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

Primary Purpose

Spastic Paraplegia, Hereditary

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Xenbilox
Resveratrol
Tahor
Sponsored by
Institut National de la Santé Et de la Recherche Médicale, France
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spastic Paraplegia, Hereditary focused on measuring spastic paraplegia, metabolism, oxysterols, neurogenetics

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Pitié-Salpêtrière Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Active Comparator

Active Comparator

Active Comparator

Arm Label

Xenbilox

Resveratrol

Tahor

Arm Description

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

Resveratrol 80mg capsule by mouth every day for 2 months

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Modification of the serum acid profile after a 2-month treatment measured in blood and urine samples
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
Change of 25-hydroxycholesterol blood level after 2 months of treatment for each treatment
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
Number of participants with adverse events related with digestive problems
During the study, adverse events related with digestive problems will be evaluate in order to asses the clinical tolerance to each treatment
Number of partcipants with adverse events related with standard biological parameters
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

Full Information

First Posted
November 14, 2014
Last Updated
March 30, 2018
Sponsor
Institut National de la Santé Et de la Recherche Médicale, France
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1. Study Identification

Unique Protocol Identification Number
NCT02314208
Brief Title
Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5
Acronym
SPA-M
Official Title
Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5
Study Type
Interventional

2. Study Status

Record Verification Date
February 2016
Overall Recruitment Status
Completed
Study Start Date
January 2015 (undefined)
Primary Completion Date
September 27, 2017 (Actual)
Study Completion Date
January 1, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Institut National de la Santé Et de la Recherche Médicale, France

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.
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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spastic Paraplegia, Hereditary
Keywords
spastic paraplegia, metabolism, oxysterols, neurogenetics

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Xenbilox
Arm Type
Active Comparator
Arm Description
Xenbilox (chenodeoxycholic acid) 1000mg capsule by mouth every day for 2 months
Arm Title
Resveratrol
Arm Type
Active Comparator
Arm Description
Resveratrol 80mg capsule by mouth every day for 2 months
Arm Title
Tahor
Arm Type
Active Comparator
Arm Description
Tahor (atorvastatin) 40mg tablet by mouth every day for 2 months
Intervention Type
Drug
Intervention Name(s)
Xenbilox
Other Intervention Name(s)
Chenodeoxycholic acid
Intervention Type
Dietary Supplement
Intervention Name(s)
Resveratrol
Intervention Type
Drug
Intervention Name(s)
Tahor
Other Intervention Name(s)
Atorvastatin
Primary Outcome Measure Information:
Title
Change of 27-hydroxycholesterol blood level after 2 months of treatment for each treatment
Description
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
Time Frame
2 months
Secondary Outcome Measure Information:
Title
Modification of the serum acid profile after a 2-month treatment measured in blood and urine samples
Description
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
Time Frame
2 months
Title
Change of 25-hydroxycholesterol blood level after 2 months of treatment for each treatment
Description
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
Time Frame
2 months
Title
Number of participants with adverse events related with digestive problems
Description
During the study, adverse events related with digestive problems will be evaluate in order to asses the clinical tolerance to each treatment
Time Frame
18 months
Title
Number of partcipants with adverse events related with standard biological parameters
Description
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
Time Frame
18 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Fanny MOCHEL, MD-PhD
Organizational Affiliation
Pitié-Salpêtrière Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Pitié-Salpêtrière Hospital
City
Paris
Country
France

12. IPD Sharing Statement

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Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5

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