LAM-001 for Pulmonary Hypertension

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

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a clinical trial to assess the efficacy and safety of LAM-001 as an add-on therapy for the treatment pulmonary hypertension.

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.

What data supports the effectiveness of the drug LAM-001 for pulmonary hypertension?

The research on sotatercept, a similar treatment for pulmonary arterial hypertension, shows that targeting specific growth pathways can help manage the condition. This suggests that LAM-001 might also be effective if it works in a similar way. ^{1 2 3 4 5}

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

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]

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]

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]

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]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Sotatercept for the Treatment of Pulmonary Arterial Hypertension. [2021]

2. England pubmed.ncbi.nlm.nih.gov

Why drugs fail in clinical trials in pulmonary arterial hypertension, and strategies to succeed in the future. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Clinical efficacy and safety of selexipag in children and young adults with idiopathic and heritable pulmonary arterial hypertension. [2023]

4. Italy pubmed.ncbi.nlm.nih.gov

Advanced therapeutic options in pulmonary hypertension: who, what and when? [2016]

5. England pubmed.ncbi.nlm.nih.gov

Treatment of pulmonary hypertension: bench to bedside. [2021]