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The Effect of an Anti-obesity Drug, Semaglutide, As Treatment in New-onset Idiopathic Intracranial Hypertension (IIH) Compared to Standard Weight Management (dietician) with Regards to Change in Weight and Intracranial Pressure

Phase 4
Active, not recruiting
Conditions
Idiopathic Intracranial Hypertension
Intracranial Pressure
Obesity
Pseudotumor Cerebri Syndrome
Papilledema
Weight Loss
Interventions
Drug: Semaglutide
Dietary Supplement: Very Low Calorie Diet
Behavioral: Dietician counselling
Registration Number
NCT06027567
Lead Sponsor
Rigmor Højland Jensen
Brief Summary

50 patients with verified new-onset Idiopathic Intracranial Hypertension are randomly allocated to standard weight management (dietician counselling) or trial intervention consisting of subcutaneous injections with Semaglutide for 10 months combined, in the initial 8 weeks following diagnosis, with a Very Low Calorie-Diet (max 800 kcal/day)

Detailed Description

Idiopathic Intracranial Hypertension is primarily observed in obese female and weight management promotes disease control by yet unsettled mechanisms. Effective, fast and lasting weight loss is crucial, however, hard to achieve. Current weight management strategy in IIH in Denmark is counselling by a dietician. This study investigates whether an initial Very Low Calorie Diet (max 800 kcal/day) for 8 weeks following the diagnosis combined with GLP1-RA treatment throughout 10 months is tolerated and more efficient in achieving substantial weight loss and reduction of intracranial pressure. Furthermore, a number of secondary outcomes are measured including headache burden, quality of life, structure and function of the optic nerve, non-invasive surrogate markers of intracranial pressure, body fat mass, bone health, fatty liver disease and a range of cerebrospinal-, blood- and urine markers of i.a. the hormonal, inflammatory, metabolic, and headache biomarker profile.

The intervention may candidate as a future first-line treatment regime.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
Female
Target Recruitment
50
Inclusion Criteria
  • Confirmed new onset definite IIH with papilledema and lumbar opening pressure ≥25 cm cerebrospinal fluid according to Friedmann diagnostic criteria
  • BMI ≥ 27
  • Use of contraceptive methods with failure rates of less than 1 % throughout the study period for group A and for at least an additional 2 months after cessation of Semaglutide
  • Written, informed consent
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Exclusion Criteria
  • Unable to provide written informed consent or participate
  • Malignant IIH with visual threat that requires surgical intervention, i.e., cerebrospinal fluid diversion (shunting), optic nerve sheet fenestration or cerebral venous sinus stenting
  • Pregnancy or breastfeeding
  • Treatment with antidiabetics, blood-thinners or medication that may increase the risk of adverse events
  • Diabetes, congestive heart failure, severe vascular disease, pancreatitis, severe ophthalmological disorders other than IIH (e.g. retinopathy)
  • History or family history of thyroid carcinomas or Multiple Endocrine Neoplasias (MEN1/MEN2)
  • History of bariatric surgery
  • Known hypersensitivity to any contents of Semaglutide®
  • Other severe/uncontrolled mental or physical disease
Read More

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
SemaglutideVery Low Calorie DietSemaglutide up-titration to 2.4 mg for 10 months initially combined with a Very Low Calorie Diet (800 kcal/day) for 8 weeks. Counselling by dietician Standard medical treatment of intracranial hypertension
SemaglutideDietician counsellingSemaglutide up-titration to 2.4 mg for 10 months initially combined with a Very Low Calorie Diet (800 kcal/day) for 8 weeks. Counselling by dietician Standard medical treatment of intracranial hypertension
Standard care (dietician)Dietician counsellingStandard weight loss intervention Counselling by dietician Standard medical treatment of intracranial hypertension
SemaglutideSemaglutideSemaglutide up-titration to 2.4 mg for 10 months initially combined with a Very Low Calorie Diet (800 kcal/day) for 8 weeks. Counselling by dietician Standard medical treatment of intracranial hypertension
Primary Outcome Measures
NameTimeMethod
Weight8 weeks

Weight change (%)

Intracranial pressure8 weeks

Change in lumbar opening pressure (%)

Secondary Outcome Measures
NameTimeMethod
Weight10 months

Weight change (%)

Remission8 weeks + 10 months

Proportion of patients with abscence of papilledema with or without intracranial pressure \<25 cm cerebrospinal fluid

Change in fat mass8 weeks + 10 months

Change in body fat percentage measured by Dual Energy X-ray Absorptiometry compared to baseline

Change in Papilledema8 weeks + 10 months

Change in Frisén Grade (0-5, 0 minimal, 5 worst)

Total fat massBaseline + 8 weeks + 10 months

Body fat percentage measured by Dual Energy X-ray Absorptiometry

Feasibility8 weeks + 10 months

Drop-out rate (proportion of patients withdrawing from participation)

Need of intracranial pressure-lowering medication_18 weeks + 10 months

Dose (mg) of intracranial pressure-lowering medication needed (Acetazolamide, Topiramate, diuretics)

Fatty liver prevalenceBaseline + 8 weeks + 10 months

Prevalence of non-alcoholic fatty liver disease evaluated by ultrasonography (subjectively assessed density of liver parenchyma compared to hepatic perivascular density and renal density), assessed by an experienced radiologist with specialization in ultrasonography

Monthly headache daysBaseline + 8 weeks + 10 months

Number of days with headache for the past 30 days preceding visit

Headche severityBaseline + 8 weeks + 10 months

Number of days with mild, moderate, and severy headache, respectively, in the past 30 days preceding visit

Intracranial Pressure10 months

Change in lumbar opening pressure (%)

Quality of Life8 weeks + 10 months

Change in total score of Quality of Life (psychological, social, physical, environmental) assessed by the World Health Organization Quality of Life Brief Version Questionnaire (0-100; 0 worst, 100 best)

Peripapillary capillary densityBaseline + 8 weeks + 10 months

Change in peripapillary capillary density (ratio of pixels of perpapillary vessels and pixels in the foveal area evaluated by Optic Coherence Tomography Angiography

Peripapillary artery-to-venule ratioBaseline + 8 weeks + 10 months

Change in peripapillary artery-to-venule diameter ratio measured by confocal Scanning Laser Ophtalmoscopy

Truncal fatBaseline + 8 weeks + 10 months

Change in percentage of truncal adiposity measured by Dual Energy X-ray Absorptiometry

Headache burden measured by HURT questionnaire8 weeks + 10 months

Change in summation of scores in the questionnaire "Headache Under Response to Treatment Questionnaire" (HURT); 0-24 points were higher numbers are worse outcome

Visual fields8 weeks + 10 months

Perimetric mean deviation (decibel) by Humphrey automated perimetry

EDI-OCT8 weeks + 10 months

Change in Papillary thickness (um) measured by Enhanced Depth Imaging Optical Coherence Tomography (EDI-OCT)

Optic disc elevationBaseline + 8 weeks + 10 months

Optic disc elevation (mm) measured by transorbital ultrasonography, (average of 3 scans of each eye with papilledema)

Headache medication - Acute analgesic useBaseline + 8 weeks + 10 months

Number of days with need of acute analgesic treatment for headache

Headache medication - preventive medicationBaseline + 8 weeks + 10 months

Need of preventive medical treatment for headache

Optic nerve sheath diameterBaseline + 8 weeks + 10 months

Optic nerve sheath diameter (mm) measured by transorbital ultrasonography (average of 3 scans of each eye with papilledema)

Android-gynoid-ratioBaseline + 8 weeks and 10 months

Change in ratio of Android versus gynoid fat percentage using Dual Energy X-ray Absorptiometry

Adverse events8 weeks + 10 months

Number of adverse events overall, and sub-categorized into adverse events (AE) (any event happening during attachment to the project) and severe adverse events (SAE) in case of the following conditions: Hospitalization or prolongation of hospitalization, death, life-threatening or significant disability/incapacity

Need of intracranial pressure-lowering medication_2Baseline + 8 weeks + 10 months

Number of patients in need of any intracranial pressure-lowering drug (Acetazolamide, Topiramate, diuretics)

Insulin like-Growth-Factor-1Baseline

Level of Insulin like-Growth-Factor-1 in serum (ug/L) in women not taking estrogen-containing contraceptives.

Insulinlike Growth Factor Binding Protein-3Baseline

Level of Insulinlike Growth Factor Binding Protein-3 in serum (ug/L) in women not taking estrogen-containing contraceptives.

Growth hormoneBaseline

Level of growth hormone in serum ug(L) in women not taking estrogen-containing contraceptives.

LutropinBaseline

Level of Lutropin in serum (IU/L) in women not taking estrogen-containing contraceptives.

FollitropinBaseline

Level of Follitropin in serum (IU/L) in women not taking estrogen-containing contraceptives.

TestosteronBaseline

Level of testosteron in serum (nmol/L) in women not taking estrogen-containing contraceptives.

EstradiolBaseline

Level of estradiol in serum (nmol/L) in women not taking estrogen-containing contraceptives.

Sex-Hormone Binding GlobulinBaseline

Level of Sex-Hormone Binding Globulin in serum (nmol/L) in women not taking estrogen-containing contraceptives.

DehydroepiandrosteroneBaseline

Level of Dehydroepiandrosterone (DHEAS) in serum (umol/L) in women not taking estrogen-containing contraceptives.

Anti-Müllerian HormoneBaseline

Level of Anti-Müllerian Hormone (pmol/L) in serum in women not taking estrogen-containing contraceptives.

AndrostenedionBaseline

Level of androstenedion (nmol/L) in serum in women not taking estrogen-containing contraceptives.

17-hydroxyprogesterone (mg/d)Baseline

Level of 17-hydroxyprogesterone (mg/d) in serum in women not taking estrogen-containing contraceptives.

Cortisol 0 minBaseline

Level of cortisol (nmol/L) in serum in women not taking estrogen-containing contraceptives.

Cortisol 30 minBaseline

Level of cortisol (nmol/L) in serum 30 minutes after stimulation with 0,25 mg SynACHTen in women not taking estrogen-containing contraceptives.

Pituitary adenylate cyclase-activating peptide (PACAP) Pituitary adenylate cyclase-activating peptide Pituitary adenylate cyclase-activating peptideBaseline + 8 weeks + 10 months

Level (picograms per milliliter in plasma and cerebrospinal fluid) of Pituitary adenylate cyclase-activating peptide (PACAP)

Calcitonin Gene Related PeptideBaseline + 8 weeks + 10 months

Calcitonin Gene Related Peptide (CGRP) level pg/mL (picograms per milliliter in plasma and cerebrospinal fluid)

Change in bone marker (CTX)Baseline + 8 weeks + 10 months

Change in carboxy-terminal collagen crosslinks (CTX) level (nanograms per liter)

Change in bone marker (PiNP)baseline + 8 weeks + 10 months

Change in procollagen type I N-propeptide (PiNP) level (micrograms per liter) compared to baseline

Regional bone densityBaseline + 8 weeks + 10 months

Change in regional bone density in grams/square cm (g/cm2) and T- and Z-scores of hip and spine measured by Dual Energy X-ray Absorptiometry compared to baseline

Androgen metabolism_1Baseline + 8 weeks + 10 months

Ratio between Etiocholanolone and Androsterone (ng/mg) in 24-hour urine

Androgen metabolism_2Baseline + 8 weeks + 10 months

Ratio between 5-alpha-tetrahydrocortisol (5a-THF) and tetrahydrocortisol (THF) in 24-hour urine

Androgen metabolism_3Baseline + 8 weeks + 10 months

Level of testosterone in 24-hour urine (ng/L)

Androgen metabolism_4Baseline + 8 weeks + 10 months

Level of 3-alpha-androstanediol in 24-hour urine (nmol/L)

Androgen metabolism_5Baseline + 8 weeks + 10 months

Level of 11-oxygenated androgens (11-OHA4) (pg/L) in 24-hour urine

Intrathecal Semaglutide10 months

Level of semaglutide in cerebrospinal fluid (picomol/L)

Ammoniaemia_1Baseline + 8 weeks + 10 months

Levels of plasma ammonium (umol/L)

Ammoniaemia_2Baseline + 8 weeks + 10 months

Correlation between plasma ammonium (umol/L) and presence of fatty liver disease as indicated by liver ultrasonography

Ketosis8 weeks + 10 months

Proportion of patients in ketosis measured by urine stix

Change in metabolic parameters8 weeks + 10 months

Change in glycated hemoglobin (mmol/mol) compared to baseline

Trial Locations

Locations (2)

Danish Headache Center, Department of Neurology, Rigshospitalet

🇩🇰

Glostrup, Denmark

Headache clinic, Department of Neurology, Odense University Hospital

🇩🇰

Odense, Denmark

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