Treatment of beta-thalassemia patients with rapamycin (sirolimus): from pre-clinical research to a clinical trial

Phase 1

• Conditions: Beta-thalassemia transfusion dependent patients; MedDRA version: 20.0Level: PTClassification code 10043391Term: Thalassaemia betaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2018-001469-18-IT
• Lead Sponsor: Dipartimento di Scienze della Vita e Biotecnologie dell'Università degli Studi di Ferrara

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-05-20
• Last Update Posted: 17 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

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.Patient willing to follow all the study requirements and perform all the study visits and to cooperate with the investigator;
5.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 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

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;
11.Ongoing treatment with drugs and agents that could increase the concentration of sirolimus in the blood including: ciclosporin, bromocriptine, cimetidine, cisapride, clotrimazole, danazol, diltiazem, fluconazole, protease inhibitors (eg for HIV and hepatitis C which require pharmacological treatment with ritonavir, indinavir, boceprevir and telaprevir), metoclopramide, nicardipine, troleandomycin, verapamil;
12.Ongoing treatment with drugs and agents that could reduce the concentration of sirolimus including carbamazepine, phenobarbital, phenytoin, rifapentine, St. John's wort (Hypericum perforatum);
13.Cytotoxic agents, systemic corticosteroids, immunosuppressants or anticoagulant therapy such as warfarin or heparin within 28 days before inclusion (prophylactic aspirin up to 100 mg / day is allowed);
14.Macrolidic antibiotics (clarithromycin).
15.Occurrence of an event which leads to the appearance of a non-selection criteria.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified