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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

EARLY_PHASE1

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-05

Study Completion Date

2021-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

NCT03887533

Niemann-Pick Disease, Type C1

TERMINATED PHASE1/PHASE2

Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid

NCT01033331

Spinal Muscular Atrophy

COMPLETED

Clinical Study to Investigate the Efficacy and Safety of NT 201 Compared to Placebo in the Treatment of Chronic Troublesome Drooling Associated With Neurological Disorders and/or Intellectual Disability

NCT02270736

Chronic Troublesome Sialorrhea Cerebral Palsy Stroke +2 more

COMPLETED PHASE3

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

NCT01747135

Niemann-Pick Disease, Type C1

COMPLETED PHASE1

Recurrent Crying Spells in Cerebral Palsy

NCT01955655

Recurrent Crying Spells

COMPLETED PHASE4

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Niemann-Pick Disease, Type C1

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

treatment

Group Type EXPERIMENTAL

Lithium Carbonate

Intervention Type DRUG

To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Lithium Carbonate

To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

7 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No