Darier disease is a rare and severe autosomal dominant skin disorder.
Conditions
Interventions
Trade Name: Dantrium (dantrolene)
Product Name: Dantrium (dantrolene)
Product Code: ATC code M03CA01
Pharmaceutical Form: Capsule
INN or Proposed INN: Dantrolene sodium
CAS Number: 14663-23-1
Concentr
Sponsors
Region Stockholm Karolinska University Hospital (Dep. of Dermatology)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: DD research subjects will be recruited at the dermatology clinic at the Karolinska University hospital, by advertisement in the Swedish Society for Dermato-Venereology as well as from the very active patient social media networks. Since DD is a familial disorder, participants will also be recruited through previous included participants. We plan to recruit 14 participants. The inclusion criteria will be histopathologically verified diagnosis combined with typical skin symptoms of hyperkeratotic papules and erythema, or clinical symptoms combined with family history in which the relative has a histopathologically verified diagnosis. Written consent to participate in the study will also be an inclusion criteria. For female participants, adequate contraception should be used. A negative pregnancy test (blood-HCG) will be a requirement. Contraceptive requirements may also apply to male participants. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 14 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 14
Exclusion criteria
Exclusion criteria: Subjects must not be included in the study if any of the following criteria are met: Pregnancy, breastfeeding, or planned pregnancy. Recent acute illness (past 4 weeks) Active substance abuse Kidney or liver disease including steatosis, cirrhosis and hepatitis B/C Heart failure Pulmonary disease such as COPD (but not common asthma) Abnormal blood chemistry (assed by blood samples) Known or suspected allergies against dantrolene or vehicle Systemic treatment for Dariers disease that cannot be paused during the study (e.g. acitretin) Mental inability, reluctance or language difficulties that result in difficulty understanding the meaning of study participation Participation or recent participation in a clinical study with an investigational product (within 30 days). Concomitant Medication that may interact: antihistamines (e.g., cetirizine, doxylamine, diphenhydramine, hydroxyzine, loratadine) antipsychotics (e.g., chlorpromazine, clozapine, haloperidol, olanzapine, quetiapine, risperidone) apalutamide benzodiazepines (e.g., alprazolam, diazepam, lorazepam) bosentan brimonidine buspirone calcium channel blockers (e.g., amlodipine, diltiazem, verapamil) cannabis chloral hydrate dabrafenib deferasirox dexamethasone efavirenz enzalutamide estrogens (e.g., conjugated estrogen, estradiol, ethinyl estradiol) etravirine methadone mirtazapine mitotane modafinil muscle relaxants (e.g., baclofen, cyclobenzaprine, methocarbamol, orphenadrine) nabilone narcotic pain relievers (e.g., codeine, fentanyl, morphine, oxycodone) pramipexole rifabutin rifampin ropinirole rotigotine St. John's wort scopolamine seizure medications (e.g., carbamazepine, clobazam, levetiracetam, phenobarbital, phenytoin, primidone, topiramate, valproic acid, zonisamide) selective serotonin reuptake inhibitors (SSRIs; e.g., citalopram, fluoxetine, paroxetine, sertraline) siltuximab sodium oxybate tapentadol thalidomide tocilizumab tramadol tricyclic antidepressants (e.g., clomipramine, desipramine, imipramine) zolpidem zopiclone
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary outcome Does dantrolene treatment reduce skin area affected? The primary outcome will be reduction of skin area affected using the Eczema Area and Severity Index (EASI; scale of 0-72). A 20 % reduction will be considered the minimal clinically important difference. ;Secondary Objective: Secondary outcome Does dantrolene treatment improve patient quality of life? As a secondary outcome, improvement in dermatological quality of life index (DLQI) will be used. A 4 points improvement in DLQI will be considered the minimal clinically important difference. ;Primary end point(s): The study ends when the last subject (the 14th) has completed the last follow-up. The study may be prematurely terminated if it appears that the treatment involved a large number of serious adverse events (SAE) or if recruitment of subjects cannot be met within reasonable time limits. If the study is prematurely terminated or suspended, the investigator should immediately inform the subjects about this and ensure appropriate treatment and follow-up. The regulatory authority should be informed as soon as possible, but no later than within 15 days. We view early termination as unlikely since dantrolene is an established clinical drug and we will only use 12.5 % (100 mg) of the maximal dose (800 mg).;Timepoint(s) of evaluation of this end point: After 6 months of active treatment plus 3 months until the follow up visit, i.e. 9 months. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Same as above.;Timepoint(s) of evaluation of this end point: Same as above. | — |
Countries
Sweden
Contacts
Public ContactJakob Wikström
Region Stockholm Karolinska University Hospital (Dep. of Dermatology)
Outcome results
None listed