Acetazolamide Trial in Normal Pressure Hydrocephalus

• Status: Not yet recruiting
• Trial end date: 2024-08-01
• Target enrollment: 0
• Participant gender: All

Summary

A pharmacological treatment to patients with normal pressure hydrocephalus (NPH) is missing. The aim is to investigate if acetazolamide given to patients with NPH improves gait function and study the pathophysiological mechanisms leading to reduced symptoms.

Patients will be randomized to acetazolamide or placebo and duration of treatment will be from diagnosis to the day of shunt surgery. Target dose is 500 mg/day. Study design is a double-blind randomized controlled trial and the plan is to include 42-50 patients. The study is investigator-initiated without financial sponsorship from the industry.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Johan Virhammar
• Collaborators: Swedish Society for Medical Research; Uppsala University
• Treatments: Acetazolamide

Criteria

Inclusion Criteria:

1. Diagnosis of idiopathic normal pressure hydrocephalus according to international guidelines

2. Age ≥ 50 years and ≤ 82 years

3. Cognitive function with Mini-Mental State Examination > 20 points

4. MRI image characteristic of normal pressure hydrocephalus, defined as callosal angle < 90 degrees, tight high convexity, dilated Sylvian fissures, and presence of periventricular white matter hyperintensities

5. Signed informed consent form

Exclusion Criteria:

1. Exclusion criteria for MRI examination

2. Participation in another medical trial

3. Other disease likely to impact the symptoms of the patient

4. Wheelchair user or unable to walk without support

5. Reduced kidney function with GFR < 50

6. Reduced liver function (increased INR or alanine transaminase concentrations in plasma)

7. Known heart failure

8. Low concentrations of electrolytes in blood plasma that, according to the investigator, prevents participation in the study

9. Angle-closure glaucoma

10. Allergy to acetazolamide, sulfonamides, or sulfonamide derivatives

11. Treatment with phenytoin, valproate, carbamazepine, lithium, oral anticoagulants, thiazide-diuretics, or acetylsalicylic acid > 100 mg/day

12. Inability to swallow capsules of the same size as the investigational medicinal products (will be tested using empty capsules when the patient is asked to participate in the study)

13. Average walking time for the three walking tests < 11 seconds

14. Average walking time for the three walking tests > 50 seconds

15. Inability to comply with the study treatment independently, and a concurrent lack of individuals to help the patient comply with the treatment during the study period

16. Fertile woman without highly effective contraception. A Fertile woman will be tested with pregnancy test before inclusion is possible.