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

September 21, 2019 updated by: Huiwen Zhang, MD, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

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

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

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

18

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Shanghai
      • Shanghai, Shanghai, China, 200092
        • Xin Hua Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

7 years to 40 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: treatment
Drug: Lithium Carbonate
To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
NPC clinical severity score
Time Frame: 52 weeks
52 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
7-ketocholesterol
Time Frame: 52 weeks
52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 5, 2017

Primary Completion (Anticipated)

September 30, 2020

Study Completion (Anticipated)

June 30, 2021

Study Registration Dates

First Submitted

June 26, 2017

First Submitted That Met QC Criteria

June 26, 2017

First Posted (Actual)

June 28, 2017

Study Record Updates

Last Update Posted (Actual)

September 24, 2019

Last Update Submitted That Met QC Criteria

September 21, 2019

Last Verified

September 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • XH-17-010

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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