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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

About this trial

This is an interventional treatment trial for Niemann-Pick Disease, Type C1

Eligibility Criteria

7 Years - 40 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

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

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

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

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

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