Study of the safety and effectiveness of drug AZD4017 at reducing pressure in the brain, in idiopathic intracranial hypertension.

Phase 1

• Conditions: Idiopathic Intracranial Hypertension; MedDRA version: 18.0Level: PTClassification code 10004277Term: Benign intracranial hypertensionSystem Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2013-003643-31-GB
• Lead Sponsor: niversity of Birmingham

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-12-11
• Last Update Posted: 3 April 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Female
• Target Recruitment: 30

Inclusion Criteria

1.Provision of informed consent prior to any study specific procedures.

2.Female patients between 18 and 55 years old

3.Diagnosis of IIH by the updated Modified Dandy criteria with acute (<6 months from Neuro-opthalmology diagnosis), active disease (papilloedema (Frisen grade greater than or equal to 1) and significantly raised intracranial pressure >25cmH2O) and normal brain imaging during previous routine diagnostic work up (evaluated by either magnetic resonance venography or computerised tomography with venography).

4.Patients must be willing to use one form of highly effective, non-hormonal contraception. This would include a vasectomised partner, tubal occlusion or copper containing IUD – all of which should be used in addition to a barrier contraceptive (condom or diaphragm or cervical/vault caps with spermicidal foam/gel/film/suppository) or abstinence (when this is in line with the preferred and usual lifestyle of the subject). Women should have been stable on their chosen method of birth control for a minimum of 2 months before entering the trial. Patients must agree to undergo a b-hCG blood test at screening and urine testing at all trial visits (including the final follow up visit 4 weeks after discontinuation of study treatment).

5. Participants are able to continue other medications to treat their IIH e.g. acetazolamide, diuretics but this dose must remain fixed throughout the study. It would be expected that there would be an equal number in each trial.

6.Must be able to understand the consent form and comply with study requirements.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1.Optic nerve sheath fenestration (as distortion of the optic nerve would prevent accurate assessment of their disease state). Patients who have had previous failed CSF shunting will be eligible for enrolment if they fulfil all other enrolment criteria.

2.Abnormal neurological examination (aside from papilloedema and consequent visual loss or VI nerve palsy).

3.Subjects with a secondary cause of raised intracranial pressure will be excluded.

4.Abnormal cerebrospinal fluid contents (except for that compatible with a traumatic LP).

5.Unable to perform a visual field test reliably.

General Exclusion Criteria:

1.Positive hCG or urine dipstick pregnancy test or planning to conceive.

2.Have eGFR calculated by MDRD equation of <60ml/min/1.73m2.

3.Have any endocrine disorder, e.g, thyroid dysfunction (potential participants with PCOS will no be excluded as this is a known associated condition).

4.Suspicion of or known Gilbert's disease.

5.Creatinine kinase >2 x upper limit of normal on 2 consecutive measurements.

6.Liver aminotransferases > 2x Upper limit of normal

7.Alkaline phosphatases > Upper limit of normal

8.Bilirubin (total) > 2 x upper limit of normal

9. Must not have donated blood within 2 months of attending the screening
visit and avoid donating blood within the following 4 months.

10.Patient is, at the time of signing the informed consent, a user of recreational or illicit drugs (including marijuana) or has had a recent history (within the last year) of drug or alcohol abuse or dependence, in the opinion of the investigator.

11.Pregnant or breastfeeding mothers, unless willing to discontinue breastfeeding by the baseline visit.

12.Have uncontrolled systemic hypertension (BP >160 systolic or >90 diastolic), on 3 successive measurements on the morning of the screening visit.

13.Are receiving systemic (including vaginal/rectal) glucocorticoid treatment at the time of the screening visit.

14.Are taking any hormone-based medication, including hormone contraceptives, at the time of screening.

15.Are taking probenicid at the time of the screening visit.

16.Have any screening laboratory abnormality that, in the investigator’s judgement, is considered to be clinically significant or any screening laboratory value which is outside the Sponsor specified ranges at screening; testing may be repeated once to see if the value returns to within the range but any laboratory abnormality must be resolved prior to the baseline visit.

17.History of any clinically significant disease or disorder which, in the opinion of the investigator, may either put the subject at risk because of participation in the study, or influence the results of the subject’s ability to participate in the study. Specifically, a diagnosis of any inflammatory disorder that might reasonably need treatment with glucocorticoids during the course of the study.

18.History or presence of sigificant gastrointestinal, hepatic, or renal disease or any other condition known to interfere with absorption, distribution, metabolism, or excretion of drugs.

19.Any clinically significant illness, medical/surgical procedure or trauma within 4 weeks of the first administration of IP as judged by the investigator.

20.Have been involved in the planning and/or conduct of the study

21.Participation in another interventional study in the prevous 1 month prior to screening

22.Previous randomisation for treatment in the present study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified