idiopathic pleuroparenchymal fibroelastosis (IPPFE)
Conditions
Interventions
Off-label use of anamorelin for patients with IPPFE (Idiopathic Pleuroparenchymal Fibroelastosis)
Sponsors
Sato Masaaki
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1) Individuals diagnosed with idiopathic pleuroparenchymal fibroelastosis (IPPFE) based on radiological findings (CT) or pathological findings from surgical lung biopsy. (2) Individuals who are scheduled to be registered for lung transplantation from a brain-dead donor as IPPFE. (3) Age: Individuals aged >=18 years at the time of consent. (4) Sex: Not limited. (5) Individuals who have received a sufficient explanation about this study, understood it adequately, and provided written informed consent of their own free will. (6) Individuals who are able to visit the outpatient clinic according to the study schedule.
Exclusion criteria
Exclusion criteria: (1) Individuals with congestive heart failure, myocardial infarction or angina pectoris, or severe cardiac conduction disorders (e.g., complete atrioventricular block). (2) Individuals with moderate or severe hepatic dysfunction (Child-Pugh classification B or C). (3) Individuals with severe hepatic, renal, or cardiovascular disorders (based on Grade 3 of JCOG version of CTCAE v5.0). (4) Individuals with uncontrolled diabetes mellitus. (5) Individuals receiving clarithromycin, itraconazole, voriconazole, ritonavir, cobicistat, or ensitrelvir. (6) Individuals who have difficulty eating due to organic abnormalities of the gastrointestinal tract. (7) Individuals with a history of hypersensitivity to anamorelin. (8) Individuals with concomitant malignant tumors. (9) Individuals with a history of severe drug allergies such as anaphylactic shock, or a history of severe adverse drug reactions. (10) Individuals who participated in a clinical trial or study and took investigational drugs within 4 weeks prior to informed consent. (11) Individuals who are pregnant or may be pregnant. (12) Individuals who are breastfeeding. (13) Others deemed inappropriate for participation in the study by the principal investigator or sub-investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in body weight from baseline at Week 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| (1) Change in body weight from baseline at weeks 3, 6, and 9 of treatment (2) Percent change in body weight from baseline at weeks 3, 6, 9, and 12 of treatment (3) Percent change in activities of daily living (ADL) score from baseline at weeks 3, 6, 9, and 12 of treatment (4) Change in modified medical research council (mMRC) dyspnea scale at weeks 6 and 12 of treatment (5) Change in 6-minute walk distance at week 12 of treatment (6) Change in pulmonary function at week 12 of treatment (7) Percent change in IGF-1 and prealbumin from baseline at weeks 3, 6, 9, and 12 of treatment (8) Frequency and proportion of adverse events | — |
Contacts
Public ContactToyokawa Gouji
The University of Tokyo Hospital
Outcome results
None listed