Add-on Clioquinol in drug-resistant epilepsy: an exploratory study

Overview

Sponsor-declared trial summary

Key facts

Sponsor

UZ Leuven

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05], Diseases [C] - Nervous System Diseases [C10]

Trial duration

16 Jul 2024 → ongoing

Decision date (initial)

2024-07-16

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

To study whether add-on clioquinol decreases the seizure frequency and severity in patients with drug-resistant epilepsy.

Secondary objectives 3

1. Safety during trial (systematic recording of adverse events)
2. Assessment of seizure severity with the National Hospital Seizure Severity Scale (NHS3 scale)
3. Assessment of overall impact of seizures, medication side effects, comorbidities, and overall Quality of Life (QoL) using the Personal Impact of Epilepsy Scale (PIES)

Conditions and MedDRA coding

Drug resistant epilepsy

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Drug-resistant epilepsy: ≥ 4 seizures in the 2 week prospective period (baseline) before visit 2, not all (4) seizures observed in 1 of the 2 weeks. Baseline period can be extended with 1 or 2 weeks.
2. Age ≥ 12 years and < 35 years at time of inclusion
3. Drug-resistant epilepsy: before inclusion failure of at least 2 AEDs
4. The patient is at the moment of inclusion on max 4 anti-epileptic drugs (VNS and ketogenic diet not included)
5. Well defined epilepsy history with convulsive seizures (with observable and countable motor component)

Exclusion criteria 3

1. Presence of C609T NQO1 SNP after SNP analysis of NQO1 based on a blood sample during baseline visit (visit 1) by Genomics Core
2. Asian etnicity
3. Abnormal low blood level of vitamin B12 or Zn

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Percentage of 50% responders after 2 weeks and 6 weeks exposure to low and higher doses of clioquinol respectively (visit 3 and visit 5)
2. Median % reduction of the seizure frequency at visit 5

Secondary endpoints 3

1. Safety during trial (systematic recording of adverse events) Clinical neurological examination during the trial will identify children with possible neuropathy Adverse events will be reported (clinical and biochemical).
2. Assessment of seizure severity (NHS3 scale)
3. Assessment of overall impact of seizures, medication side effects, comorbidities, and overall QoL (PIES).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

UZ Leuven

Sponsor organisation

UZ Leuven

Address

Herestraat 49

City

Leuven

Postcode

3000

Country

Belgium

Scientific contact point

Organisation

UZ Leuven

Contact name

Prof. Dr. Lieven Lagae

Public contact point

Organisation

UZ Leuven

Contact name

Prof. Dr. Lieven Lagae

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 13 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications