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Donepezil for Oxaliplatin-induced Neuropathy Peripheral Neuropathy: Proof of Concept Study

Phase 2
Completed
Conditions
Digestive Oncology
Supportive Care
Interventions
Drug: PLACEBO
Registration Number
NCT05254639
Lead Sponsor
University Hospital, Clermont-Ferrand
Brief Summary

The use of oxaliplatin in the treatment of colorectal or pancreas cancer induces (\>75% of patients) severe sensorimotor neuropathy decreasing the quality of life of cancer survivors. Today, no treatment remains univocal for these peripheral neuropathies. But preclinical works have demonstrated that donepezil (acetylcholinesterase inhibitor use for Alzheimer's disease) was able to prevent and treat neuropathic symptoms in oxaliplatin-treated rats.

Present study aims to assess the therapeutic efficacy of donepezil on oxaliplatin-induced peripheral neuropathy (OIPN) in cancer survivors. Bibliographic data suggests an antineuropathic effect of donepezil in human and animal models. In clinic, a study have shown in healthy volunteers that donepezil (associated with gabapentin) reduced the pain threshold (better than gabapentin alone) caused by stimulation of the sural nerve, without severe adverse effect. Similarly, two studies in patients with neuropathic pain demonstrated that donepezil increases analgesic effect of gabapentin. Finally, a case report demonstrated an analgesic effect of donepezil in painful Alzheimer's disease patients. In animals, several studies demonstrated that donepezil induces analgesic and neuroprotective effects. Recently, a preclinical study demonstrated that donepezil induced antineuropathic effect in diabetic mice with neuropathic pain. Research unit INSERM U1107 (partner of the DONEPEZOX study) demonstrated the antineuropathic effects of donepezil in several animal models of chemotherapy-induced peripheral neuropathies, and very recently, a study have confirmed these results with oxaliplatin and cisplatin. These clinical and preclinical data have thus highlighted the potential beneficial effect of donepezil on neuropathic symptoms, without any significant adverse effects. Therefore the hypothesis is that the use of donepezil could reduce the symptoms of OIPN, limit the decrease in quality of life and the appearance of comorbidities (anxiety/depression) in cancer survivors.

For this purpose, the investigators propose here a proof of concept, multicentre, phase II, randomised, double-blind, placebo-controlled clinical study. The primary objective will be the curative efficacy of donepezil on the severity of OIPN in patients who have completed oxaliplatin-based chemotherapy for the treatment of colorectal or pancreas cancer and have peripheral neuropathy of grade ≥2. This will be assessed using the EORTC QLQ-CIPN20 sensory scale. Our methodological choice to use the QLQ-CIPN20 as the primary endpoint will allow us to more accurately (and in a standardized manner) characterize neuropathic symptoms and assess the therapeutic effect of donepezil on these symptoms. In addition, as secondary objectives, we will study the effect of donepezil on neuropathic pain, the intensity of neuropathic symptoms, health-related quality of life, and the tolerance of donepezil.

The 80 patients required will be randomized (1:1) to receive either placebo or donepezil (5 mg daily for 4 weeks and then 10 mg daily for 12 weeks as a single dose and according to tolerance and efficacy). Patients will be followed for 1 month after the end of treatment to assess the OIPN. As a proof of concept study, responder rate will be assessed only for Donepezil arm (primary objective) and compared between each treatment arm (secondary objective) after a minimum of 12 weeks of treatment. A responder will be defined as a patient with a decrease of neuropathic grade according to CIPN20 sensory score compraed to baseline.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
77
Inclusion Criteria
  • Patient who received chemotherapy with oxaliplatin for all stage of colorectal or pancreas cancer,
  • QLQ-CIPN20 sensory score ≥30,
  • Diagnosis of chemotherapy-induced peripheral neuropathy treated or not by stable antineuropathic/analgesic treatment (opioids, pregabalin, gabapentin, duloxetine and other antidepressants or anticonvulsants) for at least 1 month,
  • Chemotherapy completed for at least 6 months,
  • Patients affiliated to the French national health insurance,
  • Written informed consent,
  • French language comprehension.
Exclusion Criteria
  • Cancer relapse or secondary cancer,
  • Lack of effective contraception in patients (female) of childbearing age, pregnant or breastfeeding women, women of childbearing age who have not taken a pregnancy test,
  • Patient with a chronic progressive disease with associated chronic pain (excluding oxaliplatin-induced peripheral neuropathy),
  • Diabetic patient (excluding non-insulin- or insulin-treated diabetes less than 5 years old) or presence of proven diabetic neuropathy,
  • Other types of neuropathies,
  • ALT / AST elevated more than 3 times the normal values,
  • Severe cardiovascular disease (as determined by clinician), bradycardia (< 55 bpm), cardiac conduction disorders such as sinus disease or other supraventricular conduction abnormalities such as sino-auricular or atrioventricular block (assessed by electrocardiogram),
  • History of peptic ulcer disease or active peptic ulcer disease,
  • Asthma or chronic obstructive pulmonary disease,
  • Known allergy to donepezil or piperidine derivatives,
  • Known galactose intolerance, known Lapp lactase deficiency or known glucose or galactose malabsorption syndrome (rare hereditary diseases),
  • Drug interactions: CYP3A4 inhibitors (ketoconazole, itraconazole and erythromycin); CYP2D6 inhibitors (fluoxetine, quinidine) and enzymatic inducers (rifampicin, phenytoin, carbamazepine),
  • Known dependence on alcohol and/or drugs,
  • Known psychotic disorders, patient under antipsychotics,
  • Planned surgery during the trial,
  • Impossible to undergo the medical follow-up of the trial for geographical, social or psychological reasons,
  • Person under guardianship, curatorship, safeguard of justice or person deprived of liberty.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPLACEBO5 mg/day for 4 weeks then 10 mg/day for 12 weeks
DonepezilDONEPEZIL5 mg/day for 4 weeks then 10 mg/day for 12 weeks
Primary Outcome Measures
NameTimeMethod
Quality of Life Questionnaire-Chemotherapy Induced Peripheral Neuropathy 20 (QLQ-CIPN20)through study completion, an average of 4 months.

A self-reported questionnaire, consisting of 20 questions which assess the symptoms and functional limitations of CIPN from the patients' perspective. The questionnaire is divided in 3 subscales: sensory, motor, and autonomic and gives a comprehensive picture of the nature, frequency, and severity of CIPN.

For each subscale and for the entire questionnaire, a score from 0 to 100 is generated. The higher the score, the more severe the neuropathic symptoms.

Secondary Outcome Measures
NameTimeMethod
11-point pain Numeric Rating Scale (NRS)through study completion, an average of 4 months

The NRS is an 11-point scale for patient self-reporting of pain. It is for adults and children 10 years old or older. The score of 0 = No pain and 10 = worst possible pain.

Patients' Global Impression of Change (PGIC)4 months

PGIC is aimed at assessing the general effectiveness of the treatment. This scale consists of 7 level descriptors answering the question "How are you?" distributed in three ways: (i) improved (very/medium/slightly), (ii) unchanged and (iii) aggravated (slight/medium/very).

Douleur Neuropathique-4 (DN4) interviewAt inclusion

The interview portion of the DN4 questionnaire is a clinician-administered screening tool for neuropathic pain.

The questionnaire includes 7 items, grouped into two questions. Each item, is answered as either YES or NO.

A final cumulative patient's score is obtained by allocating 1 point for each YES and 0 point for each NO. If the patient's score ≥3/7, the test is positive.

This evaluation will be carried out only if the 11-point pain NRS ≥4/10.

Neuropathic Pain Symptoms Inventory (NPSI)through study completion, an average of 4 months.

This self-reported questionnaire assesses different neuropathic pain symptoms. The French NPSI includes 12 items that discriminates and quantifies five distinct dimensions of neuropathic pain.

This evaluation will be carried out only if the 11-point pain NRS ≥4/10.

Quality of Life Questionnaire-Cancer 30 (QLQ-C30)through study completion, an average of 4 months.

This EORTC self-reported questionnaire assesses the health-related quality of life of cancer patients in clinical trials. The questionnaire includes five functional scales (physical, everyday activity, cognitive, emotional, and social), three symptom scales (fatigue, pain, nausea and vomiting), a health/quality of life overall scale, and a number of additional elements assessing common symptoms (including dyspnea, loss of appetite, insomnia, constipation, and diarrhea), as well as, the perceived financial impact of the disease.

Hospital Anxiety and Depression scale (HADS)through study completion, an average of 4 months.

This self-reported questionnaire permits to detect anxiety and depressive disorders. There are 14 items rated from 0 to 3. Seven questions relate to anxiety (total A) and 7 to the depressive dimension (total D), thus obtaining two scores (maximum score of each score = 21). To detect anxiety and depressive symptoms, the following interpretation is proposed for each of the scores (A and D):

* ≤7: absence of symptomatology

* 8 to 10: suspicious symptomatology

* ≥11: certain symptomatology

Trial Locations

Locations (34)

Hôpital privé d'Antony

🇫🇷

Antony, France

Polyclinique Bordeaux Nord Aquitaine

🇫🇷

Bordeaux, France

CH d'Argenteuil

🇫🇷

Argenteuil, France

CHU de Besançon

🇫🇷

Besançon, France

Clinique de Flandre

🇫🇷

Coudekerque-Branche, France

Centre Hospitalier Compiègne-Noyon

🇫🇷

Compiègne, France

CHU de Dijon Bourgogne

🇫🇷

Dijon, France

CHD de Vendée

🇫🇷

La Roche-sur-Yon, France

CH Le puy

🇫🇷

Le Puy-en-Velay, France

Hôpital Franco-Britanique

🇫🇷

Levallois-Perret, France

CH Saint Joseph Saint Luc

🇫🇷

Lyon, France

Hopital Saint Joseph de Marseille

🇫🇷

Marseille, France

Groupe Hospitalier des Portes de Provence

🇫🇷

Montélimar, France

Hôpital Européen de Marseille

🇫🇷

Marseille, France

Hôpital Saint-Louis - AP-HP

🇫🇷

Paris, France

Clinique La Croix du Sud

🇫🇷

Quint-Fonsegrives, France

Hôpital Privé des Cotes d'Armor

🇫🇷

Plérin, France

Institut Godinot

🇫🇷

Reims, France

CHU de Reims

🇫🇷

Reims, France

Groupe Hospitalier Saint Vincent - Clinique Saint Anne

🇫🇷

Strasbourg, France

Institut de Cancérologie Paris Nord

🇫🇷

Sarcelles, France

CH de Valence

🇫🇷

Valence, France

CHU de Bordeaux

🇫🇷

Talence, France

Hôpital d'Instruction des Armées Sainte-Anne

🇫🇷

Toulon, France

Centre Hospitalier Public du Cotentin

🇫🇷

Cherbourg, France

CH de Cholet

🇫🇷

Cholet, France

Hôpital privé Jean Mermoz

🇫🇷

Lyon, France

CHU clermont-ferrand

🇫🇷

Clermont-Ferrand, France

CHU de Poitiers

🇫🇷

Poitiers, France

Institut de Cancérologie de Bourgogne - GRReCC

🇫🇷

Dijon, France

chu de Limoges

🇫🇷

Limoges, France

CHU de Saint-Etienne

🇫🇷

Saint-Étienne, France

Clinique de la sauvegarde

🇫🇷

Lyon, France

Centre Hospitalier du Cotentin

🇫🇷

Cherbourg, France

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