LAM-001 for Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)
This study is testing LAM-001 (inhaled sirolimus) for adults with pulmonary hypertension (high blood pressure in the lungs) associated with interstitial lung disease (PH-ILD), a condition where lung tissue becomes scarred. You would receive either LAM-001 or a placebo (an inactive substance) through an inhaler once a day for 24 weeks. Researchers want to see if LAM-001 can improve how well blood flows through the lungs and if it is safe. The study aims to enroll 85 participants aged 18 to 80 years old who have a diagnosis of PH-ILD. The study is currently ongoing.
- Study design
- This is a Phase 2, randomized, double-blind (meaning neither you nor your doctor will know if you're getting the active drug or placebo), placebo-controlled study involving approximately 75 participants in this part.
- What's involved
- You would receive daily inhaled medication for 24 weeks, followed by an option to continue LAM-001 for an additional 12 months, and then a 4-week follow-up period.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will complete evaluations during a Follow-Up Period of 4 weeks after treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT05798923
LAM-001 for the Treatment of Pulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)
Recruiting
PHASE2Ages 18–80InterventionalTreatmentOrphAI Therapeutics
~85 participants
Updated 2026-08-03 on ClinicalTrials.gov
What's tested:PlaceboLAM-001
At a glance
Recruiting sites
1 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To determine the change in PVR at 24 weeks
Measured over 24 weeks
Conditions
NCT05798923
Where you'd take part
This study runs at 3 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Brigham and Women's Hospital
Boston, Massachusettsstudy coordinator listed
Not yet recruiting
University of Arizona
Tucson, Arizonastudy coordinator listed
Recruiting
Yale New Haven Hospital
New Haven, Connecticutstudy coordinator listed
Not yet recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Want this trial checked against your situation?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Idiopathic pulmonary fibrosis (IPF)
Idiopathic nonspecific interstitial pneumonia
Respiratory bronchiolitis-associated interstitial lung disease (RB-ILD)
Unclassifiable idiopathic interstitial pneumonia 2. Chronic hypersensitivity pneumonitis (CHP) 3. CTD ILD patients with lung disease findings of \<65% predicted FVC in the setting of diagnosed Connective Tissue Disease 3. Pulmonary function tests within 6 months prior to Screening as follows:
Forced vital capacity (FVC \<65% predicted and a DLCO \>30) for patients with confirmatory high- resolution computed tomography (CT) indicating fibrotic lung disease
For subjects with a history of lobectomy or pneumonectomy, and for whom there are no population- based normalization methods, assessment based on residual lung volume will be permitted to assess eligibility. 4. Hemodynamics consistent with a diagnosis of precapillary PH (mPAP \> 25 mmHg, PCWP \< 15 mmHg, PVR \> 4.0 WU) 5. Symptomatic pulmonary hypertension classified as WHO Functional Class II or III 6. 6MWD ≥ 100 and ≤ 450 meters repeated twice during Screening Period and both values within 15% of each other, calculated from the highest value. 7. On a standard of care PH therapy at stable (per SOC) dose levels for at least 90 days prior to screening.
Stable dose is defined as no change in dose
CTD ILD patients are not required to be on SOC ILD therapy but if they are, must be a stable dose for 90 days 8. Females of childbearing potential must satisfy following:
Have 2 negative pregnancy tests as verified by the investigator prior to starting study and must agree to ongoing pregnancy testing during the study and at end of study treatment.
If sexually active, must have used, and agree to continue to use, highly effective contraception without interruption, for at least 30 days prior to starting investigational product (IP), during the study (including dose interruptions), and for 90 days after discontinuation of study treatment.
Refrain from breastfeeding a child or donating blood, eggs, or ovum for the duration of the study and for at least 90 days after the last dose of study treatment. 9. Male participants must:
Agree to use a condom, defined as a male latex condom or nonlatex condom NOT made from natural (animal) membrane (for example, polyurethane), during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions, and for at least 90 days following IP discontinuation, even if he has undergone a successful vasectomy.
Refrain from donating sperm for the duration of the study and for 90 days after the last dose of study treatment. 10. Ability to adhere to the study visit schedule and understand and comply with all protocol requirements. 11. Ability to understand and provide written informed consent
Exclusion
Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels \> 3x upper limit of normal (ULN) or total bilirubin \> 1.5 x ULN within 28 days of Week 0 Visit
Estimated glomerular filtration rate \< 30 mL/min/1.73 m2 (4-variable Modification of Diet in Renal Disease equation) within 28 days of Week 0 Visit or required renal replacement therapy within 90 days 19. History of opportunistic infection (e.g., invasive candidiasis or Pneumocystis pneumonia) within 6 months prior to Screening; serious local infection (e.g., cellulitis, abscess) or systemic infection (e.g., septicemia) within 3 months prior to Screening 20. History of severe allergic or anaphylactic reaction or hypersensitivity to recombinant proteins or lactose excipients in IP 21. Major surgery within 8 weeks prior to Week 0 Visit. Participants must have completely recovered from any previous surgery prior to Week 0 Visit 22. Prior heart or heart-lung transplants 23. Life expectancy of \< 12 months (per PI determination) 24. Pregnant or breastfeeding females 25. At any time in the 30 days prior to the Screening Period received \> 20 mg/day of prednisone (or equivalent) or started or changed the dose of a systemic corticosteroid. Participants receiving stable doses of ≤ 20 mg prednisone (or equivalent) in 30 days prior to the Screening Period are permitted in the study. 26. History of active malignancy within the past 5 years, with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin 27. History of clinically significant (as determined by the investigator) non-PH related cardiac, endocrine, hematologic, hepatic, immune, metabolic, urologic, pulmonary, neurologic, neuromuscular, dermatologic, psychiatric, renal, and/or other disease that may limit participation in the study 28. Participation in another clinical trial involving intervention with another investigational drug or approved therapy for investigational use within 4 weeks prior to Week 0 Visit, or if the half-life of the previous product is known, within 5x the half-life prior to Week 0 Visit, whichever is longer 29. Participation in another clinical trial involving an investigational device within 4 weeks prior to Week 0 Visit 30. Any recreational drug use (cocaine, marijuana, etc.) within 90 days 31. Unwillingness or inability to comply with the protocol- required procedures
What this trial measures
- To determine the change in PVR at 24 weeks24 weeks
Change in PVR at 24 weeks