Pain, NeuropathicNCT04820361
Effect of Cannabinoids on Pain in Fabry Disease Patients
A study testing Cannabis sativa L., folium cum flore in people with Pain, Neuropathic, run by Albina Nowak, MD at 1 site in Switzerland.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Pain, Neuropathic
- What is being tested?
- Cannabis sativa L., folium cum flore
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- 1 siteSwitzerland
- Study dates
- 7 Jul 2022to 29 Dec 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- • 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
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- 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
Who cannot join
- • 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
Switzerland1 site, 1 recruiting
- Recruiting
University Hospital Zürich USZ
Zurich
Albina Nowak, albina.nowak@usz.ch
About this study
Official title: Effect of Cannabinoids on Pain in Fabry Disease Patients; a Prospective, Randomized, Double-blind, Placebo-controlled, Crossover, Multicenter Study
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.
Read the 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 lysosomal accumulation of lipids can also take place in the small nerve fibers. Typically, neuropathic pain occurs in late childhood or adolescence and disappears after several years, probably due to the irreversible destruction of the small nerve fibers. The pain management of Fabry patients suffering from neuropathic pain is inadequate, as patients often do not respond well to conventional pain therapies.
Aim of this study:
The aim of this study is to find out how strongly the investigational drug Sativex® (active ingredients: tetrahydrocannabinol (THC) and cannabidiol (CBD)) can influence the pain that can be caused by Fabry's disease. For this purpose, the investigational drug is compared with a placebo drug. The latter is a drug without an active ingredient. These studies provide us with important information on the origin of the pain and at the same time on the mechanism of action of Sativex®. This makes it possible to develop new forms of therapy in the future.
Procedure:
A total of 22-30 patients are divided into two groups of 11-15 patients each. Both groups will undergo the same test program. This will be divided into two phases: In the first phase, which will last 8 weeks, one group will receive Sativex® while the other group will receive a placebo. In the second phase, which will also last 8 weeks, the group that previously received the investigational drug will now receive the placebo and the previous placebo group will now receive the investigational drug. The study is double-blind, i.e. neither the patient nor the investigator knows who is receiving the investigational drug or the placebo. Patients are randomly assigned to the groups. Throughout the treatment, patients maintain their usual pain management regimen. (see Study Schedule) For 14 weeks, patients will fill out their pain diary daily. Every two weeks a blood sample is taken to determine the levels of cannabinoid metabolites. At the beginning, at the end of the first and after the second phase, the patient will fill out various questionnaires on neuropathic pain and improvement of quality of life. This will be used to assess whether pain relief is achieved.
Translated with www.DeepL.com/Translator (free version)
- Conditions
- Pain, Neuropathic
- Being tested
- Cannabis sativa L., folium cum flore, Placebo
- Medical subject headings
- Neuralgia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cannabis sativa L., folium cum floreDrug
Also called Spasmolytic
Muscle Relaxation
PlaceboDrug
Also called Spasmolytic
Muscle Relaxation
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | oral spray Receives: Drug: Placebo |
| Sativex® | Active comparator | .It contains Δ-9-Tetrahydrocannabinol (THC) and Cannabidiol (CBD) Receives: Drug: Cannabis sativa L., folium cum flore |
What is being measured
The primary outcome is the main question the study is designed to answer.
NRS
NRS is a straightforward commonly used method to illustrate pain.
Measured: Daily NRS score after 28 days will be compared to baseline NRS score, evaluated before titration phase starts
Secondary outcomes (7)
QST
Pain thresholds using the quantitative sensory testing.
Measured: At baseline and after 28 days of treatment with study drug and placebo, respectively.
WHO-Quality of life score (WHOQOL-BREF)
Improvement of quality of life.
Measured: Between baseline and treatment after 28 days.
Patient global impression of change (PGIC)
Improvement of quality of life.
Measured: For the average pain of treatment week 4.
Short form McGrill Paint Questionnaire (SF-MPQ)
Improvement of neuropathic pain.
Measured: Compared between baseline and average pain of treatment after 28 days.
Douleur Neuropathic en 4 questions (DN4-Questionnaire)
Improvement of neuropathic pain.
Measured: Compared between baseline and treatment week 4.
Profile of Mood States (POMS questionnaire)
Change in Profile of Mood States.
Measured: Between baseline and treatment week 4.
Insomnia severity Index
Change in Insomnia severity score.
Measured: Between baseline and treatment after 28 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2021
- Study start7 Jul 2022
- Primary completion29 Dec 2023 (estimated)
- Study completion29 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Pain, NeuropathicStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 22 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Albina Nowak, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Swiss National Science Foundation
- Responsible party
- Sponsor-investigator, Albina Nowak, MD, University of Zurich
- Investigators
- Albina A Nowak, PH, Principal investigator, University of Zurich
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04820361
- Sponsor's protocol ID
- Cannabinoids in Fabry pain
- Status last verified
- May 2023
- First submitted
- 8 Dec 2020
- Last updated
- 12 May 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 12620591Rolke 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.
PubMed 16697110Serpell 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.
PubMed 24420962
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Cannabinoids on Pain in Fabry Disease Patients; a Prospective, Randomized, Double-blind, Placebo-controlled, Crossover, Multicenter Study
- ClinicalTrials.gov ID
- NCT04820361
- Acronym / sponsor ID
- Cannabinoids in Fabry pain
- Conditions
- Pain, Neuropathic
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 22 participants (estimated)
- Lead sponsor
- Albina Nowak, MDUniversity, hospital or other
- Collaborators
- Swiss National Science Foundation
- Study start
- 7 Jul 2022 (actual)
- Primary completion
- 29 Dec 2023 (estimated)
- Study completion
- 29 Dec 2023 (estimated)
- First posted
- 29 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 lysosomal accumulation of lipids can also take place in the small nerve fibers. Typically, neuropathic pain occurs in late childhood or adolescence and disappears after several years, probably due to the irreversible destruction of the small nerve fibers. The pain management of Fabry patients suffering from neuropathic pain is inadequate, as patients often do not respond well to conventional pain therapies.
Aim of this study:
The aim of this study is to find out how strongly the investigational drug Sativex® (active ingredients: tetrahydrocannabinol (THC) and cannabidiol (CBD)) can influence the pain that can be caused by Fabry's disease. For this purpose, the investigational drug is compared with a placebo drug. The latter is a drug without an active ingredient. These studies provide us with important information on the origin of the pain and at the same time on the mechanism of action of Sativex®. This makes it possible to develop new forms of therapy in the future.
Procedure:
A total of 22-30 patients are divided into two groups of 11-15 patients each. Both groups will undergo the same test program. This will be divided into two phases: In the first phase, which will last 8 weeks, one group will receive Sativex® while the other group will receive a placebo. In the second phase, which will also last 8 weeks, the group that previously received the investigational drug will now receive the placebo and the previous placebo group will now receive the investigational drug. The study is double-blind, i.e. neither the patient nor the investigator knows who is receiving the investigational drug or the placebo. Patients are randomly assigned to the groups. Throughout the treatment, patients maintain their usual pain management regimen. (see Study Schedule) For 14 weeks, patients will fill out their pain diary daily. Every two weeks a blood sample is taken to determine the levels of cannabinoid metabolites. At the beginning, at the end of the first and after the second phase, the patient will fill out various questionnaires on neuropathic pain and improvement of quality of life. This will be used to assess whether pain relief is achieved.
Translated with www.DeepL.com/Translator (free version)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebooral spray |
| Sativex® | Active comparator | Drug: Cannabis sativa L., folium cum flore.It contains Δ-9-Tetrahydrocannabinol (THC) and Cannabidiol (CBD) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- • Age: 18-70 years
Key exclusion criteria (10 listed)
- • Known hypersensitivity or allergy to cannabinoids.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cannabis sativa L., folium cum floreAlso: SpasmolyticMuscle Relaxation | Drug | Sativex® |
| PlaceboAlso: SpasmolyticMuscle Relaxation | Drug | Placebo |
Primary outcome
NRS
Time frame: Daily NRS score after 28 days will be compared to baseline NRS score, evaluated before titration phase starts
NRS is a straightforward commonly used method to illustrate pain.
7 secondary outcomes
QST
Time frame: At baseline and after 28 days of treatment with study drug and placebo, respectively.
WHO-Quality of life score (WHOQOL-BREF)
Time frame: Between baseline and treatment after 28 days.
Patient global impression of change (PGIC)
Time frame: For the average pain of treatment week 4.
Short form McGrill Paint Questionnaire (SF-MPQ)
Time frame: Compared between baseline and average pain of treatment after 28 days.
Douleur Neuropathic en 4 questions (DN4-Questionnaire)
Time frame: Compared between baseline and treatment week 4.
Profile of Mood States (POMS questionnaire)
Time frame: Between baseline and treatment week 4.
Insomnia severity Index
Time frame: Between baseline and treatment after 28 days.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2023.
- Results publication: PubMed 12620591Jensen 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.
- Results publication: PubMed 16697110Rolke 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.
- Results publication: PubMed 24420962Serpell 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Albina A Nowak, PH principal investigator
University of Zurich
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Cannabis sativa L., folium cum flore in people with Pain, Neuropathic, run by Albina Nowak, MD at 1 site in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Albina Nowak, PD (+41432538872, albina.nowak@usz.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
NRS (time frame: Daily NRS score after 28 days will be compared to baseline NRS score, evaluated before titration phase starts). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04820361, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 May 2023.