Trial Outcomes & Findings for Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment (NCT NCT00944749)

Last Updated: 2021-03-23

Results Overview

The primary endpoint was hematologic response at 3 months, defined as no longer meeting criteria for Severe Aplastic Anemia (SAA) defined as bone marrow cellularity of less than 30% and severe pancytopenia with at least two of the following peripheral blood count criteria: (i) absolute neutrophil count less than 0.5×109/L; (ii) absolute reticulocyte count less than 60×109/L; and (iii) platelet count less than 20×109/L. A complete response was defined as absolute neutrophil count (ANC) above 1.0×109/L, Hgb \> 10 g/dL, and platelet count \> 100×109/L. A partial response was defined as a hematologic response that was not sufficient for a complete response.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

23 participants

Primary outcome timeframe

3-months

Results posted on

2021-03-23

Participant Flow

Participant milestones

Participant milestones
Measure	Single Arm Subjects were given a second course of immunosuppression with h-ATG/CsA in subjects' refractory to or with a suboptimal response to a course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment
Overall Study STARTED	23
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm n=23 Participants Subjects were given a second course of immunosuppression with h-ATG/CsA in subjects' refractory to or with a suboptimal response to a course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment
Age, Categorical <=18 years	6 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 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	6 Participants n=5 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	9 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3-months

Outcome measures

Outcome measures
Measure	Single Arm n=22 Participants Subjects were given a second course of immunosuppression with h-ATG/CsA in subjects' refractory to or with a suboptimal response to a course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment
Number of Participants With Complete Response at 3 Months.	0 Participants

PRIMARY outcome

Timeframe: 3 months

The primary endpoint was hematologic response at 3 months, defined as no longer meeting criteria for Severe Aplastic Anemia (SAA). A complete response was defined as absolute neutrophil count (ANC) above 1.0×109/L, Hgb \> 10 g/dL, and platelet count \> 100×109/L. A partial response was defined as a hematologic response that was not sufficient for a complete response. In subjects with a non-robust hematologic response at 3 months, improvement in one or more of the listed peripheral blood parameter (a,b,c) were recorded as a response: a) ANC - if baseline ANC below 0.5×109/L, increase in ANC by \> 0.3×109/L, if baseline ANC above 0.5×109/L, any increase in ANC by \> 0.5×109/L of blood; (b) platelets - if baseline platelet count \< 50×109/L, any increase in platelet count by \> 20×109/L of blood; c) hemoglobin - any increase in hemoglobin by 1.5 g/dl of blood in transfusion-independent patients and in absolute reticulocyte count to \> 60×109/L of blood in transfusion-dependent patients.

Outcome measures

Outcome measures
Measure	Single Arm n=22 Participants Subjects were given a second course of immunosuppression with h-ATG/CsA in subjects' refractory to or with a suboptimal response to a course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment
Number of Participants With Complete Response at 3 Months.	2 Participants

Adverse Events

Single Arm

Serious events: 14 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Single Arm n=23 participants at risk Subjects were given a second course of immunosuppression with h-ATG/CsA in subjects' refractory to or with a suboptimal response to a course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment
Blood and lymphatic system disorders Hospitalization - Neutropenic fever	30.4% 7/23
Infections and infestations Hospitalization - Infection/ Febrile neutropenia	8.7% 2/23
Blood and lymphatic system disorders Hospitalization - Hemorrhage/ bleeding	4.3% 1/23
Blood and lymphatic system disorders Death - Hemorrhage/ bleeding	4.3% 1/23
Infections and infestations Hospitalization - Infection	39.1% 9/23
Gastrointestinal disorders Hospitalization - Gastrointestinal	4.3% 1/23

Other adverse events

Other adverse events
Measure	Single Arm n=23 participants at risk Subjects were given a second course of immunosuppression with h-ATG/CsA in subjects' refractory to or with a suboptimal response to a course of r-ATG/CsA or cyclophosphamide at least 3 months post-treatment
Infections and infestations Rhinorrhea	4.3% 1/23 • Number of events 1

Additional Information

Dr Danielle Townsley

National Heart Lung and Blood Institute

Phone: 301-402-3477

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place