A Phase III Randomised, Placebo-controlled, Double-blind, Multi-centre, Clinical Trial to Determine the Efficacy and Safety of Presendin in Idiopathic Intracranial Hypertension
As posted on ClinicalTrials.gov (NCT05347147), updated at source Apr 24, 2024. Read again whenever the registry updates it. Registry figures can differ from a published paper, which may use another analysis.
| Measure | Presendinn=2 | Placebon=1 |
|---|---|---|
| Subject 1 Baseline ICP | 25 | not reported |
| Subject 1 Week 24 ICP | 28 | not reported |
| Subject 2 Baseline ICP | 28 | not reported |
| Subject 2 Week 24 ICP | 29 | not reported |
| Subject 3 Baseline ICP | not reported | 40 |
| Subject 3 Week 24 ICP | not reported | 30 |
Percent of participants affected, Adverse event data were collected over a period of up to 30 weeks, from the signing of the informed consent form to the end of the follow-up period.. The most common events the registry lists.
| Event | Presendin | Placebo | All Subjects Pre-treatment |
|---|---|---|---|
| Any serious event | 0% | 0% | 0% |
| COVID-19 | 37.5% | 0% | 0% |
| Diarrhoea | 0% | 33.3% | 0% |
| Headache | 0% | 33.3% | 0% |
| Injection site mass | 25% | 16.7% | 0% |
| Catheter site pain | 0% | 16.7% | 0% |
| Fatigue | 0% | 16.7% | 0% |
| Injection site rash | 0% | 16.7% | 0% |
| Vessel puncture site bruise | 0% | 16.7% | 0% |
| Food poisoning | 0% | 16.7% | 0% |
| Migraine | 0% | 16.7% | 0% |
| Syncope | 0% | 16.7% | 0% |
| Blood pressure systolic increased | 0% | 16.7% | 0% |
| Decreased appetite | 0% | 16.7% | 0% |
| Tinnitus | 0% | 16.7% | 0% |
| Visual impairment | 0% | 16.7% | 0% |
| Depressed mood | 0% | 16.7% | 0% |
| Injection site bruising | 12.5% | 0% | 0% |
| Injection site hypersensitivity | 12.5% | 0% | 0% |
| Injection site indentation | 12.5% | 0% | 0% |
| Injection site pain | 12.5% | 0% | 0% |
| Injection site pruritus | 12.5% | 0% | 0% |
| Injection site swelling | 12.5% | 0% | 0% |
| Abdominal abscess | 12.5% | 0% | 0% |
| Cellulitis | 12.5% | 0% | 0% |
| Influenza | 12.5% | 0% | 0% |
| Measure | Presendinn=4 | Placebon=5 |
|---|---|---|
| Subject 1: right eye, Baseline PMD | -5.89 | not reported |
| Subject 1: right eye, Week 24 PMD | 0.74 | not reported |
| Subject 2: right eye, Baseline PMD | -3.60 | not reported |
| Subject 2: right eye, Week 24 PMD | -1.09 | not reported |
| Subject 3: right eye, Baseline PMD | not reported | -2.79 |
| Subject 3: right eye, Week 24 PMD | not reported | -1.50 |
| Subject 3: left eye, Baseline PMD | not reported | -4.78 |
| Subject 3: left eye, Week 24 PMD | not reported | -2.33 |
| Subject 4: right eye, Baseline PMD | not reported | -3.16 |
| Subject 4: right eye, Week 24 PMD | not reported | -2.20 |
| Subject 4: left eye, Baseline PMD | not reported | -2.36 |
| Subject 4: left eye, Week 24 PMD | not reported | -1.60 |
| Measure | Presendinn=4 | Placebon=4 |
|---|---|---|
| Subject 1: right eye, Baseline RNFL | 115 | not reported |
| Subject 1: right eye, Week 24 RNFL | 105 | not reported |
| Subject 2: right eye, Baseline RNFL | 199 | not reported |
| Subject 2: right eye, Week 24 RNFL | 173 | not reported |
| Subject 3: right eye, Baseline RNFL | not reported | 140 |
| Subject 3: right eye, Week 24 RNFL | not reported | 155 |
| Subject 3: left eye, Baseline RNFL | not reported | 114 |
| Subject 3: left eye, Week 24 RNFL | not reported | 122 |
| Subject 4: right eye, Baseline RNFL | not reported | 432 |
| Subject 4: right eye, Week 24 RNFL | not reported | 372 |
| Subject 4: left eye, Baseline RNFL | not reported | 358 |
| Subject 4: left eye, Week 24 RNFL | not reported | 317 |
| Measure | Presendinn=3 | Placebon=4 |
|---|---|---|
| Subject 1, right eye | -6.67 | not reported |
| Subject 2, right eye | 18.64 | not reported |
| Subject 3, right eye | not reported | 8.61 |
| Subject 3, left eye | not reported | 2.61 |
| Subject 4, right eye | not reported | 0.77 |
| Subject 4, left eye | not reported | 2.35 |
| Subject 5, right eye | not reported | -1.53 |
| Subject 5, left eye | not reported | 11.41 |
| Subject 6, right eye | -11.87 | not reported |
| Subject 6, left eye | -8.10 | not reported |
| Subject 7, right eye | not reported | -2.82 |
| Subject 7, left eye | not reported | -6.46 |
| Measure | Presendinn=8 | Placebon=6 |
|---|---|---|
| Subject 1 Baseline (5 days) | 28 | not reported |
| Subject 1 28-day period 6 (14 days) | 28 | not reported |
| Subject 2 Baseline (7 days) | 20 | not reported |
| Subject 2 28-day period 6 (24 days) | 3.5 | not reported |
| Subject 3 Baseline (7 days) | not reported | 28 |
| Subject 3 28-day period 6 (9 days) | not reported | 28 |
| Subject 4 Baseline (5 days) | 28 | not reported |
| Subject 4 28-day period 4 (11 days) | 28 | not reported |
| Subject 5 Baseline (2 days) | NA | not reported |
| Subject 5 28-day period 4 (10 days) | 5.6 | not reported |
| Subject 6 Baseline (4 days) | not reported | NA |
| Subject 6 28-day period 2 (10 days) | not reported | 11.2 |
| Measure | Presendinn=8 | Placebon=6 |
|---|---|---|
| Subject 1 Baseline (5 days) | 5.6 | not reported |
| Subject 1 28-day period 6 (14 days) | 10 | not reported |
| Subject 2 Baseline (7 days) | 12 | not reported |
| Subject 2 28-day period 6 (24 days) | 1.167 | not reported |
| Subject 3 Baseline (7 days) | not reported | 8 |
| Subject 3 28-day period 6 (9 days) | not reported | 24.889 |
| Subject 4 Baseline (5 days) | 16.8 | not reported |
| Subject 4 28-day period 4 (11 days) | 28 | not reported |
| Subject 5 Baseline (2 days) | NA | not reported |
| Subject 5 28-day period 4 (10 days) | 0 | not reported |
| Subject 6 Baseline (4 days) | not reported | NA |
| Subject 6 28-day period 2 (10 days) | not reported | 5.6 |
| Measure | Presendinn=8 | Placebon=6 |
|---|---|---|
| Number of responders (subjects who completed the study) | 1 | 0 |
| Number of responders (all subjects) | 1 | 0 |
| Measure | Presendinn=8 | Placebon=6 |
|---|---|---|
| Number of responders (subjects who completed the study) | 2 | 0 |
| Number of responders (all subjects) | 2 | 0 |
| Measure | Presendinn=8 | Placebon=6 |
|---|---|---|
| Subject 1 baseline (5 days) | 6(4 to 7) | not reported |
| Subject 1 28-day period 6 (14 days) | 4(2 to 7) | not reported |
| Subject 2 Baseline (5 days) | 3(2 to 4) | not reported |
| Subject 2 28-day period 6 (3 days) | 4(2 to 6) | not reported |
| Subject 3 Baseline (7 days) | not reported | 4(2 to 5) |
| Subject 3 28-day period 6 (9 days) | not reported | 8(3 to 8) |
| Subject 4 Baseline (5 days) | 5(3 to 7) | not reported |
| Subject 4 28-day period 4 (11 days) | 7(6 to 8) | not reported |
| Subject 5 Baseline (1 day) | NA(NA to NA) | not reported |
| Subject 5 28-day period 4 (2 days) | 3(3 to 3) | not reported |
| Subject 6 Baseline (3 days) | not reported | NA(NA to NA) |
| Subject 6 28-day period 2 (5 days) | not reported | 2(2 to 9) |
| Measure | Presendinn=8 | Placebon=6 |
|---|---|---|
| Subject 1 Baseline (5 days) | 0 | not reported |
| Subject 1 28-day period 6 (14 days) | 6 | not reported |
| Subject 2 Baseline (5 days) | 16.8 | not reported |
| Subject 2 28-day period 6 (3 days) | 0 | not reported |
| Subject 3 Baseline (7 days) | not reported | 4 |
| Subject 3 28-day period 6 (9 days) | not reported | 24.89 |
| Subject 4 Baseline (5 days) | 0 | not reported |
| Subject 4 28-day period 4 (11 days) | 0 | not reported |
| Subject 5 Baseline (1 day) | NA | not reported |
| Subject 5 28-day period 4 (2 days) | 0 | not reported |
| Subject 6 Baseline (3 days) | not reported | NA |
| Subject 6 28-day period 2 (5 days) | not reported | 11.2 |
| Measure | Presendinn=8 | Placebon=6 |
|---|---|---|
| Treatment failure suspected | 0 | 1 |
| Treatment failure confirmed | 0 | 0 |
Idiopathic intracranial hypertension (IIH) has significant associated morbidity and reduced quality of life. There is a significant risk of visual loss and patients also typically suffer with chronic disabling headaches. This trial has been designed to evaluate the efficacy and safety of a new formulation of exenatide (Presendin) in the reduction of intracranial pressure (ICP) in patients with IIH.
Inclusion Criteria: 1. Age ≥18 years at the time of consent. 2. Diagnosis of new IIH by consensus criteria, including normal structural brain imaging (excluding features of raised ICP and incidentalomas), including either magnetic resonance venography or computed tomographic venography to exclude thrombosis and no evidence of a secondary causes of raised ICP. 3. Newly diagnosed patients with screening commenced no more than 4 weeks after the diagnostic LP. 4. Lumbar puncture opening pressure ≥25 cm cerebrospinal fluid (CSF) at diagnosis. 5. Presence of bilateral papilloedema (Frisén grade ≥1). Verification of papilloedema by the OCT Reading Centre. Where there is uncertainty fundus photography and/or ultrasound scan (B scan) of the optic nerves should be conducted for evaluation by the Independent Adjudication Committee (IAC). 6. Perimetric Mean Deviation defined as between -2 to -7 decibels (dB) in at least one eye. Eyes meeting this criterion will defined as 'study eyes'. 7. Reproducible visual loss present on automated perimetry including no more than 15% false positive responses (reliability confirmed by the Visual Field Reading Centre) in study eyes. 8. Two or more headache days over the 7-day period prior to screening and also the patient must meet this criterion during the 7-day screening period. 9. Females of childbearing potential must have a negative pregnancy test and must agree to use a highly effective birth control method (failure rate less than 1% per year when used consistently and correctly) during the whole trial duration including the last follow-up visit (12 weeks after ceasing drug). Female patients who are lactating must agree to stop breast-feeding OR Female patients of non-childbearing potential (defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or post-menopausal females defined as 12 months of amenorrhoea \[in questionable cases a blood sample with simultaneous follicle stimulation hormone 25-140 IE/L and oestradiol \<200 pmol/L is confirmatory\]). 10. Male patients with a female partner of childbearing potential must commit to practice methods of contraception (e.g., condom, vasectomy) and abstain from sperm donation during the trial including the last follow-up visit (12 weeks after ceasing drug). Their partners, if they are women of childbearing potential, must agree to practice contraception and to use a highly effective method of contraception during the trial, including the last follow-up visit (12 weeks after ceasing drug). 11. Able to provide written informed consent. Exclusion Criteria: IIH-related exclusion criteria: 1. Presence of venous sinus thrombosis on brain imaging by either magnetic resonance or computerised tomographic venography. 2. Previous IIH surgery including CSF shunt, optic nerve sheath fenestration or dural venous sinus stent or sub-temporal decompression. 3. Previous bariatric surgery within the last 3 months or intention during the trial. 4. Abnormal...
Trial averages do not predict what one person will experience. Results are shown as the registry posts them, without interpretation.