What side effects are associated with this type of intervention?

Common treatments for Diamond-Blackfan Anemia (DBA) include corticosteroids, blood transfusions, and hematopoietic stem cell transplantation. Corticosteroids can cause weight gain, hypertension, diabetes, osteoporosis, and increased infection risk. Blood transfusions may lead to iron overload, causing organ damage if not managed with chelation therapy. Hematopoietic stem cell transplantation risks include graft-versus-host disease and infections. Bitopertin, a Glycine Transporter 1 Inhibitor, is a newer treatment under investigation, and its specific side effects are not well-documented but may include gastrointestinal or neurological issues.

What prior FDA approvals exist?

Diamond-Blackfan Anemia (DBA) is typically treated with corticosteroids and red blood cell transfusions. For patients who do not respond to these treatments, hematopoietic stem cell transplantation may be considered. While Bitopertin, a Glycine Transporter 1 Inhibitor, is currently being studied, there is no specific mention of FDA-approved drugs in this class for DBA. Other investigational treatments and supportive therapies continue to be explored to improve patient outcomes.

What other types of research exist?

Promising novel alternatives to Bitopertin for Diamond-Blackfan Anemia patients in 2024 include gene therapy approaches targeting the genetic mutations responsible for DBA, and new erythropoiesis-stimulating agents that enhance red blood cell production. Additionally, advancements in CRISPR-Cas9 technology for gene editing and the development of small molecule drugs that can correct ribosomal protein defects are also being explored as potential treatments.

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GlyT1 Inhibitor

Bitopertin for Diamond-Blackfan Anemia

Phase 1 & 2

Recruiting

Led By David J Young, M.D.

Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up ongoing for up to 3 years, beginning 8 months (32 weeks) from drug initiation

Awards & highlights

No Placebo-Only Group

Summary

This trial tests bitopertin, a daily pill, in adults with Diamond-Blackfan anemia. The drug aims to reduce harmful levels of heme in the blood, which can help manage the disease. Participants will take the drug for several months, with periodic check-ups to monitor their response. Bitopertin has shown promise in improving anemia in a mouse model.

Who is the study for?

Adults aged 18+ with Diamond-Blackfan anemia (DBA), particularly those who have chronic severe anemia or are dependent on red blood cell transfusions. It's for patients whose DBA is steroid-refractory, meaning steroids don't work well anymore, or they can't tolerate them. Participants must not be at risk of suicide, have certain heart conditions, active infections poorly controlled by therapy, or a history of specific malignancies.

What is being tested?

The trial tests Bitopertin, a pill taken daily for 8 months to treat DBA. Dosage may increase over time based on response. The study includes initial screening with physical exams and blood tests followed by regular check-ups every four weeks through clinic visits or telehealth options. Successful participants may continue in an extended phase for up to three additional years.

What are the potential side effects?

While the side effects of Bitopertin aren't detailed here, common considerations include potential allergic reactions to the drug's components and general risks associated with taking new medications which could range from mild symptoms like nausea to more serious issues depending on individual health factors.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ ongoing for up to 3 years, beginning 8 months (32 weeks) from drug initiation

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~ongoing for up to 3 years, beginning 8 months (32 weeks) from drug initiation

This trial's timeline: 3 weeks for screening, Varies for treatment, and ongoing for up to 3 years, beginning 8 months (32 weeks) from drug initiation for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Rate of response

Secondary study objectives

Clonal evolution on bitopertin

Health-related quality of life (HRQL)

Long-term safety of drug

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Experimental -BitopertinExperimental Treatment1 Intervention

Bitopertin, up to a maximum dose of 60 mg (5 mg, 10mg, 20mg, 40mg, 60mg)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Bitopertin

2012

Completed Phase 3

~740

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Diamond-Blackfan Anemia (DBA) is typically treated with corticosteroids, which stimulate red blood cell production, and blood transfusions to manage anemia. Corticosteroids work by increasing erythropoiesis, the process of producing red blood cells, which is crucial for DBA patients who have a deficiency in this area. Blood transfusions provide immediate relief by supplying healthy red blood cells. Bitopertin, a GlyT1 inhibitor, is being studied as it may offer a novel approach by modulating glycine levels, potentially improving erythropoiesis and reducing the need for transfusions. Understanding these mechanisms is vital for DBA patients as it helps in managing chronic anemia and improving quality of life.

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