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

Shanghai Jiao Tong University

Status and phase: Unknown; Early Phase 1

Conditions: Niemann-Pick Disease, Type C1

Treatments: Drug: Lithium Carbonate

Study type: Interventional

Funder types: Other

Identifiers

NCT03201627

XH-17-010

Details and patient eligibility

About

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

Enrollment: 18 estimated patients

Sex: All

Ages: 7 to 40 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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18 participants in 1 patient group

treatment

Experimental group

Treatment:

Drug: Lithium Carbonate