Sodium Valproate for GSDV

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03112889
• Date of registration: 11/08/2015
• Prospective Registration: No
• Primary sponsor: University College, London
• Public title: Sodium Valproate for GSDV
• Scientific title: A Phase II Pilot Study to Explore Treatment With Sodium Valproate in Adults With McArdle Disease (Glycogen Storage Disorder Type V, GSDV)
• Date of first enrolment: January 2015
• Target sample size: 8
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03112889
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Denmark, United Kingdom

Contacts

• Name: Ros Quinlivan, FRCPCH, MD
• Affiliation: University College, London

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male subjects and post-menopausal or infertile females

- Diagnosed with GSDV and over 18 years of age

- Normal serum carnitine level and acylcarnitine blood profile at screening visit

Exclusion Criteria:

- Children under the age of 18 years

- People older than 64 years

- Females of child bearing potential

- Patients with Diabetes

- Inflammatory disorders especially systemic lupus erythematosis.

- A previous history of sensitivity/allergy to sodium valproate and its excipients

- Patients treated with sodium valproate for epilepsy or a psychiatric disorder within
the last 12 months prior to screening

- Patients with pre-existing liver disease or a family history of severe liver disease
affecting a first degree relative. Liver disease will be defined by abnormal liver
biopsy. Patients with GSDV may have raised serum transaminases that originate from
muscle but which may cause abnormal liver function tests measured in serum, this will
not be a reason for exclusion.

- Patients prescribed other anti-convulsant medication or any other medication known to
interact with sodium valproate (see section 9.3).

- Patients who are sensitive to local anaesthetics that would prevent muscle biopsy.

- Subjects with any co-morbid illness or disability which would prevent an exercise
assessment such as severe unstable/ untreated ischaemic heart disease, lower limb
disability such as severe muscle weakness with muscle strength assessed as worse than
MRC scale 3 in any pelvic girdle muscle.

- Inability to exercise due to a lower limb fracture would be an exclusion criterion
until there is complete recovery of the injury.

- Patients known to have porphyria or an affected first degree relative affected with
porphyria will be excluded from the study.

- Patients known to have mitochondrial disease or where there is a first degree relative
with mitochondrial disease.

- Patients with a history of abnormal acyl carnitine profile or low serum carnitine
level

- Male participants unwilling to use contraception



Age minimum: 18 Years
Age maximum: 64 Years
Gender: All

Health Condition(s) or Problem(s) studied

Glycogen Storage Disease Type V

McArdle Disease

Intervention(s)

Drug: Sodium Valproate

Primary Outcome(s)

Change in VO2peak [Time Frame: Week 1, Week 16 and Week 28]

Secondary Outcome(s)

Adverse events log [Time Frame: Week 4, Week 8, Week 16, Week 20, Week 24, Week 28, +- Week 40]

Blood lactate responses to exercise [Time Frame: Week 1, Week 16 and Week 28]

Quality of life [Time Frame: Week 1, Week 16 and Week 28]

Change in total walked distance [Time Frame: Week 1, Week 16 and Week 28]

Presence of phosphorylase positive fibres [Time Frame: Week 0 and Week 28]

Safety of sodium valproate assessed by blood exams and self-reported adverse events [Time Frame: For the duration of the trial and within 3 months of Visit 3 (+- Week 40)]

Secondary ID(s)

11/0090

Source(s) of Monetary Support

Please refer to primary and secondary sponsors