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

• Status: Unknown status
• Trial end date: 2021-06-30
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
• Treatments: Lithium Carbonate

Criteria

Inclusion Criteria:

1. aged from 7 years to 40 years

2. onset of neurological symptoms prior to 15 years of age

3. confirmed diagnosis of NPC1 determined by either two NPC1 mutations or one NPC1 mutation with elevated plasma 7-ketocholesterol

4. ability to walk either independently or with assistance

5. Subject or parent/guardian able to communicate with investigator, understand and abide the rules of this clinical trial.

6. parent/guardian able to accompany the subjects to participate in the clinical trial

7. Subject or parent/guardian must provide written informed consent

Exclusion Criteria:

1. One NPC clinical severity scale score reached 5

2. Female in pregnancy or breastfeeding

3. The predicted life span less than 1 year

4. Severe liver insufficiency (defined as hepatic laboratory parameters, AST and/or ALT greater than three-times the upper limit of normal

5. Renal insufficiency, with the serum creatine greater than 1.5 times the upper limit of normal

6. Free with neurological symptoms

7. Take antidiuretic hormone