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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03201627
• Date of registration: 26/06/2017
• Prospective Registration: Yes
• Primary sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
• Public title: Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
• Scientific title: a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type
• Date of first enrolment: July 5, 2017
• Target sample size: 18
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03201627
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Early Phase 1

Countries of recruitment

China

Key inclusion & exclusion 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



Age minimum: 7 Years
Age maximum: 40 Years
Gender: All

Health Condition(s) or Problem(s) studied

Niemann-Pick Disease, Type C1

Intervention(s)

Drug: Lithium Carbonate

Primary Outcome(s)

NPC clinical severity score [Time Frame: 52 weeks]

Secondary Outcome(s)

7-ketocholesterol [Time Frame: 52 weeks]

Secondary ID(s)

XH-17-010

Source(s) of Monetary Support

Please refer to primary and secondary sponsors