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Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers

This study has been completed.
Sponsor:
Collaborators:
Boston Medical Center
University of Illinois at Chicago
Icahn School of Medicine at Mount Sinai
University of Tennessee Health Science Center
Schneider Children's Medical Center, Israel
Information provided by (Responsible Party):
Susan P. Perrine, Boston University
ClinicalTrials.gov Identifier:
NCT00004412
First received: October 18, 1999
Last updated: March 18, 2015
Last verified: March 2015
Results First Received: July 22, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Skin Ulcers
Sickle Cell Anemia
Interventions: Drug: Arginine Butyrate
Other: Standard local care dressing

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Study began 1998, contacted physicians at several medical centers with patients under their care for SCD with refractory leg ulcers. Electronic materials describing the study and its sponsor were transmitted to medical centers.

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
Standard Local Care Dressing Standard local care includng cleaning, saline irrigation, dressing changes only for 8 weeks twice a week.
Arginine Butyrate Arginine Butyrate IV plus Standard local care dressing for a total of 12 weeks. Low dose 250 mg/kg or, increased dose 750 mg/kg. First week AB given 5 days in a row, over 8 to 12hours.
Crossover Patients are randomly assigned (following a table of random numbers prepared by a blinded statistician) between two arms of the study. Arm I is Allevyn Hydrophilic PU dressing (standard local care) only, and Arm II is standard local care plus Arginine Butyrate (AB), the Investigational New Drug. Ulcers observed & traced weekly. Ulcer area calculated by computerized planimetry. After 12 weeks of therapy, if the ulcer size decreased by at least 25%, the AB may be continued for another 8 weeks (twice), or until the ulcer closes, plus an additional 2 weeks. The patients randomized to the Control Arm ( standard local care) were given the option of crossing over to Arm II If, ulcers did not close after 8 weeks of standard local care.

Participant Flow for 2 periods

Period 1:   Randomized Period
    Standard Local Care Dressing     Arginine Butyrate     Crossover  
STARTED     11     12     0  
COMPLETED     8     12     0  
NOT COMPLETED     3     0     0  
Moved to Crossover Period                 3                 0                 0  

Period 2:   Crossover Period
    Standard Local Care Dressing     Arginine Butyrate     Crossover  
STARTED     8 [1]   12 [2]   3 [3]
COMPLETED     8 [4]   12 [5]   3 [6]
NOT COMPLETED     0     0     0  
[1] Eight subjects were randomized to Standard of Care dressing Arm
[2] Twelve patients were randomized to the Arginine Butyrate plus Standard local care dressing Arm
[3] Three of Randomized Period's Arm I elected to move to Crossover Period's Crossover Arm.
[4] Eight subjects in this arm completed study without crossing over.
[5] Twelve subjects in this arm completed the study.
[6] Three subjects, from Arm I, who crossed- over to Arm II completed the study.



  Baseline Characteristics
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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
Standard Local Care Dressing Standard local care dressing including cleaning, saline irrigation, dressing changes only for 8 weeks twice a week.
Arginine Butyrate Arginine Butyrate IV plus Standard local care dressing for a total of 12 weeks. Low dose 250 mg/kg or, increased dose 750 mg/kg. First week AB given 5 days in a row, over 8 to 12hours.
Crossover Patients are randomly assigned (following a table of random numbers prepared by a blinded statistician) between two arms of the study. Arm I is Standard local care dressing only, and Arm II is standard local care plus Arginine Butyrate (AB), the Investigational New Drug. Ulcers observed & traced weekly. Ulcer area calculated by computerized planimetry. After 12 weeks of therapy, if the ulcer size decreased by at least 25%, the AB may be continued for another 8 weeks (twice), or until the ulcer closes, plus an additional 2 weeks. The patients randomized to the Control Arm ( standard local care) were given the option of crossing over to Arm II If, ulcers did not close after 8 weeks of standard local care.
Total Total of all reporting groups

Baseline Measures
    Standard Local Care Dressing     Arginine Butyrate     Crossover     Total  
Number of Participants  
[units: participants]
  8     12     3     23  
Age  
[units: participants]
       
<=18 years     0     0     0     0  
Between 18 and 65 years     8     12     3     23  
>=65 years     0     0     0     0  
Age  
[units: years]
Mean (Standard Deviation)
  31  (12.57478)     33  (9.14985)     31  (2.3094)     32  (1.1547)  
Gender  
[units: participants]
       
Female     5     5     1     11  
Male     3     7     2     12  
Region of Enrollment  
[units: participants]
       
United States     8     12     2     22  
Israel     0     0     1     1  



  Outcome Measures
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1.  Primary:   Healing Defined as a Decrease in Ulcer Area by at Least 25% of the Initial Area   [ Time Frame: participants were followed for an average of 3 months ]

2.  Secondary:   % Ulcers Which Completely Healed in Each Group, After 3 Months   [ Time Frame: two additional courses of 8 week cycles ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Douglas V. Faller, Ph.D., M.D., Susan P. Perrine, M.D.
Organization: Boston University School of Medicine
phone: (617) 638-5639
e-mail: sperrine@bu.edu


Publications of Results:

Responsible Party: Susan P. Perrine, Boston University
ClinicalTrials.gov Identifier: NCT00004412     History of Changes
Other Study ID Numbers: 199/13302
BUSM-FDR001376 ( Other Identifier: Food & Drug Administration )
IND 36,957 ( Other Identifier: Food & Drug Administration )
Study First Received: October 18, 1999
Results First Received: July 22, 2013
Last Updated: March 18, 2015
Health Authority: United States: Food and Drug Administration