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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
Study Summary
This trial will assess the efficacy of bitopertin to treat Diamond-Blackfan anemia (a bone marrow failure syndrome) by rebalancing heme and globin chain production. Safety, response rate, relapse rate, clonal evolution, survival, and HRQL will be measured.
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.Check my eligibility
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.See study design
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
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Rate of response
Secondary outcome measures
Clonal evolution on bitopertin
Health-related quality of life (HRQL)
Long-term safety of drug
+4 moreTrial 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
Find a Location
Who is running the clinical trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,837 Previous Clinical Trials
47,851,564 Total Patients Enrolled
David J Young, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
1 Previous Clinical Trials
15 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I was diagnosed with Diamond-Blackfan anemia before age 3, have low red blood cell counts, and either a family history or a confirmed gene mutation.I do not have any severe illnesses that would prevent me from following the treatment plan.I am a woman who can have children and am not using strong birth control methods.I am able to understand the study details and give informed consent.I cannot take pills.I was diagnosed with DBA after age 3 and have a confirmed gene mutation.I have severe anemia or needed a blood transfusion recently.I am 18 years old or older.I will be tested for gene mutations linked to DBA as I don't have a molecular diagnosis.I have been diagnosed with MDS, AML, or another active cancer.My condition worsened after or didn't respond to steroid treatment, or I can't tolerate steroids.I have a serious heart condition.I have an infection that isn't getting better with treatment.I have recently undergone chemotherapy or radiation therapy.I haven't taken androgens or corticosteroids in the last 4 weeks.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Does this research require the participation of individuals aged seventy-five and over?
"This clinical trial is open to those above the age of majority, but not exceeding 100 years old."
Answered by AI
What types of individuals make ideal candidates for this research trial?
"Patients with diamond-blackfan anemia and ages between 18 to 100 are eligible for this trial. Approximately 30 individuals will be chosen in total."
Answered by AI
Are there any available spots for participants in this experiment?
"Clinicaltrials.gov data confirms that this trial, which was initially advertised on May 2nd 2023 and has not been updated since April 26th 2023, is no longer actively recruiting participants. Fortunately, there are many other trials currently in search of volunteers for various medical studies."
Answered by AI
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