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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03201627
Recruitment Status : Active, not recruiting
First Posted : June 28, 2017
Last Update Posted : September 24, 2019
Sponsor:
Information provided by (Responsible Party):
Huiwen Zhang, MD, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

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

Condition or disease Intervention/treatment Phase
Niemann-Pick Disease, Type C1 Drug: Lithium Carbonate Early Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type
Actual Study Start Date : July 5, 2017
Estimated Primary Completion Date : September 30, 2020
Estimated Study Completion Date : June 30, 2021


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




Primary Outcome Measures :
  1. NPC clinical severity score [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  1. 7-ketocholesterol [ Time Frame: 52 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03201627


Locations
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China, Shanghai
Xin Hua Hospital
Shanghai, Shanghai, China, 200092
Sponsors and Collaborators
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

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Responsible Party: Huiwen Zhang, MD, Associate Prof., Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
ClinicalTrials.gov Identifier: NCT03201627     History of Changes
Other Study ID Numbers: XH-17-010
First Posted: June 28, 2017    Key Record Dates
Last Update Posted: September 24, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pick Disease of the Brain
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Frontotemporal Dementia
Frontotemporal Lobar Degeneration
Dementia
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Metabolic Diseases
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Histiocytosis, Non-Langerhans-Cell
Histiocytosis
Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders
Lithium Carbonate
Antidepressive Agents
Psychotropic Drugs
Enzyme Inhibitors