Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Danielle Townsley, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT01193283
First received: August 31, 2010
Last updated: October 13, 2015
Last verified: October 2015
Results First Received: October 13, 2015  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Aplastic Anemia
Neutropenia
Pancytopenia
Severe Aplastic Anemia
Interventions: Drug: Cyclophosphamide
Drug: Cyclosporine

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
SAA Hematologic Response

Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.

Cyclophosphamide: 30 my/kg for 4 days

Cyclosporine: daily to a trough of 100 t0 200 ng/ml


Participant Flow:   Overall Study
    SAA Hematologic Response
STARTED   22 
COMPLETED   21 
NOT COMPLETED   1 
Death                1 



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
SAA Hematologic Response

Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.

Cyclophosphamide: 30 my/kg for 4 days

Cyclosporine: daily to a trough of 100 t0 200 ng/ml


Baseline Measures
   SAA Hematologic Response 
Overall Participants Analyzed 
[Units: Participants]
 22 
Age 
[Units: Participants]
 
<=18 years   4 
Between 18 and 65 years   16 
>=65 years   2 
Gender 
[Units: Participants]
 
Female   7 
Male   15 
Ethnicity (NIH/OMB) 
[Units: Participants]
 
Hispanic or Latino   8 
Not Hispanic or Latino   14 
Unknown or Not Reported   0 
Region of Enrollment 
[Units: Participants]
 
United States   22 


  Outcome Measures

1.  Primary:   The Primary Objective is to Evaluate the Safety and Activity Profile of Cyclophosphamide and Cyclosporine in Severe Aplastic Anemia (SAA) Patients.   [ Time Frame: 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Danielle Townsley
Organization: NIH NHLBI
phone: 301-402-3477
e-mail: townsleydm@nhlbi.nih.gov


Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Danielle Townsley, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT01193283     History of Changes
Other Study ID Numbers: 100176
10-H-0176 ( Other Identifier: NIH NHLBI )
Study First Received: August 31, 2010
Results First Received: October 13, 2015
Last Updated: October 13, 2015
Health Authority: United States: Food and Drug Administration
United States: Federal Government