UnknownEarly Phase 1

Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease

China18 participantsStarted 2017-07-05

Plain-language summary

This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.

Age range7 Years – 40 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. aged from 7 years to 40 years
✓. onset of neurological symptoms prior to 15 years of age
✓. confirmed diagnosis of NPC1 determined by either two NPC1 mutations or one NPC1 mutation with elevated plasma 7-ketocholesterol
✓. ability to walk either independently or with assistance
✓. Subject or parent/guardian able to communicate with investigator, understand and abide the rules of this clinical trial.
✓. parent/guardian able to accompany the subjects to participate in the clinical trial
✓. Subject or parent/guardian must provide written informed consent

✕. One NPC clinical severity scale score reached 5
✕. Female in pregnancy or breastfeeding
✕. The predicted life span less than 1 year
✕. Severe liver insufficiency (defined as hepatic laboratory parameters, AST and/or ALT greater than three-times the upper limit of normal
✕. Renal insufficiency, with the serum creatine greater than 1.5 times the upper limit of normal
✕. Free with neurological symptoms
✕. Take antidiuretic hormone

NPC clinical severity score

Timeframe: 52 weeks

NCT IDNCT03201627

SponsorXinhua Hospital, Shanghai Jiao Tong University School of Medicine

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2020-09-30