A Study to Investigate the Safety, Tolerability, Efficacy, Pharmacokinetics, And Pharmacodynamics of Bitopertin (RO4917838) in Adults with Non Transfusion Dependent ß-Thalassemia.
- Conditions
- ß-ThalassemiaMedDRA version: 20.0 Level: LLT Classification code 10074356 Term: Non-transfusion dependent thalassemia System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2016-004799-23-IT
- Lead Sponsor
- F. HOFFMANN - LA ROCHE LTD.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 12
• Males and females 18 to 55 years of age inclusive (at screening)
• For women of childbearing potential: agreement to remain abstinent or to use two adequate methods of contraception, including at least one method with a failure rate of < 1% per year, during the treatment period and for at least 28 days after the last dose of study drug
• Confirmed diagnosis of ß-thalassemia
• Patients with clinically defined NTD anemia, defined as:
- Patients with mean Hb concentrations of >7.5 g/dL and <9.5 g/dL in two consecutive measurements (one performed during screening and the other within one day prior to Day 1)
- Having received <=4 transfusions of RBC units within one year prior to study enrollment and no transfusion within 12 weeks prior to study enrollment.
Are the trial subjects under 18? no
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 24
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Inability to meet study requirements
- Participation in any investigational study or taken an investigational drug within 90 days (or 5 times the half-life of the investigational drug, whichever is longer). In addition, participation in two or more studies with an experimental drug within 5 months prior to screening.
- Any history of gene therapy
- History of hemolytic anemia except for ß-thalassemia
- Severe symptomatic splenomegaly and/or hepatomegaly with hypersplenism
- Use of an erythropoiesis-stimulating agent <=24 weeks prior to randomization
- Iron chelation therapy, if initiated <=24 weeks prior to randomization
- Hydroxyurea treatment, if initiated <=24 weeks prior to randomization
- Patients with depressive symptoms, major depression, bipolar, obsessive compulsive, schizoaffective disorders, dementia, delirium, diagnosis of mental retardation, severe organic brain syndrome, and patients who in the Investigator’s judgment are at significant risk of suicide or violent behavior
- Patients with any history of depressive episodes or treatment with antidepressants
- Concomitant disease, condition or laboratory abnormality (as suggested by ALT, AST value of >4x upper limit of normal, creatinine clearance based upon the Cockcroft-Gault formula of <30 mL/min) at screening that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would, in the opinion of the investigator, pose an unacceptable risk to the subject in this study
- Clinically significant ECG abnormality at screening, including sinus bradycardia, atrial fibrillation, 2nd or 3rd degree atrioventricular block, prolonged QTc, history of congenital long QT syndromes, or risk of Torsades de Pointes because of family history of sudden death
- Positive result on the serum pregnancy test or breastfeeding at screening, or intend to become pregnant during the course of the trial
- Any inhibitor of CYP3A4 taken within 2 weeks (or within 5-times the elimination half life, whichever is longer) prior to dosing
- Taking any nutrients known to modulate CYP3A activity within 2 weeks prior to dosing
- Any inducer of CYP3A4 taken within 4 weeks (or within 5-times the elimination half life, whichever is longer) prior to dosing
- Suspicion of regular consumption of drugs of abuse
- Positive urine test for drugs of abuse or alcohol at screening or baseline visit according to local law
- Evidence of active hepatitis C virus infection, or active infectious hepatitis B, or known positive human immunodeficiency virus test
- Clinically-significant cardiovascular, neurologic, gastrointestinal, renal, hepatic, broncho pulmonary, neurological, psychiatric, other hematological and endocrine (i.e., pancreatic) diseases not related to thalassemia considered by the Investigator as not adequately controlled prior to Day 1, or cirrhosis
- Diagnosis of cancer within the past 5 years prior to baseline unless treatment has resulted in complete freedom from disease for at least 2 years
- Uncontrolled allergic disease or history of anaphylactic reaction
- Pulmonary hypertension re
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method