Cerebrotendinous xanthomatosis
Conditions
Interventions
investigational material(s)
Generic name etc : FPF1011
INN of investigational material : Chenodeoxycholic acid
Therapeutic category code : 399 Agents affecting metabolism, n.e.c.
Dosage and Administra
Sponsors
Fujimoto Pharmaceutical Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Main inclusion criteria. 1) The patient diagnosed with cerebrotendinous xanthomatosis. 2) Written informed consent. 3) In case of female patient of childbearing potential, the result of the pregnancy test (urine) conducted prior to registration must be negative, and she must consent to use suitable contraception not to get pregnant. 4) Outpatient.
Exclusion criteria
Exclusion criteria: Main exclusion criteria. 1) Patient who possibly changes the dosage and administration of restricted concomitant drugs or the usage of the restricted combination therapy. 2) Patient with the serious complications in circulation, respiratory, blood, liver, kidney, digestive system, and so on. 3) Patient with allergy to commercial CDCA formulations. 4) Breastfeeding woman. 5) Patient who has received any other investigational product or post-marketing clinical trial drug within 12 weeks prior to giving informed consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy Serum cholestanol concentrations | — |
Secondary
| Measure | Time frame |
|---|---|
| efficacy safety 1. Safety evaluations a) Adverse event b) Laboratory tests (blood, urine) c) Vital signs [body temperature, systolic blood pressure, diastolic blood pressure, and pulse rate] d) Body weight e) 12-Lead electrocardiogram at rest 2. Efficacy evaluations a) MMSE-J b) Rankin Scale c) MRI (brain, spinal cord) d) Bone density (DXA) e) The diseases or symptoms related to cerebrotendinous xanthomatosis | — |
Countries
Japan
Outcome results
None listed