Phase 1b Study to Assess LAM-001 in Female Subjects with LAM
Research type
Research Study
Full title
A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of LAM-001 in Patients with Lymphangioleiomyomatosis
IRAS ID
241881
Contact name
Tim Harrison
Contact email
Sponsor organisation
LAM Therapeutics
Eudract number
2017-005071-33
Duration of Study in the UK
0 years, 5 months, 8 days
Research summary
This is a clinical research study of a potential new treatment called LAM-001 in participants with lymphanioleiomyomatosis (LAM).\n\nLAM is a rare disease of the lungs affecting women, which may require longterm treatment. Symptoms include fatigue, shortness of breath, chest pain, coughing up blood and fluid retention in the lymph system and/or lungs.\n\nSymptoms of LAM are currently treated with bronchodilators, oxygen and sometimes surgery. LAM-oo1 is an inhaled mTOR inhibitor which is being tested in this study to determine if the drug is safe, how well participants can tolerate the study drug and assess how much of the inhaled drug the body absorbs. This study will include a minimum of 6 participants from 4 centres in the UK and the Netherlands. This number could increase to a maximum of 24, based on Safety Review Committee and Sponsor review of information and side effects and drug levels in the blood. Study participation will include a short screening period followed by either two weeks of treatment (Period 1) or four weeks of treatment (Period 2) and a four week follow up period. In Part 1, participants will receive LAM-001 daily for 14 days, and will be followed up after 28 days. If the Safety Review Committee’s approves, patients may continue to receive LAM-001 for 12 weeks in Part 2 of the study. \n\nThe study is sponsored by LAM Therapeutics.
REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
18/EE/0062
Date of REC Opinion
5 Apr 2018
REC opinion
Further Information Favourable Opinion