Niemann-Pick Disease, Type C1Early Phase 1NCT03201627
Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
A Early Phase 1 study testing Lithium Carbonate in people with Niemann-Pick Disease, Type C1, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial summary
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1. Read more about this study
- Who is it for?
- Ages 7 to 40 years
- Conditions the study is for
- Niemann-Pick Disease, Type C1
- What is being tested?
- Lithium Carbonate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 5 Jul 2017to 30 Jun 2021
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
- Conditions
- Niemann-Pick Disease, Type C1
- Being tested
- Lithium Carbonate
- Medical subject headings
- Niemann-Pick Disease, Type C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lithium CarbonateDrug
To observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1
| Study group | Role | What they receive |
|---|---|---|
| treatment | Experimental | Not described Receives: Drug: Lithium Carbonate |
What is being measured
The primary outcome is the main question the study is designed to answer.
NPC clinical severity score
Measured: 52 weeks
Secondary outcomes (1)
7-ketocholesterol
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2017
- Study start5 Jul 2017
- Primary completion30 Sep 2020 (estimated)
- Study completion30 Jun 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Niemann-Pick Disease, Type C1Studies of the same condition, open and under way.
- More trials run by Xinhua Hospital, Shanghai Jiao Tong University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Huiwen Zhang, MD, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03201627
- Sponsor's protocol ID
- XH-17-010
- Status last verified
- Sep 2019
- First submitted
- 26 Jun 2017
- Last updated
- 24 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 34177581
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- a Single-center, Prospective, Open, and Non-randomized Case-control Study of Lithium Carbonate Effect on Niemann Disease C1 Type
- ClinicalTrials.gov ID
- NCT03201627
- Acronym / sponsor ID
- XH-17-010
- Conditions
- Niemann-Pick Disease, Type C1
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Study start
- 5 Jul 2017 (actual)
- Primary completion
- 30 Sep 2020 (estimated)
- Study completion
- 30 Jun 2021 (estimated)
- First posted
- 28 Jun 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| treatment | Experimental | Drug: Lithium Carbonate |
Who may qualify
- Ages
- 7 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lithium CarbonateTo observe whether lithium carbonate has protective role on the brain of Niemann-Pick disease C1 | Drug | treatment |
Primary outcome
NPC clinical severity score
Time frame: 52 weeks
1 secondary outcome
7-ketocholesterol
Time frame: 52 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2019.
- Derived from this study: PubMed 34177581Han 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Lithium Carbonate in people with Niemann-Pick Disease, Type C1, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
NPC clinical severity score (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03201627, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2019.