Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5

NCT02314208 | Completed Phase 2

• 1 other identifier: C14-04
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Spastic Paraplegia, Hereditary

Keywords

spastic paraplegia; metabolism; oxysterols; neurogenetics

Outcome Measures

Primary Outcomes (1)

• Change of 27-hydroxycholesterol blood level after 2 months of treatment for each treatment

  27-hydroxycholesterol will be measured on fasten blood samples for the 12 patients included before and after the 2-month treatment period for each treatment

  2 months

Secondary Outcomes (4)

• Modification of the serum acid profile after a 2-month treatment measured in blood and urine samples

  2 months

• Change of 25-hydroxycholesterol blood level after 2 months of treatment for each treatment

  2 months

• Number of participants with adverse events related with digestive problems

  18 months

• Number of partcipants with adverse events related with standard biological parameters

  18 months

Study Arms (3)

Xenbilox

ACTIVE COMPARATOR

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

Drug: Xenbilox

Resveratrol

ACTIVE COMPARATOR

Resveratrol 80mg capsule by mouth every day for 2 months

Dietary Supplement: Resveratrol

Tahor

ACTIVE COMPARATOR

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

Drug: Tahor

Interventions

Xenbilox DRUG

Also known as: Chenodeoxycholic acid

Xenbilox

Resveratrol DIETARY_SUPPLEMENT

Resveratrol

Tahor DRUG

Also known as: Atorvastatin

Tahor

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Institut National de la Santé Et de la Recherche Médicale, France: lead

Study Sites (1)

Pitié-Salpêtrière Hospital

Paris, France

Location

Related Publications (1)

• Marelli C, Lamari F, Rainteau D, Lafourcade A, Banneau G, Humbert L, Monin ML, Petit E, Debs R, Castelnovo G, Ollagnon E, Lavie J, Pilliod J, Coupry I, Babin PJ, Guissart C, Benyounes I, Ullmann U, Lesca G, Thauvin-Robinet C, Labauge P, Odent S, Ewenczyk C, Wolf C, Stevanin G, Hajage D, Durr A, Goizet C, Mochel F. Plasma oxysterols: biomarkers for diagnosis and treatment in spastic paraplegia type 5. Brain. 2018 Jan 1;141(1):72-84. doi: 10.1093/brain/awx297.

  PMID: 29228183 BACKGROUND

MeSH Terms

Conditions

Spastic Paraplegia, Hereditary; Paraplegia

Interventions

Chenodeoxycholic Acid; Resveratrol; Atorvastatin

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Paralysis; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Deoxycholic Acid; Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes; Stilbestrols; Stilbenes; Benzylidene Compounds; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polyphenols; Phenols; Pyrroles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heptanoic Acids; Fatty Acids; Lipids

Study Officials

• Fanny MOCHEL, MD-PhD

  Pitié-Salpêtrière Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2014
• First Posted: December 11, 2014
• Study Start: January 8, 2015
• Primary Completion: September 27, 2017
• Study Completion: January 1, 2018
• Last Updated: September 2, 2025

Record last verified: 2025-02

Locations

FR (1)