Acetazolamide for Treatment Resistant Schizophrenia

NCT04887792 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: STUDY20010237
• Study Type: interventional
• Target: 60
• Locations: 2 countries
• Sites: 2

Brief Summary

This is a double blind adjunctive randomized controlled trial for schizophrenia using acetazolamide.

Conditions

Schizophrenia; Schizo Affective Disorder

Keywords

Schizophrenia; Schizo Affective Disorder; acetazolamide

Outcome Measures

Primary Outcomes (1)

• Change in positive symptoms

  Clinical Severity as determined by the Positive and Negative Syndrome Scale (PANSS) positive symptom subscale. The PANSS is a standardized, clinical interview that rates the presence and severity of positive and negative symptoms, as well as general psychopathology for people with schizophrenia within the past week. Symptom severity for each item is rated according to which anchoring points in the 7-point scale (1 = absent; 7 = extreme) best describe the presentation of the symptom. 7 Items, (minimum score = 7, maximum score = 49)

  24 weeks

Secondary Outcomes (9)

• Clinical Severity

  24 weeks

• Clinical Severity

  24 weeks

• Cognition

  24 weeks

• Clinical Severity

  24 weeks

• Social Function

  24 weeks

Study Arms (2)

Acetazolamide

ACTIVE COMPARATOR

acetazolamide capsules

Drug: Acetazolamide

Placebo

ACTIVE COMPARATOR

Identical gelatin capsules

Drug: Placebo

Interventions

Acetazolamide DRUG

ACZ 250 mg/day in gelatin capsules will be administered initially and increased over 7-10 days to 2g/day.

Also known as: Diamox

Acetazolamide

Placebo DRUG

Identical gelatin capsules will be prepared by filling with inert excipients.

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Written informed consent.
• Both genders, ages 18-55 years (older patients may not tolerate high ACZ dose).
• PANSS total score \> 60 and Score \> 4 on one or more items of the 'positive' syndrome items (P1-P7), following treatment at therapeutic doses for 6 weeks with different APDs on 2 occasions.
• Stable dose of antipsychotic drug (APD) for \> 1 month, continued throughout the study.
• Not participating in another randomized controlled clinical trial (RCT).

You may not qualify if:

• Substance abuse in the past month/dependence past 6 months with the exception of methadone prescribed for opiate withdrawal.
• History or current medical/neurological illnesses that may lead to unstable course, e.g., epilepsy.
• Pregnancy.
• Acetazolamide (ACZ) contraindications: hypersensitivity to ACZ; history of renal hyperchloremic acidosis; Addison's disease/adrenal failure; chronic closed angle-closure glaucoma.
• Current or prior treatment with ACZ or history of hypersensitivity to ACZ.
• Intellectual disability as defined in DSM 5.

Sponsors & Collaborators

• Vishwajit Nimgaonkar, MD PhD: lead
• Stanley Medical Research Institute: collaborator

Study Sites (2)

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

St John's Medical College Hospital

Bangalore, India

RECRUITING

MeSH Terms

Conditions

Schizophrenia; Psychotic Disorders

Interventions

Acetazolamide

Condition Hierarchy (Ancestors)

Schizophrenia Spectrum and Other Psychotic Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Thiadiazoles; Thiazoles; Sulfur Compounds; Organic Chemicals; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Vishwajit L Nimgaonkar, M.D., Ph.D.

  University of Pittsburgh

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Vishwajit L Nimgaonkar, M.D., Ph.D.

CONTACT

412-246-6356; vishwajitNL@upmc.edu

Maribeth A Wesesky, BPS

CONTACT

4122957017; weseskyma@upmc.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Participant, care providers, Investigators and assessors will all be blinded. Study staff responsible for randomization will be unblinded.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Psychiatry and Human Genetics

Model Details: This study will be a randomized placebo-controlled sequential parallel comparison design (SPCD).

Study Record Dates

• First Submitted: December 15, 2020
• First Posted: May 14, 2021
• Study Start: February 1, 2022
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 30, 2026
• Last Updated: April 3, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); IN (1)