A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-10-26

Study Completion Date

2018-06-29

Brief Summary

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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-thalassemia.

This study consists of two parts:

Part 1 - The main study - 16 weeks in total: Participants will undergo a 6-week dose-escalation period followed by 10 weeks of treatment at the attained target dose.

Part 2 - Open Label Extension (OLE) - up to an additional 12 months. Participants will be given the option to enroll into the OLE once the 16-week treatment of Part 1 has been completed. Participants who decide not to enroll in the OLE, at the end of Part 1 will enter a 6-week follow-up period.

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Detailed Description

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Conditions

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Beta-Thalassemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Bitopertin

Part 1 - The main study - 16 weeks in total:

Participants will undergo a 6-week dose-escalation period followed by 10 weeks of treatment at the attained target dose of bitopertin.

Part 2 - Open Label Extension (OLE) - up to an additional 12 months:

Participants will be given the option to enroll into the OLE once the 16-week treatment of Part 1 has been completed.

Participants who decide not to enroll in the OLE, at the end of Part 1 will enter a 6-week follow-up period.

Group Type EXPERIMENTAL

Bitopertin

Intervention Type DRUG

Bitopertin will be administered orally once daily at doses up to 120 milligrams (mg).

Interventions

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Bitopertin

Bitopertin will be administered orally once daily at doses up to 120 milligrams (mg).

Intervention Type DRUG

Other Intervention Names

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RO4917838

Eligibility Criteria

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Inclusion Criteria

* 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 Criteria

* 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)

Minimum Eligible Age

18 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No