A Phase 2a Study of LAM-001 for the Treatment of Pulmonary Hypertension

NCT05798923 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: LAM-001-PAH-CLN01
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 4

Brief Summary

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

Conditions

Pulmonary Hypertension

Keywords

Pulmonary Hypertension

Outcome Measures

Primary Outcomes (1)

• Change from baseline VO2 max

  24 weeks

Secondary Outcomes (19)

• Change in VE/VCO2 slope (ventilatory efficiency)

  24 weeks

• Change in cardiac output (CO)

  24 weeks

• Change in cardiac index (CI)

  24 weeks

• Change in stroke volume (SV)

  24 weeks

• Change in mean pulmonary arterial pressure (mPAP)

  24 weeks

Study Arms (1)

LAM-001

EXPERIMENTAL

Drug: LAM-001

Interventions

LAM-001 DRUG

LAM-001 administered via dry powder inhalation

LAM-001

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years
• Screening consistent with a diagnosis of precapillary PH (mPAP \> 25 mmHg, PCWP \< 18 mmHg, PVR \>4WU) that is due to either:
• WSPH Group 1 PH (i.e., PAH of any of the following subtypes)
• Idiopathic PAH
• Heritable PAH
• Drug- or toxin-induced PAH
• PAH associated with connective tissue disease
• PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following shunt repair
• Confirmed diagnosis of WSPH Group 3 PH with advanced lung disease as defined by CT imaging (see Section 5.3.1) within 6- months of screening that demonstrates diffuse parenchymal lung disease or FVC \< 65% of predicted for this cohort only, associated with one of the following:
• i. Idiopathic interstitial pneumonia (IIP) including:
• Idiopathic pulmonary fibrosis (IPF)
• Idiopathic nonspecific interstitial pneumonia
• Respiratory bronchiolitis-associated interstitial ling disease (RB-ILD)
• Unclassifiable idiopathic interstitial pneumonia ii. Chronic hypersensitivity pneumonitis (CHP) iii. Occupational lung disease (drug or radiation-induced) iv. Combined pulmonary fibrosis and emphysema (CPFE)
• Symptomatic pulmonary hypertension classified as WHO functional class III

You may not qualify if:

• Started or stopped receiving any general supportive therapy for pulmonary hypertension within 30 days prior to Week 0 Visit
• Received IV inotropes (e.g., dobutamine, dopamine, norepinephrine, vasopressin) within 30 days prior to Week 0 Visit
• History of atrial septostomy within 180 days prior to Screening Visit
• History of more than moderate obstructive sleep apnea that is untreated
• Prior exposure to oral sirolimus or any other mTOR inhibitor within last three months
• Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior to Week 0 Visit or planned initiation during the study (participants who are stable in the maintenance phase of a program and who will continue for the duration of the study are eligible)
• Uncontrolled systemic hypertension as evidenced by sitting systolic BP \> 160 mmHg or sitting diastolic BP \> 100 mmHg during Screening Visit after a period of rest
• Systolic BP \< 90 mmHg during Screening Visit or at baseline
• History of known pericardial constriction
• RHC contraindicated during the study per investigator
• Personal or family history of long QTc syndrome or sudden cardiac death
• Cerebrovascular accident within 3 months of Week 0 Visit
• History of restrictive or constrictive cardiomyopathy
• Left ventricular ejection fraction \< 45% on echocardiogram performed within 6 months prior to Screening Period (or done as a part of the Screening Period), or PCWP \> 18 mmHg as determined in the Screening Period RHC
• Any current symptomatic coronary disease (myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or cardiac anginal chest pain in the past 6 months prior to Screening Visit)

Sponsors & Collaborators

• OrphAI Therapeutics: lead

Study Sites (4)

University of Arizona

Tucson, Arizona, 85748, United States

Location

Yale New Haven Hospital

New Haven, Connecticut, 06510, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

MeSH Terms

Conditions

Hypertension, Pulmonary

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 13, 2023
• First Posted: April 5, 2023
• Study Start: April 3, 2023
• Primary Completion: May 16, 2025
• Study Completion (Estimated): September 30, 2027
• Last Updated: June 22, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)