Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type
1 other identifier
interventional
18
1 country
1
Brief Summary
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for early_phase_1
Started Jul 2017
Longer than P75 for early_phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 26, 2017
CompletedFirst Posted
Study publicly available on registry
June 28, 2017
CompletedStudy Start
First participant enrolled
July 5, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
June 30, 2021
CompletedSeptember 24, 2019
September 1, 2019
3.2 years
June 26, 2017
September 21, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
NPC clinical severity score
52 weeks
Secondary Outcomes (1)
7-ketocholesterol
52 weeks
Study Arms (1)
treatment
EXPERIMENTALInterventions
To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Xin Hua Hospital
Shanghai, Shanghai Municipality, 200092, China
Related Publications (1)
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.
PMID: 34177581DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- early phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Associate Prof.
Study Record Dates
First Submitted
June 26, 2017
First Posted
June 28, 2017
Study Start
July 5, 2017
Primary Completion
September 30, 2020
Study Completion
June 30, 2021
Last Updated
September 24, 2019
Record last verified: 2019-09
Data Sharing
- IPD Sharing
- Will not share