Effect of Cannabinoids on Pain in Fabry Disease Patients

NCT04820361 | Unknown

• 1 other identifier: Cannabinoids in Fabry pain
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

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 accumulation of glycosphingolipids in a wide variety of cells, resulting in major organ system damage. Patients with Fabry disease can suffer from neuropathic pain, since lysosomal accumulation affects small unmyelinated nerve fibers. Neuropathic pain is one of the prominent and debilitating symptoms significantly interfering with life quality in FD patients. Current treatment of Fabry patients with neuropathic pain is deficient, as they respond poorly to a conventional pain therapy, often require a high-dose opioids treatment and presentation to the Emergency Department. Sativex® has been shown to be a successful treatment option in neuropathic pain of different origin with minimal neuropsychological influence: in multiple sclerosis (MS), chemotherapy-induced neuropathic pain and other. It contains Δ-9-Tetrahydrocannabinol (THC) and Cannabidiol (CBD) and has recently been licensed in Switzerland for treatment of neuropathic chronic pain in MS. Sativex® is an oral spray.

Conditions

Pain, Neuropathic

Outcome Measures

Primary Outcomes (1)

• NRS

  NRS is a straightforward commonly used method to illustrate pain.

  Daily NRS score after 28 days will be compared to baseline NRS score, evaluated before titration phase starts

Secondary Outcomes (7)

• QST

  At baseline and after 28 days of treatment with study drug and placebo, respectively.

• WHO-Quality of life score (WHOQOL-BREF)

  Between baseline and treatment after 28 days.

• Patient global impression of change (PGIC)

  For the average pain of treatment week 4.

• Short form McGrill Paint Questionnaire (SF-MPQ)

  Compared between baseline and average pain of treatment after 28 days.

• Douleur Neuropathic en 4 questions (DN4-Questionnaire)

  Compared between baseline and treatment week 4.

Study Arms (2)

Placebo

PLACEBO COMPARATOR

oral spray

Drug: Placebo

Sativex®

ACTIVE COMPARATOR

.It contains Δ-9-Tetrahydrocannabinol (THC) and Cannabidiol (CBD)

Drug: Cannabis sativa L., folium cum flore

Interventions

Cannabis sativa L., folium cum flore DRUG

Muscle Relaxation

Also known as: Spasmolytic

Sativex®

Placebo DRUG

Muscle Relaxation

Also known as: Spasmolytic

Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Albina Nowak, MD: lead
• Swiss National Science Foundation: collaborator

Study Sites (1)

University Hospital Zürich USZ

Zurich, Switzerland

RECRUITING

Related Publications (3)

• Jensen TS, Baron R. Translation of symptoms and signs into mechanisms in neuropathic pain. Pain. 2003 Mar;102(1-2):1-8. doi: 10.1016/s0304-3959(03)00006-x. No abstract available.

  PMID: 12620591 RESULT

• Rolke R, Baron R, Maier C, Tolle TR, Treede -DR, Beyer A, Binder A, Birbaumer N, Birklein F, Botefur IC, Braune S, Flor H, Huge V, Klug R, Landwehrmeyer GB, Magerl W, Maihofner C, Rolko C, Schaub C, Scherens A, Sprenger T, Valet M, Wasserka B. Quantitative sensory testing in the German Research Network on Neuropathic Pain (DFNS): standardized protocol and reference values. Pain. 2006 Aug;123(3):231-243. doi: 10.1016/j.pain.2006.01.041. Epub 2006 May 11.

  PMID: 16697110 RESULT

• Serpell M, Ratcliffe S, Hovorka J, Schofield M, Taylor L, Lauder H, Ehler E. A double-blind, randomized, placebo-controlled, parallel group study of THC/CBD spray in peripheral neuropathic pain treatment. Eur J Pain. 2014 Aug;18(7):999-1012. doi: 10.1002/j.1532-2149.2013.00445.x. Epub 2014 Jan 13.

  PMID: 24420962 RESULT

MeSH Terms

Conditions

Neuralgia

Interventions

Parasympatholytics

Condition Hierarchy (Ancestors)

Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Autonomic Agents; Peripheral Nervous System Agents; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses

Study Officials

• Albina A Nowak, PH

  University of Zurich

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Albina Nowak, PD

CONTACT

+41432538872; albina.nowak@usz.ch

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: PROSPECTIVE, RANDOMIZED, DOUBLE-BLIND, PLACEBO- CONTROLLED, CROSSOVER, MULTICENTER STUDY.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsorl Investigator

Study Record Dates

• First Submitted: December 8, 2020
• First Posted: March 29, 2021
• Study Start: July 7, 2022
• Primary Completion: December 29, 2023
• Study Completion: December 29, 2023
• Last Updated: May 12, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1)