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Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS)

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ClinicalTrials.gov Identifier: NCT00605657
Recruitment Status : Completed
First Posted : January 31, 2008
Results First Posted : March 21, 2013
Last Update Posted : March 21, 2013
Sponsor:
Information provided by (Responsible Party):
Koneti Rao, National Institute of Allergy and Infectious Diseases (NIAID)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions ALPS
Hypersplenism
Lymphadenopathy
Interventions Drug: Valproic Acid
Procedure: CT Scan
Procedure: Blood Sample
Enrollment 6

Recruitment Details All patients were started on valproic acid at the NIH Clinical Center then followed locally
Pre-assignment Details  
Arm/Group Title Valproic Acid
Hide Arm/Group Description valproic acid administered starting at 10 mg/kg/day in divided doses twice daily x 3 days, increase to 15mg/kg/day x 3 days, increase to 20 mg/kg/day on day 7, increase to 30 mg/kg/day on day 14, maximum dose of 40mg/kg/day on day 21, if tolerated, to achieve plasma level 50-100 mcg/mL.
Period Title: Overall Study
Started 5
Completed 3
Not Completed 2
Reason Not Completed
Adverse Event             1
Protocol Violation             1
Arm/Group Title Valproic Acid
Hide Arm/Group Description valproic acid administered starting at 10 mg/kg/day in divided doses twice daily x 3 days, increase to 15mg/kg/day x 3 days, increase to 20 mg/kg/day on day 7, increase to 30 mg/kg/day on day 14, maximum dose of 40mg/kg/day on day 21, if tolerated, to achieve plasma level 50-100 mcg/mL.
Overall Number of Baseline Participants 5
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
<=18 years
4
  80.0%
Between 18 and 65 years
1
  20.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female
1
  20.0%
Male
4
  80.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 5 participants
5
1.Primary Outcome
Title Number of Participants With Response
Hide Description Reduction of lymph node and/or spleen size measured by CT imaging, or physical exam and abdominal ultrasound. A clinical response is defined as a greater than 40% reduction in lymph node size and/or greater than 40% reduction in spleen size. A CT scan with contrast measured lymph node size as well as spleen size.
Time Frame 3 monthly (12 week) intervals
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Valproic Acid
Hide Arm/Group Description:
valproic acid administered starting at 10 mg/kg/day in divided doses twice daily x 3 days, increase to 15mg/kg/day x 3 days, increase to 20 mg/kg/day on day 7, increase to 30 mg/kg/day on day 14, maximum dose of 40mg/kg/day on day 21, if tolerated, to achieve plasma level 50-100 mcg/mL.
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
0
2.Secondary Outcome
Title To Determine Whether the Treatment Alters, in Favorable Directions, Laboratory Markers of ALPS (e.g., Number of DNT Cells, Immunoglobin Levels, Vitamin B12 Levels, IL-10 Levels, Autoantibody Titers, Fas Mediated Apoptosis)
Hide Description [Not Specified]
Time Frame 3 monthly (12 week) intervals
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
outcome measure not assessed because 0 participants had a response
Arm/Group Title Valproic Acid
Hide Arm/Group Description:
valproic acid administered starting at 10 mg/kg/day in divided doses twice daily x 3 days, increase to 15mg/kg/day x 3 days, increase to 20 mg/kg/day on day 7, increase to 30 mg/kg/day on day 14, maximum dose of 40mg/kg/day on day 21, if tolerated, to achieve plasma level 50-100 mcg/mL.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Valproic Acid
Hide Arm/Group Description valproic acid administered starting at 10 mg/kg/day in divided doses twice daily x 3 days, increase to 15mg/kg/day x 3 days, increase to 20 mg/kg/day on day 7, increase to 30 mg/kg/day on day 14, maximum dose of 40mg/kg/day on day 21, if tolerated, to achieve plasma level 50-100 mcg/mL.
All-Cause Mortality
Valproic Acid
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Valproic Acid
Affected / at Risk (%) # Events
Total   1/5 (20.00%)    
Blood and lymphatic system disorders   
low serum IgG  1 [1]  1/5 (20.00%)  1
Respiratory, thoracic and mediastinal disorders   
pneumomediastinum * 1 [2]  1/5 (20.00%)  1
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, No source vocabulary
[1]
possibly related to treatment
[2]
not treatment related
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Valproic Acid
Affected / at Risk (%) # Events
Total   5/5 (100.00%)    
Blood and lymphatic system disorders   
decreased platelets  [1]  3/5 (60.00%)  3
decreased hemoglobin  [2]  3/5 (60.00%)  3
decreased neutrophil count  [3]  4/5 (80.00%)  4
leukopenia  [4]  2/5 (40.00%)  2
elevated alkaline phosphatase  [5]  1/5 (20.00%)  1
Gastrointestinal disorders   
vomiting * [6]  1/5 (20.00%)  1
elevated AST  [7]  2/5 (40.00%)  2
elevated ALT  [8]  1/5 (20.00%)  1
diarrhea  [9]  1/5 (20.00%)  1
gastroenteritis  [9]  1/5 (20.00%)  1
abdominal pain  [9]  1/5 (20.00%)  1
nausea  [9]  1/5 (20.00%)  1
General disorders   
fever  1 [5]  2/5 (40.00%)  2
hyperglycemia  [5]  1/5 (20.00%)  1
fatigue  [10]  5/5 (100.00%)  5
rhinorrhea  [5]  1/5 (20.00%)  1
Respiratory, thoracic and mediastinal disorders   
cough * [5]  2/5 (40.00%)  2
sinus congestion * [5]  2/5 (40.00%)  2
Skin and subcutaneous tissue disorders   
pruritis * [8]  1/5 (20.00%)  1
rash  [8]  2/5 (40.00%)  2
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, No source vocabulary
[1]
Only probably related in one patient. Other patients had baseline low platelets.
[2]
unlikely or unrelated in all patients
[3]
unlikely or unrelated in all cases
[4]
unlikely or unrelated
[5]
unrelated
[6]
vomiting unrelated, due to the flu in one case. Possibly related in other
[7]
one probably related and one unlikely related
[8]
probably related
[9]
possibly related
[10]
not related in two cases, possibly related in one case.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: V. Koneti Rao, MD
Organization: NIAID
Phone: 301-496-6502
Responsible Party: Koneti Rao, National Institute of Allergy and Infectious Diseases (NIAID)
ClinicalTrials.gov Identifier: NCT00605657     History of Changes
Other Study ID Numbers: 080053
08-I-0053 ( Other Grant/Funding Number: DIR NIAID )
First Submitted: January 15, 2008
First Posted: January 31, 2008
Results First Submitted: February 14, 2013
Results First Posted: March 21, 2013
Last Update Posted: March 21, 2013