Trial Outcomes & Findings for Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia (NCT NCT01231841)

Last Updated: 2023-06-22

Results Overview

Patients will be classified as responders if they have transfusion independence and meet two of the following three criteria: ANC greater than 500/mm3; platelet count greater than 20,000/mm3; and reticulocyte count greater than 40,000/mm3. Transfusion independence is defined as no need for transfusions for one month prior to response assessment.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

At 6 months

Results posted on

2023-06-22

Participant Flow

Patients with severe aplastic anemia were recruited over a period of four years (2005-2009) in the hematology clinic at the Cleveland Clinic, a hospital in Cleveland, Ohio.

Participant milestones

Participant milestones
Measure	Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) Patients received rabbit anti-thymocyte globulin IV daily over 4-24 hours on days 1-5 (3.5 mg/kg/day). Beginning on day 6, patients received oral cyclosporine twice daily (5 mg/kg/day) for a period of 6 months and then tapered.
Overall Study STARTED	20
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	rATG n=20 Participants Patients receive anti-thymocyte globulin IV daily over 4-24 hours on days 1-5. Beginning on day 6, patients receive oral cyclosporine twice daily for 6 months followed by a taper. Treatment continues in the absence of disease progression or unacceptable toxicity
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=5 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants
Age, Continuous	51.95 years STANDARD_DEVIATION 18.43 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: At 6 months

Outcome measures

Outcome measures
Measure	Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) n=20 Participants Patients received rabbit anti-thymocyte globulin IV daily over 4-24 hours on days 1-5 (3.5 mg/kg/day). Beginning on day 6, patients received oral cyclosporine twice daily (5 mg/kg/day) for a period of 6 months and then tapered.
Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months	9 participants

Adverse Events

rATG

Serious events: 11 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	rATG n=20 participants at risk Patients receive anti-thymocyte globulin IV daily over 4-24 hours on days 1-5. Beginning on day 6, patients receive oral cyclosporine twice daily for 6 months followed by a taper. Treatment continues in the absence of disease progression or unacceptable toxicity
Musculoskeletal and connective tissue disorders chest pain	10.0% 2/20 • Number of events 2 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
Infections and infestations colitis	10.0% 2/20 • Number of events 2 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
Musculoskeletal and connective tissue disorders compression fractures	5.0% 1/20 • Number of events 1 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
Blood and lymphatic system disorders febrile neutropenia	15.0% 3/20 • Number of events 4 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
Gastrointestinal disorders gastrointestinal bleed	5.0% 1/20 • Number of events 1 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
General disorders headache	5.0% 1/20 • Number of events 1 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
Infections and infestations pneumonia	5.0% 1/20 • Number of events 2 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
Infections and infestations sepsis	20.0% 4/20 • Number of events 4 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
Immune system disorders serum sickness	10.0% 2/20 • Number of events 2 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.
Infections and infestations urinary tract infection	5.0% 1/20 • Number of events 2 The endpoints of this study evaluated response only. As such, data on non-serious adverse events were not collected or reported for any patients on this trial. Data regarding serious adverse events were collected and reported according to FDA and IRB policies and regulations.

Other adverse events

Adverse event data not reported

Additional Information

Jaroslaw Maciejewski, MD PhD, Principal Investigator

Cleveland Clinic Foundation

Phone: 216-445-5962

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place