Trial Outcomes & Findings for Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia (NCT NCT01362595)

Last Updated: 2022-12-02

Results Overview

The primary outcome is the type of response observed at 9 months. Response to treatment can be one of the following: 1. Complete response (CR): Hb \> 9 gm/dL and transfusion-independence as defined in DBA 2. Partial response (PR): Hb \< 9 gm/dL and increased reticulocyte count to greater than baseline. 3. No response (NR): no change in transfusion requirements and no significant change reticulocyte count from baseline 4. Progression: worsening of disease as defined by the need for more frequent transfusions

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

55 participants

Primary outcome timeframe

9 Months

Results posted on

2022-12-02

Participant Flow

55 subjects were screened and of those 12 did not meet eligibility criteria

Participant milestones

Participant milestones
Measure	Leucine No alternative treatment arm leucine: Dosage of leucine will be dependent on body surface area (BSA): leucine 700 mg/m2/dose by mouth three times a day
Overall Study STARTED	55
Overall Study COMPLETED	43
Overall Study NOT COMPLETED	12

Reasons for withdrawal

Reasons for withdrawal
Measure	Leucine No alternative treatment arm leucine: Dosage of leucine will be dependent on body surface area (BSA): leucine 700 mg/m2/dose by mouth three times a day
Overall Study Screen failure	10
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Leucine n=43 Participants No alternative treatment arm leucine: Dosage of leucine will be dependent on body surface area (BSA): leucine 700 mg/m2/dose by mouth three times a day
Age, Categorical <=18 years	34 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	10.4 years n=5 Participants
Sex: Female, Male Female	22 Participants n=5 Participants
Sex: Female, Male Male	21 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	29 Participants n=5 Participants
Race (NIH/OMB) More than one race	12 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	43 participants n=5 Participants

PRIMARY outcome

Timeframe: 9 Months

Outcome measures

Outcome measures
Measure	Leucine n=43 Participants No alternative treatment arm leucine: Dosage of leucine will be dependent on body surface area (BSA): leucine 700 mg/m2/dose by mouth three times a day
Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia Complete Hematologic Response	2 Participants
Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia Partial hematologic response	5 Participants
Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia No hematologic response	36 Participants
Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia Progression	0 Participants

SECONDARY outcome

Timeframe: 9 months

Adverse events occurring while participants were on Leucine

Outcome measures

Outcome measures
Measure	Leucine n=43 Participants No alternative treatment arm leucine: Dosage of leucine will be dependent on body surface area (BSA): leucine 700 mg/m2/dose by mouth three times a day
Severe Adverse Events Attributable to Leucine No Adverse events	41 Participants
Severe Adverse Events Attributable to Leucine Withdrew early	2 Participants

Adverse Events

Leucine

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Adrianna Vlachos

Feinstein Institutes for Medical Research

Phone: 516-562-1504

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place