Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
Primary Purpose
Niemann-Pick Disease, Type C1
Status
Unknown status
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
Lithium Carbonate
Sponsored by
About this trial
This is an interventional treatment trial for Niemann-Pick Disease, Type C1
Eligibility Criteria
Inclusion Criteria:
- aged from 7 years to 40 years
- onset of neurological symptoms prior to 15 years of age
- confirmed diagnosis of NPC1 determined by either two NPC1 mutations or one NPC1 mutation with elevated plasma 7-ketocholesterol
- ability to walk either independently or with assistance
- Subject or parent/guardian able to communicate with investigator, understand and abide the rules of this clinical trial.
- parent/guardian able to accompany the subjects to participate in the clinical trial
- Subject or parent/guardian must provide written informed consent
Exclusion Criteria:
- One NPC clinical severity scale score reached 5
- Female in pregnancy or breastfeeding
- The predicted life span less than 1 year
- Severe liver insufficiency (defined as hepatic laboratory parameters, AST and/or ALT greater than three-times the upper limit of normal
- Renal insufficiency, with the serum creatine greater than 1.5 times the upper limit of normal
- Free with neurological symptoms
- Take antidiuretic hormone
Sites / Locations
- Xin Hua Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
treatment
Arm Description
Outcomes
Primary Outcome Measures
NPC clinical severity score
Secondary Outcome Measures
7-ketocholesterol
Full Information
NCT ID
NCT03201627
First Posted
June 26, 2017
Last Updated
September 21, 2019
Sponsor
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
1. Study Identification
Unique Protocol Identification Number
NCT03201627
Brief Title
Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
Official Title
a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type
Study Type
Interventional
2. Study Status
Record Verification Date
September 2019
Overall Recruitment Status
Unknown status
Study Start Date
July 5, 2017 (Actual)
Primary Completion Date
September 30, 2020 (Anticipated)
Study Completion Date
June 30, 2021 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Niemann-Pick Disease, Type C1
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
18 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
treatment
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Lithium Carbonate
Intervention Description
To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1
Primary Outcome Measure Information:
Title
NPC clinical severity score
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
7-ketocholesterol
Time Frame
52 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
7 Years
Maximum Age & Unit of Time
40 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
aged from 7 years to 40 years
onset of neurological symptoms prior to 15 years of age
confirmed diagnosis of NPC1 determined by either two NPC1 mutations or one NPC1 mutation with elevated plasma 7-ketocholesterol
ability to walk either independently or with assistance
Subject or parent/guardian able to communicate with investigator, understand and abide the rules of this clinical trial.
parent/guardian able to accompany the subjects to participate in the clinical trial
Subject or parent/guardian must provide written informed consent
Exclusion Criteria:
One NPC clinical severity scale score reached 5
Female in pregnancy or breastfeeding
The predicted life span less than 1 year
Severe liver insufficiency (defined as hepatic laboratory parameters, AST and/or ALT greater than three-times the upper limit of normal
Renal insufficiency, with the serum creatine greater than 1.5 times the upper limit of normal
Free with neurological symptoms
Take antidiuretic hormone
Facility Information:
Facility Name
Xin Hua Hospital
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200092
Country
China
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
34177581
Citation
Han S, Zhang H, Yi M, Liu X, Maegawa GHB, Zou Y, Wang Q, Wu D, Ye Z. Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1. Front Pharmacol. 2021 Jun 9;12:667361. doi: 10.3389/fphar.2021.667361. eCollection 2021.
Results Reference
derived
Learn more about this trial
Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
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