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Completed

NCT Number: NCT02314208

Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Pitié-Salpêtrière Hospital

Paris, France

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Xenbilox

Drug

Other names: Chenodeoxycholic acid

Resveratrol

Dietary Supplement

Tahor

Drug

Other names: Atorvastatin

Primary outcomes

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

    Time frame: 2 months

    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

Secondary outcomes

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

    Time frame: 2 months

    In blood and urines samples of the 12 patients, the following criteria will be assesed in order to observe an evolution of the acid profile:

    • total biliary acids
    • ratio of primary/secondary biliary acids
    • the ratio cholic acids/chenodeoxycholic acids
    • the ratio chenodeoxycholic acids/ lithocholic acids

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

    Time frame: 2 months

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

  3. Number of participants with adverse events related with digestive problems

    Time frame: 18 months

    During the study, adverse events related with digestive problems will be evaluate in order to asses the clinical tolerance to each treatment

  4. Number of partcipants with adverse events related with standard biological parameters

    Time frame: 18 months

    Biological tolerance will be evaluate on patients blood samples at each visit. The following criteria will be assessed:

    • ionogram and fasting glucose
    • kidney and liver function with urea, creatinine,lipase, amylase, bilirubine, creatine phosphokinase, transaminases,sodium, potassium, calcium, phosphorus

Sponsors and collaborators

Lead sponsor

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

Other Gov

Registry information

Acronym: SPA-M

Important dates

Study start
2015
Primary completion
2017
Study completion
2018
First posted
Dec 11, 2014
Registry last updated
Sep 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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