Trial Outcomes & Findings for Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS) (NCT NCT00605657)

Last Updated: 2013-03-21

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

6 participants

Primary outcome timeframe

3 monthly (12 week) intervals

Results posted on

2013-03-21

Participant Flow

All patients were started on valproic acid at the NIH Clinical Center then followed locally

Participant milestones

Participant milestones
Measure	Valproic Acid 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 Study STARTED	5
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Valproic Acid 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 Study Adverse Event	1
Overall Study Protocol Violation	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Valproic Acid n=5 Participants 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.
Age, Categorical <=18 years	4 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants

PRIMARY outcome

Timeframe: 3 monthly (12 week) intervals

Outcome measures

Outcome measures
Measure	Valproic Acid n=3 Participants 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.
Number of Participants With Response	0 participants

SECONDARY outcome

Timeframe: 3 monthly (12 week) intervals

Population: outcome measure not assessed because 0 participants had a response

Outcome measures

Outcome data not reported

Adverse Events

Valproic Acid

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Valproic Acid n=5 participants at risk 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.
Respiratory, thoracic and mediastinal disorders pneumomediastinum	20.0% 1/5 • Number of events 1
Blood and lymphatic system disorders low serum IgG	20.0% 1/5 • Number of events 1

Other adverse events

Other adverse events
Measure	Valproic Acid n=5 participants at risk 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.
General disorders fever	40.0% 2/5 • Number of events 2
Blood and lymphatic system disorders decreased platelets	60.0% 3/5 • Number of events 3
Blood and lymphatic system disorders decreased hemoglobin	60.0% 3/5 • Number of events 3
Blood and lymphatic system disorders decreased neutrophil count	80.0% 4/5 • Number of events 4
General disorders hyperglycemia	20.0% 1/5 • Number of events 1
Gastrointestinal disorders vomiting	20.0% 1/5 • Number of events 1
Respiratory, thoracic and mediastinal disorders cough	40.0% 2/5 • Number of events 2
Respiratory, thoracic and mediastinal disorders sinus congestion	40.0% 2/5 • Number of events 2
General disorders fatigue	100.0% 5/5 • Number of events 5
General disorders rhinorrhea	20.0% 1/5 • Number of events 1
Gastrointestinal disorders elevated AST	40.0% 2/5 • Number of events 2
Gastrointestinal disorders elevated ALT	20.0% 1/5 • Number of events 1
Gastrointestinal disorders diarrhea	20.0% 1/5 • Number of events 1
Gastrointestinal disorders gastroenteritis	20.0% 1/5 • Number of events 1
Skin and subcutaneous tissue disorders pruritis	20.0% 1/5 • Number of events 1
Blood and lymphatic system disorders leukopenia	40.0% 2/5 • Number of events 2
Gastrointestinal disorders abdominal pain	20.0% 1/5 • Number of events 1
Gastrointestinal disorders nausea	20.0% 1/5 • Number of events 1
Blood and lymphatic system disorders elevated alkaline phosphatase	20.0% 1/5 • Number of events 1
Skin and subcutaneous tissue disorders rash	40.0% 2/5 • Number of events 2

Additional Information

V. Koneti Rao, MD

NIAID

Phone: 301-496-6502

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place