai-therapeutics.com orphai-therapeutics.com/lam-001-product-page-menu

LAM-001 OrphAI Therapeutics has initiated clinical studies of LAM-001 in the rare lung diseases Pulmonary Hypertension (PH) and Bronchiolitis Obliterans Syndrome (BOS).

3. rarediseaseadvisor.com rarediseaseadvisor.com/news/initiation-of-phase-2-study-investigating-lam-001-for-pah-treatment/

Phase 2 Study Investigating LAM-001 for PAH Treatment ... The 24-week, single-arm, open-label, phase 2, exploratory study is set to evaluate the safety, tolerability, and efficacy of LAM-001 in 15 adult ...

4. medicine.yale.edu medicine.yale.edu/trial/lam-001-pah-clno1/

A Phase 2a Study of LAM-001 for the Treatment of Pulmonary ... This is a research study that aims to evaluate the effectiveness of sirolimus in individuals with pulmonary hypertension.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7892490/

Clinical Outcomes and Treatment Options in Patients With ... At 5 and 10 years, the estimated PH-related death-free and lung transplantation-free survival rate was 100% (95% CI: 100–100%) and 87.5% (95% CI: 38.7–98.1%), ...

6. clinicaltrialsarena.com clinicaltrialsarena.com/news/ai-therapeutics-trial-cardiopulmonary/

AI Therapeutics begins trial of cardiopulmonary disease ... The trial will measure LAM-001's tolerability, safety and activity in WHO-functional Class III adults with advanced PAH. September 1, 2023.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT06018766

NCT06018766 | LAM-001 in Lung Transplant Recipients ... The goal of this clinical trial is to learn about the safety and effectiveness of LAM-001 in patients who have developed bronchiolitis obliterans syndrome (BOS) ...