Trial Outcomes & Findings for Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia (NCT NCT05828108)
NCT ID: NCT05828108
Last Updated: 2026-06-30
Results Overview
Response was defined as either an increase in hemoglobin or a decrease in transfusion rate. Robust response was defined as achievement of transfusion independence.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
15 participants
Primary outcome timeframe
Up to 32 Weeks
Results posted on
2026-06-30
Participant Flow
Participants who complete Stage 1 of the clinical trial and achieve a predefined response will be eligible for enrollment in the extension phase.
Participant milestones
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
|---|---|
|
Stage 1: Week 0 to Week 32
STARTED
|
15
|
|
Stage 1: Week 0 to Week 32
5 mg Bitopertin
|
15
|
|
Stage 1: Week 0 to Week 32
10 mg Bitopertin
|
15
|
|
Stage 1: Week 0 to Week 32
20 mg Bitopertin
|
15
|
|
Stage 1: Week 0 to Week 32
40 mg Bitopertin
|
14
|
|
Stage 1: Week 0 to Week 32
60 mg Bitopertin
|
13
|
|
Stage 1: Week 0 to Week 32
COMPLETED
|
12
|
|
Stage 1: Week 0 to Week 32
NOT COMPLETED
|
3
|
|
Stage 2: Extension Phase (Wk 33 to 156)
STARTED
|
0
|
|
Stage 2: Extension Phase (Wk 33 to 156)
COMPLETED
|
0
|
|
Stage 2: Extension Phase (Wk 33 to 156)
NOT COMPLETED
|
0
|
Reasons for withdrawal
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
|---|---|
|
Stage 1: Week 0 to Week 32
Lost to Follow-up
|
1
|
|
Stage 1: Week 0 to Week 32
Physician Decision
|
2
|
Baseline Characteristics
Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia
Baseline characteristics by cohort
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
n=15 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=20 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
15 Participants
n=20 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=20 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=20 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=20 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=20 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=20 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=20 Participants
|
PRIMARY outcome
Timeframe: Up to 32 WeeksPopulation: Three participants were unevaluable due to early discontinuation.
Response was defined as either an increase in hemoglobin or a decrease in transfusion rate. Robust response was defined as achievement of transfusion independence.
Outcome measures
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
n=12 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 10 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 20 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 40 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 60 mg.
|
|---|---|---|---|---|---|
|
Number of Participants With Response
|
0 Participants
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: 12 weeksResponse was defined as either an increase in hemoglobin or a decrease in transfusion rate. Robust response was defined as achievement of transfusion independence.
Outcome measures
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
n=15 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 10 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 20 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 40 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 60 mg.
|
|---|---|---|---|---|---|
|
Response Rate
|
0 Participants
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 32 weeksPopulation: No participants were evaluable, as no participants met criteria for response, therefore no participants were at risk of relapse
Relapse as demonstrated by new or increasing transfusion requirements and/or according to clinical outcome
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 32 WeeksPopulation: Three participants were unevaluable due to early discontinuation.
Intra-patient dose escalation occurred every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). Tolerability was defined as achieving a maximum tolerated dose of up to 60 mg daily without unacceptable toxicity or intolerance.
Outcome measures
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
n=12 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 10 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 20 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 40 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 60 mg.
|
|---|---|---|---|---|---|
|
Number of Participants Tolerating Study Drug
|
11 Participants
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 32 WeeksPopulation: 2 participants withdrew from study due to non-treatment related adverse events
Safety was assessed by the number and severity of treatment-related adverse events at each dose level using the Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0. Adverse events were graded as follows: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe or medically significant), Grade 4 (life-threatening), and Grade 5 (death related to adverse event).
Outcome measures
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
n=15 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
n=15 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 10 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
n=15 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 20 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
n=14 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 40 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
n=13 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 60 mg.
|
|---|---|---|---|---|---|
|
Number of Treatment-Related Adverse Events (AEs), Including Serious Adverse Events (SAEs) by Dose Levels
Grade 2
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
1 Number of Adverse Events
|
|
Number of Treatment-Related Adverse Events (AEs), Including Serious Adverse Events (SAEs) by Dose Levels
Grade 3
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
|
Number of Treatment-Related Adverse Events (AEs), Including Serious Adverse Events (SAEs) by Dose Levels
Grade 5
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
|
Number of Treatment-Related Adverse Events (AEs), Including Serious Adverse Events (SAEs) by Dose Levels
Grade 4
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
SECONDARY outcome
Timeframe: Up to 32 weeksPopulation: Three participants were unevaluable due to early discontinuation.
Rates of clonal evolution on bitopertin as measured by karyotypic, histologic, and flow cytometric changes
Outcome measures
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
n=12 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 10 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 20 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 40 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 60 mg.
|
|---|---|---|---|---|---|
|
Rate of Clonal Evolution
|
0 Participants
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: 32 WeeksRate of overall survival according to clinical outcomes. Overall survival is defined as the number of participants alive following initiation of treatment.
Outcome measures
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin
n=15 Participants
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation every 4 weeks (5 mg, 10 mg, 20 mg, 40 mg, up to a maximum of 60 mg). If response criteria are met after 8 weeks at a given dose level, that dose will be designated the minimum effective dose (MED) and continued for the remainder of the study unless dose modification is clinically indicated.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 10 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 20 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 40 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 60 mg.
|
|---|---|---|---|---|---|
|
Number of Participants Surviving
|
15 participants
|
—
|
—
|
—
|
—
|
Adverse Events
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 5 mg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
Serious events: 4 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 5 mg
n=15 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 5 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
n=15 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 10 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
n=15 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 20 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
n=14 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 40 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
n=13 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 60 mg.
|
|---|---|---|---|---|---|
|
Infections and infestations
Endocarditis infective
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Cardiac disorders
Pulmonary valve disease
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Nervous system disorders
Syncope
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
6.7%
1/15 • Number of events 2 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/13 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
Other adverse events
| Measure |
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 5 mg
n=15 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 5 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 10 mg
n=15 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 10 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 20 mg
n=15 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 20 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 40 mg
n=14 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 40 mg.
|
Steroid Refractory Diamond-Blackfan Anemia in Bitopertin at 60 mg
n=13 participants at risk
Participants with refractory Diamond-Blackfan Anemia will receive oral bitopertin once daily with dose escalation to 60 mg.
|
|---|---|---|---|---|---|
|
Infections and infestations
Pharyngitis
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Nervous system disorders
Dizziness
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/15 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
0.00%
0/14 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
7.7%
1/13 • Number of events 1 • Up to 32 weeks
Adverse events (AEs) will start being recorded after informed consent has been obtained until 7 (for non-serious AEs) or 30 days (for Serious Adverse Events \[SAEs\]) after administration of the last dose of study drug or until study termination until adequate resolution or stabilization. At each study visit, the investigator will inquire about the occurrence of AE/SAEs since the last visit.
|
Additional Information
David J. Young, MD, PhD
The National Heart, Lung, and Blood Institute (NHLBI)
Phone: 301.827.7823
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place