Trial Outcomes & Findings for Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease (NCT NCT01054768)

Results Overview

C-Reactive Protein (CRP) was measured by a clinical laboratory. Measuring the quantity of serum CRP is helpful in recognizing inflammatory states.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

37 participants

Primary outcome timeframe

6 months

Results posted on

2021-08-03

Participant Flow

First subject was enrolled 08/10/09. Length of subject involvement was 7 months. Each subject was seen once a month at Children's Hospital \& Research Center Oakland

Exclusions included those with 3 packed red blood transfusions in the past year, illness that could contribute to inflammation, acute sickle cell disease related symptoms requiring a hospital visit, those pregnant or breast feeding, or in other investigational drug or device studies, participants on hydroxyurea or a regular transfusion therapy.

Participant milestones

Participant milestones
Measure	Alpha-lipoic Acid and Acetyl-L-carnitine alpha-lipoic acid and acetyl-L-carnitine 1400 mg tablet twice a day for 6 months. alpha-lipoic acid and acetyl-L-carnitine: none to report	Placebo 1400 mg placebo tablet twice a day for 6 months. Placebo: none to report
Overall Study STARTED	17	20
Overall Study COMPLETED	9	17
Overall Study NOT COMPLETED	8	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Alpha-lipoic Acid and Acetyl-L-carnitine n=17 Participants alpha-lipoic acid and acetyl-L-carnitine 1400 mg tablet twice a day for 6 months. alpha-lipoic acid and acetyl-L-carnitine: none to report	Placebo n=20 Participants 1400 mg placebo tablet twice a day for 6 months. Placebo: none to report	Total n=37 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=5 Participants	16 Participants n=7 Participants	32 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	8 Participants n=7 Participants	15 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	12 Participants n=7 Participants	22 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: Reason for discontinuation: started transfusions (2), alternative treatment (2), adverse events (4), subject preference (3).

C-Reactive Protein (CRP) was measured by a clinical laboratory. Measuring the quantity of serum CRP is helpful in recognizing inflammatory states.

Outcome measures

Outcome measures
Measure	Alpha-lipoic Acid and Acetyl-L-carnitine n=9 Participants alpha-lipoic acid and acetyl-L-carnitine 1400 mg tablet twice a day for 6 months. alpha-lipoic acid and acetyl-L-carnitine: none to report	Placebo n=17 Participants 1400 mg placebo tablet twice a day for 6 months. Placebo: none to report
C-Reactive Protein	11.4 mg/l Standard Deviation 20.9	6.8 mg/l Standard Deviation 10.3

Adverse Events

Alpha-lipoic Acid and Acetyl-L-carnitine

Serious events: 8 serious events

Other events: 6 other events

Deaths: 0 deaths

Placebo

Serious events: 9 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Alpha-lipoic Acid and Acetyl-L-carnitine n=17 participants at risk alpha-lipoic acid and acetyl-L-carnitine 1400 mg tablet twice a day for 6 months. alpha-lipoic acid and acetyl-L-carnitine: none to report	Placebo n=20 participants at risk 1400 mg placebo tablet twice a day for 6 months. Placebo: none to report
Blood and lymphatic system disorders Vaso-occlusive pain	35.3% 6/17 • Number of events 13 • 2 years	45.0% 9/20 • Number of events 13 • 2 years
Respiratory, thoracic and mediastinal disorders Pneumonia/Acute Chest Syndrome	11.8% 2/17 • Number of events 2 • 2 years	0.00% 0/20 • 2 years

Other adverse events

Other adverse events
Measure	Alpha-lipoic Acid and Acetyl-L-carnitine n=17 participants at risk alpha-lipoic acid and acetyl-L-carnitine 1400 mg tablet twice a day for 6 months. alpha-lipoic acid and acetyl-L-carnitine: none to report	Placebo n=20 participants at risk 1400 mg placebo tablet twice a day for 6 months. Placebo: none to report
Respiratory, thoracic and mediastinal disorders Respiratory Symptoms	17.6% 3/17 • Number of events 3 • 2 years	45.0% 9/20 • Number of events 12 • 2 years
Gastrointestinal disorders Nausea	11.8% 2/17 • Number of events 2 • 2 years	35.0% 7/20 • Number of events 7 • 2 years
Renal and urinary disorders Urinary Urgency/UTI	11.8% 2/17 • Number of events 2 • 2 years	25.0% 5/20 • Number of events 6 • 2 years

Additional Information

Ashutosh Lal, MD

Children's Hospital & Research Center at Oakland

Phone: 5104283172

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place