Status:

UNKNOWN

Effect of Cannabinoids on Pain in Fabry Disease Patients

Lead Sponsor:

Albina Nowak, MD

Collaborating Sponsors:

Swiss National Science Foundation

Conditions:

Pain, Neuropathic

Eligibility:

All Genders

18-70 years

Phase:

NA

Brief Summary

Fabry Disease (FD) is a rare lysosomal storage disorder due to the absence or deficiency of hydrolase α-galactosidase A (α-Gal A) activity in lysosomes. This dysfunction results in progressive accumul...

Detailed Description

Fabry disease is an X-linked lysosomal storage disease caused by a deficiency of the enzyme α-galactosidase A. Patients suffering from Fabry's disease may suffer from neuropathic pain, since the lysos...

Eligibility Criteria

Inclusion

  • • Age: 18-70 years
  • Patients with genetically confirmed Fabry disease
  • On treatment with Enzyme Replacement Therapy (ERT)
  • Sufficient command of German language
  • Pain duration of more than 3 months
  • Stable analgesic regimen for at least 2 weeks preceding the study entry day
  • Baseline worst last week pain intensity ≥4 on numerical rating scale (NRS)
  • Signed and dated informed consent
  • ERT or chaperone therapy at a stable dose for at least 3 last months

Exclusion

  • • Known hypersensitivity or allergy to cannabinoids.
  • Women who are pregnant or breast feeding; intention to become pregnant during the course of the study, lack of safe contraception, defined as: female subjects of childbearing potential, not using and not willing to continue using a medically reliable method of contraception for the entire study duration, such as oral, injectable, or implantable contraceptives, or intrauterine contraceptive devices, or who are not willing or able to use any other second (additional) considered sufficiently reliable by the investigator in individual cases. Female subjects who are surgically sterilized/hysterectomized or post-menopausal for longer than 2 years are not considered as being of child bearing potential.
  • Dementia
  • Other pain not of neuropathic origin thought by the investigator to be of nature or severity to interfere with the patient's assessment of neuropathic pain due to Fabry disease.
  • Patients with known or suspected non-compliance, drug or alcohol abuse, including Marijuana cigarettes.
  • Patients with known schizophrenia, other psychotic disorders, personality disorders or another severe psychiatric disorder or positive family history with these disorders, except depression.
  • Patients with another clinically significant disease (e.g. renal failure, hepatic dysfunction, severe cardiovascular or convulsive diseases).
  • Participation in another study with investigational drugs within the 30 days preceding and during the present study.
  • Previous enrolment into the current study
  • Enrolment of the investigator, his/her family members, employees and other dependent persons.

Key Trial Info

Start Date :

July 7 2022

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 29 2023

Estimated Enrollment :

22 Patients enrolled

Trial Details

Trial ID

NCT04820361

Start Date

July 7 2022

End Date

December 29 2023

Last Update

May 12 2023

Active Locations (1)

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Page 1 of 1 (1 locations)

1

University Hospital Zürich USZ

Zurich, Switzerland