Study of Lithium Carbonate to Treat Niemann-Pick Type C1... | NCT03201627

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

NCT03201627

Sponsor

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Status

Unknown status

Last Update Posted

Sep 24, 2019Actual

Enrollment

18Estimated

Phase

Early Phase 1

Conditions

Niemann-Pick Disease, Type C1

Interventions

Lithium Carbonate

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Sep 2019

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Active, not recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 5, 2017Actual

Primary Completion Date

Sep 30, 2020Estimated

Completion Date

Jun 30, 2021Estimated

First Submitted Date

Jun 26, 2017

First Submission Date that Met QC Criteria

Jun 26, 2017

First Posted Date

Jun 28, 2017Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 21, 2019

Last Update Posted Date

Sep 24, 2019Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Huiwen Zhang, MD, Associate Prof., Xinhua Hospital, Shanghai Jiao Tong University School of MedicinePrincipal Investigator

Lead Sponsor

Xinhua Hospital, Shanghai Jiao Tong University School of MedicineOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Early Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

18Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
treatment	Experimental		Drug: Lithium Carbonate

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Lithium Carbonate	Drug	To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1	treatment

Measure	Description	Time Frame
NPC clinical severity score		52 weeks

Measure	Description	Time Frame
7-ketocholesterol		52 weeks

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

7 Years

Maximum Age

40 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
Xin Hua Hospital		Shanghai	Shanghai Municipality	200092	China

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
34177581	Derived	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.

ID	Term
D009542	Niemann-Pick Diseases
D013106	Sphingolipidoses
D020140	Lysosomal Storage Diseases, Nervous System
D020739	Brain Diseases, Metabolic, Inborn

ID	Term
D002254	Carbonates
D000468	Alkalies
D007287	Inorganic Chemicals
D002255	Carbonic Acid