Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5 (SPA-M)

Institut National de la Santé Et de la Recherche Médicale, France

Status and phase: Completed; Phase 2

Conditions: Spastic Paraplegia, Hereditary

Treatments: Dietary Supplement: Resveratrol; Drug: Xenbilox; Drug: Tahor

Study type: Interventional

Funder types: Other

Identifiers

NCT02314208

C14-04

Details and patient eligibility

About

The purpose of this project is to study the efficacy of three candidate molecules (Xenbilox, Tahor and Resveratrol) in order to decrease the production of oxysterols by reducing the synthesis of cholesterol and/or regulate the production of bile acids and/or enabling neuroprotective action within the motor neuron.

Full description

The primary objective of the study is:

- decrease the accumulation of metabolites which can have a negative impact on neurological and systemic function of patients with SPG5.

The secondary objectives of the study are:

• confirm the clinical and biological tolerance of the different candidate molecules under study
• improve the serum bile acid profile of patients with SPG5

Enrollment: 12 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• patients that have confirmed through genetic testing their status as carriers of 2 mutations in the CYP7B1 gene
• age ≥ 18 years
• patients that have signed the informed consent form
• presence of health care coverage

Exclusion criteria

• known hypersensitvity to chenodeoxycholic acid, atorvastatin, resveratrol or to any of their byproducts
• cholesterol lowering medications other than the study treatment
• hepatic failure with transaminases >3 times the normal level
• progressive biliary pathology
• chronic diarrhea
• serious mental illness
• significant comorbid neurological disorder
• incapacity to understand information about the protocol
• unwilling or unable to participate in any part of the study
• participation in another clinical trial during the study period
• person deprived of liberty by judicial or administrative decision
• adult subject under legal protection or unable to consent
• pregnant or breastfeeding women
• lack of health care coverage
• absence of a signed informed consent form

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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12 participants in 3 patient groups

Xenbilox

Active Comparator group

Description:

Xenbilox (chenodeoxycholic acid) 1000mg capsule by mouth every day for 2 months

Treatment:

Drug: Xenbilox

Resveratrol

Active Comparator group

Description:

Resveratrol 80mg capsule by mouth every day for 2 months

Treatment:

Dietary Supplement: Resveratrol

Tahor

Active Comparator group

Description:

Tahor (atorvastatin) 40mg tablet by mouth every day for 2 months

Treatment:

Drug: Tahor