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Study · Randomized controlled trial · n = 15 · 2023

4 results

The effect of GLP-1RA exenatide on idiopathic intracranial hypertension: a randomized clinical trial.

Mitchell JL, Lyons HS, Walker JK, Yiangou A, Grech O, Alimajstorovic Z, Greig NH, Li Y, Tsermoulas G, Brock K, Mollan SP, Sinclair AJ

Brain : a journal of neurology1 May 2023Cited by 133 (Europe PMC)

PMID36907221 (opens in a new tab)PMCIDPMC10151178 (opens in a new tab)DOI10.1093/brain/awad003 (opens in a new tab)PepBankPUB-36907221
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Summary

Therapeutics to reduce intracranial pressure are an unmet need. Preclinical data have demonstrated a novel strategy to lower intracranial pressure using glucagon-like peptide-1 (GLP-1) receptor signalling. Here, we translate these findings into patients by conducting a randomized, placebo-controlled, double-blind trial to assess the effect of exenatide, a GLP-1 receptor agonist, on intracranial pressure in idiopathic intracranial hypertension. Telemetric intracranial pressure catheters enabled long-term intracranial pressure monitoring. The trial enrolled adult women with active idiopathic intracranial hypertension (intracranial pressure >25 cmCSF and papilloedema) who receive subcutaneous exenatide or placebo. The three primary outcome measures were intracranial pressure at 2.5 h, 24 h and 12 weeks and alpha set a priori at less than 0.1. Among the 16 women recruited, 15 completed the study (mean age 28 ± 9, body mass index 38.1 ± 6.2 kg/m2, intracranial pressure 30.6 ± 5.1 cmCSF). Exenatide significantly and meaningfully lowered intracranial pressure at 2.5 h -5.7 ± 2.9 cmCSF (P = 0.048); 24 h -6.4 ± 2.9 cmCSF (P = 0.030); and 12 weeks -5.6 ± 3.0 cmCSF (P = 0.058). No serious safety signals were noted. These data provide confidence to proceed to a phase 3 trial in idiopathic intracranial hypertension and highlight the potential to utilize GLP-1 receptor agonist in other conditions characterized by raised intracranial pressure.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study4

4 results from studies that gave the peptide. 4 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In women with idiopathic intracranial hypertension, exenatide lowered intracranial pressure versus placebo at 2.5 hours.

    In plain wordsIn women with idiopathic intracranial hypertension, exenatide lowered pressure inside the skull more than placebo at 2.5 hours.

    Peptide
    Exenatide (exendin-4)
    Outcome
    DecreaseIntracranial pressure at 2.5 hours
    Study

    n = 15

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Cerebrospinal fluid dynamics · Nervous system and cognition
    Studied dose
    Dose not stated · subcutaneous · 2.5 hoursAs given in this study (human).

    Measurements

    Intracranial pressure effect
    −5.7 cmCSF
    2.5 hours

    Source sentences

    Summary
    Exenatide significantly and meaningfully lowered intracranial pressure at 2.5 h -5.7 ± 2.9 cmCSF (P = 0.048); 24 h -6.4 ± 2.9 cmCSF (P = 0.030); and 12 weeks -5.6 ± 3.0 cmCSF (P = 0.058).
    2 context sentences
    Summary · context
    Here, we translate these findings into patients by conducting a randomized, placebo-controlled, double-blind trial to assess the effect of exenatide, a GLP-1 receptor agonist, on intracranial pressure in idiopathic intracranial hypertension.
    Summary · context
    The trial enrolled adult women with active idiopathic intracranial hypertension (intracranial pressure >25 cmCSF and papilloedema) who receive subcutaneous exenatide or placebo. The three primary outcome measures were intracranial pressure at 2.5 h, 24 h and 12 weeks and alpha set a priori at less than 0.1.
  2. 02HumanRandomized controlled trialEfficacy outcome

    In women with idiopathic intracranial hypertension, exenatide lowered intracranial pressure versus placebo at 24 hours.

    In plain wordsIn women with idiopathic intracranial hypertension, exenatide lowered pressure inside the skull more than placebo at 24 hours.

    Peptide
    Exenatide (exendin-4)
    Outcome
    DecreaseIntracranial pressure at 24 hours
    Study

    n = 15

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Cerebrospinal fluid dynamics · Nervous system and cognition
    Studied dose
    Dose not stated · subcutaneous · 24 hoursAs given in this study (human).

    Measurements

    Intracranial pressure effect
    −6.4 cmCSF
    24 hours

    Source sentences

    Summary
    Exenatide significantly and meaningfully lowered intracranial pressure at 2.5 h -5.7 ± 2.9 cmCSF (P = 0.048); 24 h -6.4 ± 2.9 cmCSF (P = 0.030); and 12 weeks -5.6 ± 3.0 cmCSF (P = 0.058).
    2 context sentences
    Summary · context
    Here, we translate these findings into patients by conducting a randomized, placebo-controlled, double-blind trial to assess the effect of exenatide, a GLP-1 receptor agonist, on intracranial pressure in idiopathic intracranial hypertension.
    Summary · context
    The trial enrolled adult women with active idiopathic intracranial hypertension (intracranial pressure >25 cmCSF and papilloedema) who receive subcutaneous exenatide or placebo. The three primary outcome measures were intracranial pressure at 2.5 h, 24 h and 12 weeks and alpha set a priori at less than 0.1.
  3. 03HumanRandomized controlled trialEfficacy outcome

    In women with idiopathic intracranial hypertension, exenatide lowered intracranial pressure versus placebo at 12 weeks under the prespecified significance threshold.

    In plain wordsIn women with idiopathic intracranial hypertension, exenatide lowered skull pressure more than placebo at 12 weeks under the prespecified significance threshold.

    Peptide
    Exenatide (exendin-4)
    Outcome
    DecreaseIntracranial pressure at 12 weeks
    Study

    n = 15

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Cerebrospinal fluid dynamics · Nervous system and cognition
    Studied dose
    Dose not stated · subcutaneous · 12 weeksAs given in this study (human).

    Measurements

    Intracranial pressure effect
    −5.6 cmCSF
    12 weeks

    Source sentences

    Summary
    Exenatide significantly and meaningfully lowered intracranial pressure at 2.5 h -5.7 ± 2.9 cmCSF (P = 0.048); 24 h -6.4 ± 2.9 cmCSF (P = 0.030); and 12 weeks -5.6 ± 3.0 cmCSF (P = 0.058).
    2 context sentences
    Summary · context
    Here, we translate these findings into patients by conducting a randomized, placebo-controlled, double-blind trial to assess the effect of exenatide, a GLP-1 receptor agonist, on intracranial pressure in idiopathic intracranial hypertension.
    Summary · context
    The trial enrolled adult women with active idiopathic intracranial hypertension (intracranial pressure >25 cmCSF and papilloedema) who receive subcutaneous exenatide or placebo. The three primary outcome measures were intracranial pressure at 2.5 h, 24 h and 12 weeks and alpha set a priori at less than 0.1.
  4. 04HumanRandomized controlled trialSafety outcome

    In women with idiopathic intracranial hypertension, no serious safety signals were noted during the exenatide trial.

    In plain wordsIn women with idiopathic intracranial hypertension, the exenatide trial reported no serious safety signals.

    Peptide
    Exenatide (exendin-4)
    Outcome
    Not applicableSerious safety signals
    Study

    n: not reported in source

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    No serious safety signals were noted.
    2 context sentences
    Summary · context
    Here, we translate these findings into patients by conducting a randomized, placebo-controlled, double-blind trial to assess the effect of exenatide, a GLP-1 receptor agonist, on intracranial pressure in idiopathic intracranial hypertension.
    Summary · context
    The trial enrolled adult women with active idiopathic intracranial hypertension (intracranial pressure >25 cmCSF and papilloedema) who receive subcutaneous exenatide or placebo. The three primary outcome measures were intracranial pressure at 2.5 h, 24 h and 12 weeks and alpha set a priori at less than 0.1.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    22 Oct 2026, 02:26 UTC

    Study results updated

  2. Version 1

    21 Oct 2026, 18:59 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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