Change in ICP From Baseline to Week 24 Measured by Lumbar Puncture (LP), Where a Higher LP Value Indicates Greater ICP
| Group | Value (cm CSF) |
|---|---|
| Presendin | 25 |
| Placebo | not posted |
Trial · Interventional
TerminatedPhase 3Results postedRegistry dataShort name IIH EVOLVE
This trial was stopped early.
Reason given at the registry: Following a strategic evaluation of its IIH EVOLVE Phase III clinical trial investigating Presendin™, the Invex Board has made the difficult decision that the continuation of the trial is not viable and therefore the trial has been terminated.
Results were posted for this trial.
This trial does not test a peptide that PepBank tracks.
United States, Australia, Germany, Israel, New Zealand, United Kingdom
1 trial · 1 stopped early · 1 with posted results
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Experimental
Presendin
2.0 mg
Placebo comparator
Placebo
Outcomes the sponsor listed as primary.
Change in ICP From Baseline to Week 24 Measured by Lumbar Puncture (LP), Where a Higher LP Value Indicates Greater ICP
Time frame · Baseline to Week 24
ICP was measured by LP (opening pressure) using an LP manometer; Baseline and Week 24 ICP values (measured in cm CSF) are presented for each subject. A standard operating procedure was followed by all study sites for all study-related ICP measurements by LP.
Change in Perimetric Mean Deviation (PMD), Measured by Humphrey Visual Field (HVF) Analysis (24-2 SITA-Standard), Where a Larger Negative Result Indicates Greater Visual Loss
Time frame · Baseline to Week 24
Papilloedema by Change in Retinal Nerve Fibre Layer (RNFL) Thickness, With a Greater Thickness of RNFL Indicating Greater Swelling and Greater Extent of Papilloedema
Time frame · Baseline to Week 24
Papilloedema by Percent Change in Optic Nerve Head Size, Measured by Optical Coherence Tomography (OCT), Where a Larger Optic Nerve Head Size Reflects Greater Swelling and a Greater Extent of Papilloedema
Time frame · Baseline to Week 24
The Number of Monthly Headache Days (MHD)
Time frame · Baseline to Week 24
Monthly headache days (according to daily headache diary) = number of days recorded in a 28-day window where data were collected on \>7 days and where ≥1 headache on a day met the following criteria: * Onset, continuation, or recurrence of headache * Any severity or phenotype of headache * Lasts at least 30 minutes Baseline headache frequency was calculated over the 7 days prior to the randomization visit; ≥5 days of headache data were needed to obtain a valid baseline value. The number of headache days recorded for a period were linearly scaled by the total number of days of data collected in the period for each subject to give the MHD during the baseline period (linearly scaled to a maximum of 28 days) and the last 28-day period during which data were collected on \>7 days for each subject prior to study completion or discontinuation. Period 1 = Weeks 1-4 Period 2 = Weeks 5-8 Period 3 = Weeks 9-12 Period 4 = Weeks 13-16 Period 5 = Weeks 17-20 Period 6 = Weeks 21-24
Number of Moderate to Severe MHD
Time frame · Baseline to Week 24
Moderate to severe (m-s) MHD (according to daily headache diary) = number of days recorded in a 28-day window where data were collected on \>7 days and where ≥1 headache on a day met the following criteria: * Severity was of moderate or severe pain and lasted at least 4 hours or, * Required acute headache analgesics Baseline m-s headache frequency was calculated over the 7 days prior to the randomization visit; ≥5 days of headache data were needed to obtain a valid baseline value. The number of m-s headache days recorded for a period were linearly scaled by the total number of days of data collected in the period for each subject to give the m-s MHD during the baseline period (linearly scaled to a max 28 days) and the last 28-day period during which data were collected on \>7 days for each subject prior to study completion or discontinuation. Period 1 = Weeks 1-4 Period 2 = Weeks 5-8 Period 3 = Weeks 9-12 Period 4 = Weeks 13-16 Period 5 = Weeks 17-20 Period 6 = Weeks 21-24
Number of MHD Responders (Defined as a ≥50% Reduction in MHD)
Time frame · Baseline to Week 24
A subject was considered a responder if they had at least a 50% reduction in MHD from baseline to Week 24. Subjects who dropped out prior to Week 24 were considered non-responders.
Number of Moderate to Severe MHD Responders (Defined as a ≥50% Reduction in Moderate to Severe MHD)
Time frame · Baseline to Week 24
A subject was considered a responder if they had at least a 50% reduction in moderate to severe MHD from baseline to Week 24. Subjects who dropped out prior to Week 24 were considered non-responders.
Headache Severity
Time frame · Baseline to Week 24
Headache severity was assessed by a 10-point Numeric Rating Scale (NRS), 0-10 where 0 = no pain and 10 = most severe pain. Severity of headaches was assessed on days where a headache occurred. Headache free days were not counted. Baseline headache severity was calculated over the 7 days prior to the randomization visit; at least 5 of 7 days of headache severity data had to be recorded by the subject to obtain a valid baseline value. * 28-day period 1 = Weeks 1-4 * 28-day period 2 = Weeks 5-8 * 28-day period 3 = Weeks 9-12 * 28-day period 4 = Weeks 13-16 * 28-day period 5 = Weeks 17-20 * 28-day period 6 = Weeks 21-24
Use of Acute Headache Analgesic Medications (Acute Headache Analgesics in Days Per Month)
Time frame · Baseline to Week 24
Number of days recorded in a 28-day window, where at least one dose of an acute headache analgesic was recorded. The baseline acute headache analgesic use was calculated over the 7 days prior to the randomization visit; at least 5 of 7 days had to be recorded by the subject to obtain a valid baseline value. The number of acute headache analgesic use days was linearly scaled for each subject to give the number of acute headache analgesic use days during the baseline period and the last 28-day period during which headache data were collected on more than 7 days for each subject prior to study completion or discontinuation. * 28-day period 1 = Weeks 1-4 * 28-day period 2 = Weeks 5-8 * 28-day period 3 = Weeks 9-12 * 28-day period 4 = Weeks 13-16 * 28-day period 5 = Weeks 17-20 * 28-day period 6 = Weeks 21-24
Visual Acuity
Time frame · Baseline to Week 24
Corrected visual acuity will be recorded using a Logarithm of the Minimum Angle of Resolution (LogMAR) scoring chart, with a range of -0.3 to 1.00, where a lower score indicates better visual acuity.
Number of Patients With Treatment Failure
Time frame · Baseline to Week 24
Treatment failure is defined as the initiation of either medical therapy or a surgical intervention to lower ICP during the study.
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
| Group | Value (cm CSF) |
|---|---|
| Presendin | 25 |
| Placebo | not posted |
| Group | Value (dB) |
|---|---|
| Presendin | -5.89 |
| Placebo | not posted |
| Group | Value (µm) |
|---|---|
| Presendin | 115 |
| Placebo | not posted |
| Group | Value (percentage change) |
|---|---|
| Presendin | -6.67 |
| Placebo | not posted |
| Group | Value (Monthly headache days) |
|---|---|
| Presendin | 28 |
| Placebo | not posted |
| Group | Value (Monthly headache days) |
|---|---|
| Presendin | 5.6 |
| Placebo | not posted |
| Group | Value (Participants) |
|---|---|
| Presendin | 1 |
| Placebo | 0 |
| Group | Value (Participants) |
|---|---|
| Presendin | 2 |
| Placebo | 0 |
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Presendin2.0 mg Presendin: Presendin is supplied as 2 parts, one vial consisting of a drug part (white or greyish white powder in a clear vial) and one pre-filled syringe containing the diluent part (colourless liquid). The drug part is suspended in the diluent part solution and administered SC as a suspension. | 0 / 8 | 0 / 8 | 8 / 8 |
| PlaceboPlacebo: Placebo is supplied as 2 parts (visually identical to the Presendin vial and pre-filled diluent syringe). The drug part will exclude the active pharmaceutical ingredient (exenatide acetate) and the diluent part will be the same as the active treatment diluent. The drug part is suspended in the diluent part solution and administered SC as a suspension. | 0 / 6 | 0 / 6 | 6 / 6 |
| All Subjects Pre-treatmentAll subjects after signing the informed consent form and before being assigned to treatment groups or the start of dosing of the drug product | 0 / 14 | 0 / 14 | 2 / 14 |
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