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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 October 2017
Main ID:  NCT00605657
Date of registration: 15/01/2008
Prospective Registration: No
Primary sponsor: Koneti Rao
Public title: Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS)
Scientific title: Pilot (Phase I-II) Study of Valproic Acid (Depakote) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS)
Date of first enrolment: January 2008
Target sample size: 6
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT00605657
Study type:  Interventional
Study design:   
Phase:  Phase 1/Phase 2
Countries of recruitment
United States
Contacts
Name:     Koneti Rao, MD
Address: 
Telephone:
Email:
Affiliation:  DIR, NIAID, NIH
Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

1. All subjects must fulfill the published criteria for the diagnosis of ALPS
(documented nonmalignant lymphadenopathy and/or splenomegaly of at least 1-year
duration; greater than1% TCR alpha/beta+ CD4-CD8- T cells in the peripheral
blood). This must include clinically documented lymphadenopathy involving more
than 2 nodes in more than 1 regional group of nodes measuring greater than 2cm in
size and/or a palpable spleen.

2. Age greater than or equal to 2 years through less than or equal to 70 years.

3. Must have a personal primary care physician who is willing to follow the protocol
required evaluations during the study period.

4. Must be willing to sign a consent form.

5. Patients on immunosuppression (e.g., corticosteroid, mycophenolate mofetil,
azathioprine, cyclophosphamide) are eligible if the dose of the immunosuppressive
drug has been stable for at least 3 months prior to enrollment and their
hematologic parameters do not meet the exclusion criteria (1) as outlined below.

EXCLUSION CRITERIA:

1. A hemoglobin concentration of less than 8 gm/dL, a platelet count of less than 75
K/mm(3), or an absolute neutrophil count of less than 500/mm(3), at study entry.

2. Liver disease determined by an alanine aminotransferase (ALT), aspartate
aminotransferase (AST), or bilirubin 2.5 times greater than the upper limit of normal.

3. History of pancreatitis by clinical features and/or laboratory abnormalities in the
last 12 months.

4. Renal dysfunction determined by a calculated urine creatinine clearance of less than
70 mL/min/1.73 m(2) in children and less than 60 mL/min in adults, or using the
Schwartz formula or Levy formula based on serum creatinine.

5. Patients clinically suspected of suffering from urea cycle disorders will be excluded.

6. Patients with history of seizure disorders and/or those already receiving valproic
acid will be excluded.

7. Sensitive to or have ever had an allergic reaction to Depakote.

8. Not able to abstain from alcohol during the length of the study.

9. Pregnancy. Female adults and adolescents who have attained menarche must have a
negative pregnancy test at study entry and commit to using an acceptable method of
barrier or hormonal contraception (e.g., condoms, diaphragms, oral contraceptives, or
long acting progestin agents) if sexually active during the study and for 3 months
after the last dose of valproic acid.

10. Lactating mothers who are breast feeding their babies will not be eligible.

11. ALPS patients who have been treated with bone marrow toxic chemotherapy regimens for
Non-Hodgkin's lymphoma or other malignancies are not eligible for this pilot study.

12. Unwilling or unable to comply with the need to have periodic blood tests to monitor
possible side effects of treatment, or other major requirements of this study will be
an exclusion criteria.



Age minimum: 2 Years
Age maximum: 70 Years
Gender: All
Health Condition(s) or Problem(s) studied
Lymphadenopathy
ALPS
Hypersplenism
Intervention(s)
Procedure: Blood Sample
Procedure: CT Scan
Drug: Valproic Acid
Primary Outcome(s)
Number of Participants With Response [Time Frame: 3 monthly (12 week) intervals]
Secondary Outcome(s)
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) [Time Frame: 3 monthly (12 week) intervals]
Secondary ID(s)
08-I-0053
080053
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available: Yes
Date Posted: 21/03/2013
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT00605657
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