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

Niemann-Pick Disease, Type C1 Clinical Trial

Official title:

a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03201627
• Other study ID #: XH-17-010
• Status: Active, not recruiting
• Phase: Early Phase 1
• Start date: July 5, 2017
• Est. completion date: June 30, 2021

Study information

• Verified date: September 2019
• Source: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 18
• Est. completion date: June 30, 2021
• Est. primary completion date: September 30, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years to 40 Years

Eligibility

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

Related Conditions & MeSH terms

• Niemann-Pick Disease, Type C
• Niemann-Pick Disease, Type C1
• Niemann-Pick Diseases
• Pick Disease of the Brain

Intervention

Drug:
• Lithium Carbonate
To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1

Locations

Country	Name	City	State
China	Xin Hua Hospital	Shanghai	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	NPC clinical severity score		52 weeks
Secondary	7-ketocholesterol		52 weeks