A personalized medicine approach for beta-thalassemia transfusion dependent patients: testing SIROLIMUS in a first pilot clinical trial. - SIRTHALACLI

Rare Partners s.r.l. Impresa Sociale0 sites20 target enrollmentAugust 3, 2018

Overview

No summary available.

Registry

who.int

Start Date

August 3, 2018

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

Rare Partners s.r.l. Impresa Sociale

•1\.Responsive to the in vitro (ErPCs) treatment with Sirolimus according to laboratory specific definition (\= 20% increase of HbF in comparison with samples not treated with Sirolimus)
•2\.AST or ALT \< 3x ULN (Upper Limit of Normal)
•3\.Documented Platelet count \>150\.000/ul
•4\.Fertile women using an adequate system of contraception
•5\.Patients willing to follow all the study requirements and perform all the study visits and to cooperate with the investigator
•6\.Selection criteria must still be fulfilled at the time of the inclusion visit
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1\.2 Adults (18\-64 years) yes
•F.1\.2\.1 Number of subjects for this age range 20

•1\.Uncontrolled hypertension defined as systolic pressure (PA) \= 140 mm Hg or diastolic pressure \= 90 mm Hg.
•2\.Class 3 or higher heart failure (New York Heart Association, NYHA).
•3\.QTc\> 450 msec on selection ECG.
•4\.White blood cell \[WBC] count \<3000 cells per µL and/or Granulocytes \<1500/mm3
•5\.Total cholesterol \> 240 mg/dl
•6\.Triglycerides \> 200 mg/dl
•7\.Proteinuria with urinary protein \>1g/24 hrs;
•8\.Any active infection requiring parenteral antibiotic therapy within 28 days prior to inclusion or oral antibiotics within 14 days prior to inclusion;
•9\.Enrolment into one other drug or device trial since selection;
•10\.Major surgery (including splenectomy) performed after the study selection visit .