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

Early Phase 1

• Conditions: Niemann-Pick Disease, Type C1
• Interventions: Drug: Lithium Carbonate

• Registration Number: NCT03201627
• Lead Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Brief Summary

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

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2017-06-26
• Study First Submitted QC: 2017-06-26
• Study First Posted: 2017-06-28
• Study Start: 2017-07-05
• Status Verified: 2019-09
• Last Update Submitted: 2019-09-21
• Last Update Posted: 2019-09-24
• Primary Completion: 2020-09-30
• Study Completion: 2021-06-30

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 18

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
treatment	Lithium Carbonate	-

Primary Outcome Measures

Name	Time	Method
NPC clinical severity score	52 weeks

Secondary Outcome Measures

Name	Time	Method
7-ketocholesterol	52 weeks

Trial Locations

Locations (1)

Xin Hua Hospital; 🇨🇳 Shanghai, Shanghai, China