MedPath

Alpha-lipoic Acid Adjunctive Therapy in Schizophrenia

Phase 2
Completed
Conditions
Oxidative Stress
Schizophrenia
Interventions
Drug: Placebo Oral Tablet
Registration Number
NCT03788759
Lead Sponsor
Nucleo De Pesquisa E Desenvolvimento De Medicamentos Da Universidade Federal Do Ceara
Brief Summary

Schizophrenia is a devastating mental disorder with a prevalence of approximately 1% worldwide. While effective in reducing positive symptoms, current treatments have limited effects on cognitive and social cognition/processing deficits of schizophrenia, which are closely linked to real-world dysfunction and lack of socio-occupational integration. There is compelling evidence for impaired antioxidant defense system and inflammatory abnormalities in schizophrenia. A new therapeutic approach to the disease might well be to hinder oxidative damage, inflammation and its clinical sequelae. Alpha-lipoic acid (ALA) is a naturally occurring compound, synthesized in the mitochondria, that is currently approved to treat diabetic neuropathic pain. Drug repurposing is a fast, and cost-effective method that can overcome drug discovery challenges of targeting neuropsychiatric disorders. In a pilot investigation, adjunctive treatment with ALA led to robust improvement in negative and cognitive symptoms of ten patients with schizophrenia. This project aims to investigate the efficacy of ALA as a disease-modifying drug for the treatment of schizophrenia, by improving sociability and cognition, as well as to correlate patients' response with biomarkers that will shed light on the pathophysiology of this complex disease. It comprises 1) a prospective, randomized, double-blind, placebo-controlled trial to evaluate efficacy of ALA to treat cognitive and negative symptoms of patients with schizophrenia and 2) an investigation of changes in biomarkers of oxidative stress in response to adjunctive treatment with ALA. The proposed study could establish a new adjunctive treatment for schizophrenia, recognize a novel pharmacological approach and help unveil the biological basis of the disease.

Detailed Description

The underlying pathogenesis of schizophrenia remains unknown, but aberrant reduction-oxidation has gained increasing support as an hypothesis to help explain the pathophysiology of the disease. Alpha-lipoic acid (ALA) is a naturally occurring antioxidant, essential for the function of different enzymes of mitochondria's oxidative metabolism, that is currently approved to treat diabetic neuropathic pain9. ALA and its reduced form, dihydrolipoic acid (DHLA), have important advantages over other antioxidant agents such as vitamin E and C, partly due to their amphiphilic properties, which confer antioxidant actions in the membrane as well as in the cytosol. A preclinical study conducted in our lab showed that ALA alone and combined with clozapine reverses schizophrenia associated symptoms and pro-oxidant changes induced by ketamine in mice. Before the widespread use of antipsychotics, two studies found that low doses of ALA relieved symptoms in patients with schizophrenia.

More recently, my colleagues and I conducted an open label proof of concept study that provided encouraging evidence that low doses of ALA might be an effective adjunctive treatment for schizophrenia. Based on promising preliminary results, the investigators will now test ALA in a more rigorous placebo-controlled clinical study.

Specific Aim1: To conduct a prospective, randomized, double-blind, placebo-controlled trial to evaluate the efficacy of adjuvant treatment with low doses (100mg) of ALA to treat cognitive and negative symptoms of patients with schizophrenia. The investigators will randomize 50 patients over 4 months.

Specific Aim 2: To quantify changes in biomarkers of oxidative stress in response to adjunctive treatment with ALA. The hypothesis is that changes in these biomarkers will mediate the clinical response to ALA.

Research Plan: To carry out a proof of concept 4-month prospective, randomized, double-blind, controlled trial of alpha-lipoic acid, at doses of 100 mg/day or identical placebo tablets, added to ongoing antipsychotics in 50 stable patients (ages 18-60 years, 25 patients per group) with diagnosis of schizophrenia. The study will be conducted at the Drug Research and Development Center (NPDM), at the Universidade Federal do Ceará, Fortaleza, Brazil. This center has a long history of performing placebocontrolled trials in clinical medicine (http://www.npdm.ufc.br/) and has the necessary infrastructure to successfully complete the proposed study protocol. All participants will give written informed consent prior to study enrollment.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
48
Inclusion Criteria
  • Capacity to provide informed consent;
  • Schizophrenia diagnosis (made by research psychiatrists using the Structured Clinical Interview, SCID-5, for Diagnostic and Statistical Manual of Mental Disorders);
  • Negative and/or cognitive symptoms despite adequate antipsychotic treatment;
  • Ages 18-60 years
Exclusion Criteria
  • 6-month history of any drug or alcohol abuse or dependence;
  • Changes in psychotropic medications within the last 4 weeks;
  • Actual valproate use (potential interaction with ALA);
  • General medical illness including autoimmune disorders, known chronic infections such as HIV or hepatitis C, and liver or renal failure that could adversely impact on patient outcome;
  • Women who are planning to become pregnant, are pregnant, or are breastfeeding.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Placebo groupPlacebo Oral Tablet25 subjects will be randomized to placebo.
Experimental groupAlpha-lipoic acid25 subjects will be randomized to 100mg of alpha-lipoic acid.
Primary Outcome Measures
NameTimeMethod
Change in the Brief Psychiatry Rating Scale (BPRS) scoresBaseline and 16 weeks

18-item rating scale to assess changes in psychopathology; each item is scored 0-6, yielding a total between 0 and 108.

Secondary Outcome Measures
NameTimeMethod
Brain resting state activityBaseline and 16 weeks

Functional Magnetic Resonance Imaging (fMRI) scans before and after treatment

Change in plasma Aspartate Aminotransferase (AST)Baseline and 16 weeks

AST in U/L

Change in Neutrophil Count (NC)Baseline and 16 weeks

NC in number per microliter

Change in serum level of Interleukin-4Baseline and 16 weeks

IL-4 in pg/mL

Change in serum level of Thiobarbituric acid reactive substances (TBARS)Baseline and 16 weeks

TBARS in mmol of malonaldehyde/mL

Gut Microbiota CompositionBaseline and 16 weeks

Analyses of patient's gut microbiota

Change in Hemoglobin concentration (HC)Baseline and 16 weeks

HC in g/dL

Change in Glycohemoglobin (HbA1c)Baseline and 16 weeks

HbA1c in %

Change in serum level of EotaxinBaseline and 16 weeks

Eotaxin in ng/mL

Change in serum level of IsoprostanesBaseline and 16 weeks

Isoprostanes in pg/mL

Rey Auditory Verbal Learning TestBaseline and 16 weeks

Participants are asked to repeat list of 15 unrelated words; another list of 15 unrelated words are given and participants must again repeat the original list of 15 words and then again after 30 minutes. Score range: 0-15

Change in Body Mass Index (BMI)Baseline and 16 weeks

Weight and height will be combined to report BMI in kg/m\^2

Change in Abdominal CircumferenceBaseline and 16 weeks

Abdominal Circumference in cm

Change in serum level of NitriteBaseline and 16 weeks

Nitrite in nanomole/mililiter

Change in serum level of Tumor necrosis factor alpha (TNF-α)Baseline and 16 weeks

TNF-α in pg/mL

Change in serum level of TryptophanBaseline and 16 weeks

Tryptophan in micrograms/mL

F-A-S testBaseline and 16 weeks

It assesses phonemic fluency by requesting an individual to orally produce as many words as possible that begin with the letters F, A, and S within a prescribed time frame, usually 1 min.

Change in the Simpson-Angus Extrapyramidal Symptoms Scale (SAS) scoresBaseline and 16 weeks

10-item rating scale to assess extrapyramidal symptoms; each item is scored 0-4, yielding a total between 0 and 40.

Change in White blood cell count (WBC)Baseline and 16 weeks

WBC in number per microliter

Change in serum level of Folic AcidBaseline and 16 weeks

Folic Acid in ng/mL

Change in serum level of Interleukin 1 β (IL-1β)Baseline and 16 weeks

IL-1β in pg/mL

Change in serum level of SerotoninBaseline and 16 weeks

Serotonin in ng/mL

Change in Block Corsi TestBaseline and 16 weeks

This test assesses visuo-spatial short term working memory. Participants are asked to mimick a researcher as he/she taps a sequence of up to nine identical spatially separated blocks. The test measures both the number of correct sequences and the longest sequence remembered.

Category (Animal) FluencyBaseline and 16 weeks

Participants have to produce as many words as possible from a category in a given time (usually 60 seconds). Performance measure is the total number of words

Change in plasma Aspartate Aminotransferase (AST) Alanine Aminotransferase (ALT)Baseline and 16 weeks

ALT in U/L

Change in Hematocrit (Ht)Baseline and 16 weeks

Ht in %

Change in Platelet Count (PC)Baseline and 16 weeks

PC in number per microliter

Change in serum level of Vitamin B12Baseline and 16 weeks

Vitamin B12 in pg/mL

Change in Plasma Glutathione (GSH)Baseline and 16 weeks

GSH in ng/mL

Change in serum level of Interferon gamma (IFNγ)Baseline and 16 weeks

IFNγ in pg/mL

Change in Trail Making TestBaseline and 16 weeks

Trail Making Test measured in time and number of errors. It tests visual attention and task switching. It consists of two parts in which the subject is instructed to connect a set of 25 dots as quickly as possible while still maintaining accuracy. Provide information about visual search speed, scanning, speed of processing, mental flexibility, executive functioning.

Change in Indoleamine 2,3-dioxygenase (IDO) enzymatic activityBaseline and 16 weeks

IDO activity in U IDO mol\^-1/mg\^-1

Change in serum level of CalprotectinBaseline and 16 weeks

Serum Calprotectin in ng/mL

Change in Subtest Digit SpanBaseline and 16 weeks

Individual tries to repeat digits forward, backward, and in ascending order. This test measures short term memory, working memory. The score is the maximum number of digits correctly remembered.

Trial Locations

Locations (1)

Núcleo de Pesquisa e Desenvolvimento de Medicamentos - UFC

🇧🇷

Fortaleza, CE, Brazil

© Copyright 2025. All Rights Reserved by MedPath