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Trial record 2 of 16 for:    "spastic paraplegia type 11" OR "Spastic Paraplegia, Hereditary" OR "Spastic Paraplegia"

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

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ClinicalTrials.gov Identifier: NCT02314208
Recruitment Status : Completed
First Posted : December 11, 2014
Last Update Posted : April 3, 2018
Sponsor:
Information provided by (Responsible Party):
Institut National de la Santé Et de la Recherche Médicale, France

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.

Condition or disease Intervention/treatment Phase
Spastic Paraplegia, Hereditary Drug: Xenbilox Dietary Supplement: Resveratrol Drug: Tahor Phase 2

Detailed 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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5
Study Start Date : January 2015
Actual Primary Completion Date : September 27, 2017
Actual Study Completion Date : January 1, 2018


Arm Intervention/treatment
Active Comparator: Xenbilox
Xenbilox (chenodeoxycholic acid) 1000mg capsule by mouth every day for 2 months
Drug: Xenbilox
Other Name: Chenodeoxycholic acid

Active Comparator: Resveratrol
Resveratrol 80mg capsule by mouth every day for 2 months
Dietary Supplement: Resveratrol
Active Comparator: Tahor
Tahor (atorvastatin) 40mg tablet by mouth every day for 2 months
Drug: Tahor
Other Name: Atorvastatin




Primary Outcome Measures :
  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 Outcome Measures :
  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



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02314208


Locations
France
Pitié-Salpêtrière Hospital
Paris, France
Sponsors and Collaborators
Institut National de la Santé Et de la Recherche Médicale, France
Investigators
Principal Investigator: Fanny MOCHEL, MD-PhD Pitié-Salpêtrière Hospital

Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
ClinicalTrials.gov Identifier: NCT02314208     History of Changes
Other Study ID Numbers: C14-04
First Posted: December 11, 2014    Key Record Dates
Last Update Posted: April 3, 2018
Last Verified: February 2016

Keywords provided by Institut National de la Santé Et de la Recherche Médicale, France:
spastic paraplegia
metabolism
oxysterols
neurogenetics

Additional relevant MeSH terms:
Spastic Paraplegia, Hereditary
Muscle Spasticity
Paraplegia
Muscular Diseases
Musculoskeletal Diseases
Muscle Hypertonia
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Paralysis
Hereditary Sensory and Motor Neuropathy
Nervous System Malformations
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Atorvastatin Calcium
Resveratrol
Chenodeoxycholic Acid
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors