Current Clinical Trials and Studies
A major part of The LAM Foundation’s mission is supporting and promoting promising research that leads to an improved understanding of LAM biology and helps develop novel treatment options. As a rare disease, patient engagement and enrollment is critical to the successful and timely conduct of LAM studies. The following is a summary of the currently open LAM research studies with information about the study premise, main details, and study team contact(s) to inquire about more details and to refer patients.
Tissue Donation – National Disease Research Interchange (NDRI)
National Disease Research Interchange (NDRI) NDRI’s Private Donor Program gives individuals and their families an opportunity to provide an invaluable resource for researchers working to discover and advance new treatments or cures. A variety of tissue samples from women with LAM, including lung, kidney, uterus, blood and chyle fluid are currently needed by LAM researchers.
Contact info:
If you are having surgery and would be willing to make a tissue donation or would like to learn more about the tissue donation process, please contact Patient Services at patientservices@thelamfoundation.org or (513) 777-6889.
Menstrual Variation in LAM
Principal Investigator: Nishant Gupta, MD at the University of Cincinnati
This study aims to evaluate the impact of hormonal variations during the menstrual cycle on LAM symptoms and lung function. Participants will receive a small home spirometry device and ovulation test kits. You will be asked to keep a calendar of your menstrual cycle, perform home spirometry, and complete a short symptom and quality-of-life questionnaire twice per week. You will perform these activities from home for 7 menstrual cycles. You may be eligible if you:
- Are premenopausal
- Not currently taking hormonal therapy
- You are not currently pregnant and do not plan to become pregnant in the next 12 months.
- You have not had a hysterectomy, uterine ablation, or have had both ovaries removed.
Contact info:
Contact the lead study coordinator, Emillie Hoh, for more information at hohek@ucmail.uc.edu.
LAM Patient Needs Assessment and Research Priorities Survey (LAM PREP)
The LAM Foundation seeks to better understand LAM patients’ health needs and research priorities. The LAM Patient Needs Assessment, and Research Priorities Survey (LAM-PREP) will allow the LAM Foundation (The LAM Foundation) to support scientific projects that can maximally benefit LAM patients and compare them to the scientific and research priorities of LAM professionals and The LAM Foundation. This organized, formal, and systematic approach will guide the development and implementation a comprehensive plan for LAM research priorities to allocate resources and improve the health outcomes of those affected by LAM.
LAM community participation will occur between June and December 2023 via focus groups and an online survey. Information will be forthcoming via email and social media.
Contact info:
Contact The LAM Foundation at patientservices@thelamfoundation.org or call 513-777-6889 for more information.
NIH/NHLBI Trials:
Discovery of Sirolimus Sensitive Biomarkers in Blood
- NHLBI Protocol number: 18-H-0003
- Joel Moss, MD, PhD, National Institutes of Health
This study is an observational study designed to determine if blood and urine markers after 1 dose and again after 3 months can be used to evaluate the correct dose of sirolimus for people with LAM. Women ages 18-90 with LAM whose doctors have decided they should start taking a 2 mg dose of sirolimus to treat it and who are able to travel to NIH are eligible to participate.
Pathogenesis of Lymphangioleiomyomatosis (LAM)
- NHLBI Protocol number: 95-H-1086
- Joel Moss, MD, PhD, National Institutes of Health
This study is looking at the relationship between lymphatic disorders and gene variations found in the lungs, blood, and other parts of the body. Researchers hope this will improve our understanding of these types of disorders and how genetic factors affect them. Adult women and men with confirmed LAM and a diagnosis of TSC-LAM or Sporadic LAM are eligible.
Cutaneous Tumorigenesis in Patients with Tuberous Sclerosis
- NHLBI Protocol number: 00-H-0051
- Joel Moss, MD, PhD, National Institutes of Health
This study is investigating what causes multiple skin tumors to develop in people with tuberous sclerosis. To participate in the study, you must an adult woman or man, with definite or possible TSC, based on clinical and/or genetic criteria.
Genetic Factors in the Pathogenesis of Lung Disease
- NHLBI Protocol number: 96-H-0100
- Joel Moss, MD, PhD, National Institutes of Health
This study will investigate the genes involved in the breathing process and in the development of lung diseases such as asthma or sarcoidosis to improve understanding of the role they play. Adult women and men with lung disease (includes healthy volunteers) are eligible.
Bronchodilator Effects of Nebulized Versus Inhaled Albuterol in Subjects With Lymphangioleiomyomatosis
- NHLBI Protocol number: 13-H-0051
- Joel Moss, MD, PhD, National Institutes of Health
LAM is a progressive lung disease that usually strikes women during their childbearing years, usually between the onset of puberty and menopause. Doctors at the National Institutes of Health (NIH) are conducting a research study to compare two methods, albuterol (study drug) given in metered dose inhaler and sprayer, and to determine which of these two methods best improves lung function in women with LAM. Adult women who have impaired lung function because of LAM are eligible.
For all NIH/NHLBI trials:
If you are interested in learning more, please contact:
Tat’Yana Worthy, RN, MSN
Research Nurse Specialist, National Institutes of Health
(301)-496-3632 (o)
TAT’YANA WORTHY, RN MSNStudy site: Bethesda, MD
Visit https://go.usa.gov/xuuz9 for more details.