Status:
COMPLETED
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia
Lead Sponsor:
Hoffmann-La Roche
Conditions:
Beta-Thalassemia
Eligibility:
All Genders
18-55 years
Phase:
PHASE2
Brief Summary
This proof-of-mechanism study is being performed to investigate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of multiple oral doses of bitopertin in adults with NTD beta-...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of beta-thalassemia
- Clinically defined non-transfusion-dependent anemia (Part 1 only), defined as Hb concentrations \>7.5 grams per deciliter (g/dL) and \<9.5 g/dL, less than or equal to 4 transfusions of red blood cell units within 1 year prior to study enrollment, and no transfusion within 12 weeks prior to study enrollment
- Completion of 16 weeks of treatment with bitopertin in Part 1 of this study with more than 80% compliance from expected use of study medication (based on patient diary and study drug accountability; Part 2 only)
- A favorable benefit-risk ratio from treatment with bitopertin as assessed by the Investigator (Part 2 only)
Exclusion
- Any history of gene therapy
- History of hemolytic anemia except for beta-thalassemia
- Severe symptomatic splenomegaly and/or hepatomegaly with hypersplenism (Part 1 only)
- Any use of an erythropoiesis-stimulating agent within 24 weeks prior to enrollment.
- Initiation of iron chelation therapy or hydroxyurea within 24 weeks prior to enrollment (Part 1 only)
- Depression, treatment with anti-depressants, or other psychiatric illnesses and/or drug abuse
- Clinically significant/uncontrolled comorbid disease
- Pregnant or breastfeeding females
- Use of cytochrome P450 (CYP) 3A4 inhibitors within 2 weeks or CYP3A4 inducers within 4 weeks prior to study drug
- Active hepatitis B or C or known positive human immunodeficiency virus (HIV) test result
- Diagnosis of cancer within previous 5 years unless treatment has resulted in complete freedom from disease for at least 2 years
- Any major illness within 1 month or febrile illness within 1 week prior to study drug
- Pulmonary hypertension requiring oxygen therapy (Part 1 only)
Key Trial Info
Start Date :
October 26 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 29 2018
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT03271541
Start Date
October 26 2017
End Date
June 29 2018
Last Update
October 5 2018
Active Locations (4)
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1
Centro della Microcitemia e delle Anemie Congenite - Ospedale Galliera; Oncologia /Cardiologia
Genoa, Liguria, Italy, 16128
2
Ospedale Maggiore di Milano; Cardio-Metabolic Diseases
Milan, Lombardy, Italy, 20122
3
Chronic Care Center
Baabda, Lebanon, 1003
4
Siriraj Hospital; Division of Haematology-Oncology
Bangkok Noi, Thailand, 10700