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Prospective, single-center, cross-over trial to verify safety and effectiveness of nicotinamide riboside for patients with Werner syndrome (EMPOWER)

Cross-over trial to verify safety and effectiveness of nicotinamide riboside for patients with Werner syndrome (EMPOWER)

Yokote Koutaro

Chiba University Hospital

1-8-1 Inohana, Chuo-ku, Chiba-shi, Chiba, JAPAN

kyokote@faculty.chiba-u.jp

Koshizaka Masaya

Chiba University Hospital

1-8-1 Inohana, Chuo-ku, Chiba-shi, Chiba, JAPAN

+81-43-226-2092

overslope@chiba-u.jp

Recruiting

Nov. 27, 2019

Interventional

randomized controlled trial

double blind

placebo control

crossover assignment

treatment purpose

Patients who meet all of the following criteria are included.
1) Patients who are diagnosed with Werner syndrome
2) Patients who explained the study contents using explanatory documents and obtained written consent for study participation from the person or the representative
3) Male or female 20 years old and more
4) Patients whose hematocrit value is judged to be clinically acceptable by the investigator or co-investigator
5) Patients who can comply with the protocol requirements by the investigator or co-investigator

Patients who meet any of the following criteria are not included.
1) Disease or (including laboratory values) that may be influenced by the interpretation of data or safety, or the protocol evaluation cannot be performed safely, as the investigator or co-author decides Patients with clinically significant abnormalities.
2) Patients who have significant dehydration as judged by the principal investigator or co-investigator
3) Patients with pericardial fluid, ascites and pleural effusion
4) Patients participating in another clinical trial within 30 days before obtaining consent
5) Clinical signs seen at initial screening are the final stage of Werner syndrome progression, and safe completion of the study and patients judged to be difficult to assess, including:
- Patients who received continuous or intermittent home oxygen therapy for 6 months before obtaining consent
- Patients who received at least 2 hospitalizations for pneumonia during the 12 months prior to obtaining consent
- Patients who show at least 10% net weight loss and do not recover as stated on the chart. This includes significant net weight loss over the last six months (beyond the estimated measurement error).
- Patients requiring CKD stage 5 and/or peritoneal or hemodialysis
- Patients whose liver function test value is more than 3 times the upper limit of normal
6) Patients with a history of hypersensitivity to nicotinamide riboside
7) Patients with a history of serious drug hypersensitivity or allergic reaction such as anaphylaxis
8) Patients whose estimated survival time is judged to be at terminal stage less than 2 years
9) Patients who cannot comply with study requirements and procedures
10) Patients with malignancy or patients with a history of malignancy
11) Patients suspected of having cancer on chest X-ray
12) Patients who are positive for tumor marker test (CEA, CA19-9) (if positive, upper and lower gastrointestinal endoscopy must show negative for cancer, for enrollment)
13) Pregnant, brestfeeding, possibly pregnant or planned patients
14) Patients who are considered inappropriate by the investigator or co-investigators
15) Patients taking nicotinamide riboside within 30 days of screening visit

Both

Werner syndrome

Subjects ingest oral capsules of NR (nicotinamide riboside) once daily (total daily dose: nicotinamide riboside 1000 mg).

Werner syndrome

nicotinamide riboside

C16.320.925 / C18.452.284.960

Safety at 26 and 52 weeks of the tested drugs in patients with Werner syndrome

- Size of skin ulcer
- NAD levels in whole blood
- Inflammatory response
- Blood test (lipid, blood glucose, liver function etc.)
- Handgrip strength test
- Walking speed
- Visceral fat
- Skeletal muscle index by DEXA (Dual energy X ray absorption) method
- Heel pad thickness
- CAVI (Cardio-ankle vascular index)
- ABI (Ankle brachial pressure index)
- PWV (Pulse wave velocity)
- Insulin resistance by glucose clamp method
- Glomerular filtration rate calculated with Cockroft-Gault, MDRD, CKD-EPI and cystatin C based methods
- Urinary albumin excretion, urinary NAG, beta2-microglobulin, L-FABP, Kim-1
- Lipoprotein particle profile calculated by NMR or HPLC
- Resting metabolic rate

- Metabolomics
- qPCR (Quantitative polymerase chain reaction)
- Western blotting
- RNA sequence
- DNA methylation profile

+81-43-226-2616

Nov. 07, 2019

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