Actively Recruiting

Phase 2
Age: 18Years +
All Genders
ID06018766

A Randomized, Placebo-controlled Phase 2 Study to Evaluate the Safety and Effectiveness of Inhaled LAM-001 Added to Standard Immunosuppression for Bronchiolitis Obliterans Syndrome After Lung Transplantation

Led by Steven Hays, MD · Updated on 2026-05-18

30

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

Sponsors

S

Steven Hays, MD

Lead Sponsor

O

OrphAI Therapeutics

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effectiveness of LAM-001 in patients who have developed bronchiolitis obliterans syndrome (BOS), a form of chronic rejection following lung transplantation. This phase 2 trial aims to determine if inhaled LAM-001 can slow the progression of BOS, a serious condition that limits long-term survival after lung transplant. BOS currently has no effective treatment and is characterized by a steady decline in lung function despite available therapies. LAM-001 is designed to deliver sirolimus directly to the lungs, potentially reducing side effects seen with oral treatment. Participants in this randomized, placebo-controlled study will inhale either LAM-001 or a placebo daily for 48 weeks using a dry powder inhaler. The study includes 10 visits over this period, a mix of in-person and telehealth appointments. During the trial, participants will continue their standard immunosuppression therapy. Researchers will monitor lung function, including weekly home spirometry, and perform bronchoscopy, lab tests, and physical exams to assess safety and treatment effects. Throughout the study, participants will undergo regular assessments such as pulmonary function tests and bronchoscopy when needed. Lung function changes will be closely tracked, focusing on forced expiratory volume (FEV1) to evaluate treatment impact. Safety will also be monitored continuously. The total participation time is 48 weeks, with researchers comparing outcomes between those receiving LAM-001 and placebo to determine the drug's tolerability and effectiveness in slowing BOS progression.

CONDITIONS

Brief Title

LAM-001 in Lung Transplant Recipients With Bronchiolitis Obliterans Syndrome.

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age over 18 years old
  • Recipient of a double lung transplant at least 12 months before joining
  • Clinical diagnosis of chronic lung allograft dysfunction with BOS phenotype, including FEV1 between 85-51% of baseline, diagnosis within 12 months, and persistent FEV1 decline over 30 days
  • Currently on stable standard immunosuppression with Prednisone, Mycophenolate or Azathioprine, and Tacrolimus or Cyclosporine for at least 4 weeks
  • No oral sirolimus or everolimus treatment for at least 4 weeks prior to screening
  • Stable enough for routine post-transplant bronchoscopy with BAL and biopsy if needed
  • Able to understand study purposes and risks and give informed consent
  • Women of childbearing potential must have a negative pregnancy test within 7 days before joining
  • Women of childbearing potential must agree to use effective contraception during the study and for 90 days after last dose
  • Women of childbearing potential must avoid breastfeeding and egg donation during study and 90 days after last dose
  • Male participants must agree to use condoms with women of childbearing potential during the study and 90 days after last dose
  • Male participants must avoid sperm donation during the study and 90 days after last dose
Not Eligible

You will not qualify if you...

  • Pregnant, breastfeeding, or unwilling to use birth control during the study
  • Any condition or abnormality that may compromise safety or data quality as judged by the investigator
  • History of lung re-transplantation or currently listed for re-transplant
  • Other known causes for lung function loss such as acute infection, acute rejection, or restrictive allograft syndrome
  • Acute antibody-mediated rejection at screening (except stable patients with detectable donor-specific antibodies)
  • Active unresolved infection within 4 weeks prior to screening
  • Mechanical ventilation within 12 weeks before randomization
  • Resting oxygen saturation below 89% on room air or use of supplemental oxygen at rest at screening
  • Functional airway stenosis needing intervention after BOS diagnosis
  • Known allergy to sirolimus or everolimus
  • Currently enrolled in another investigational trial for obstructive chronic lung allograft dysfunction
  • Chronic renal failure with serum creatinine above 2.5 mg/dL or dialysis
  • Liver disease with bilirubin or transaminases above specified limits
  • Active malignancy within past 2 years except treated localized skin cancers
  • History of malignancy likely to cause significant disability or need major intervention within 6 months
  • Severe allergic reaction to lactose (lactose intolerance allowed)
  • Uncontrolled high blood pressure

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 screening and enrollment visit

Treatment

Duration - 48 weeks

Participants receive inhaled LAM-001 or placebo added to their standard immunosuppression to treat bronchiolitis obliterans syndrome after lung transplantation.

Regular visits during the 48-week treatment period

Trial Site Locations

Total: 1 location

1

University of California, San Francisco

San Francisco, California, United States, 94143

Actively Recruiting

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Research Team

S

Steven Hays, MD

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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Frequently Asked Questions

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Published Research Related To This Trial

Rapamycin prevents bronchiolitis obliterans through increasing infiltration of regulatory B cells in a murine tracheal transplantation model.

Yunge Zhao, Jacob R Gillen, Akshaya K Meher...

https://pubmed.ncbi.nlm.nih.gov/26481278

Rapamune (RAPA, rapamycin, sirolimus): mechanism of action immunosuppressive effect results from blockade of signal transduction and inhibition of cell cycle progression.

S N Sehgal

https://pubmed.ncbi.nlm.nih.gov/9721431

Selective Effects of mTOR Inhibitor Sirolimus on Naïve and CMV-Specific T Cells Extending Its Applicable Range Beyond Immunosuppression.

Szilvia Bak, Sabine Tischer, Anna Dragon...

https://pubmed.ncbi.nlm.nih.gov/30619313

Preliminary characterization of oral lesions associated with inhibitors of mammalian target of rapamycin in cancer patients.

Stephen Sonis, Nathaniel Treister, Sant Chawla...

https://pubmed.ncbi.nlm.nih.gov/19862817