Gaucher Disease, Type IIINCT03519646
Eliglustat on Gaucher Disease Type IIIB
A study testing Eliglustat in people with Gaucher Disease, Type III, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases. Read more about this study
- Who is it for?
- Ages 6 years and olderHealthy volunteers can take part
- Conditions the study is for
- Gaucher Disease, Type III
- What is being tested?
- Eliglustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteTaiwan
- Study dates
- 23 Apr 2018to 11 Sep 2020
- Phase
- Interventional
- 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 6 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Gaucher disease patients diagnosed by low B-glucocerebrosidase deficiency and GBA mutation .
- The participant is at least 6 years old at time of enrollment.
- Under stable Cerezyme dosage for at least for 3 months.
- Presence of lymphadenopathy.
- Patient (and/or their parent/legal guardian) is willing to participate and able to provide signed informed consent.
Who cannot join
- The participant is CYP2D6 ultra-rapid metabolizer.
- The participant had received substrate reduction therapy for Gaucher disease within 3 months of enrollment.
- The participant had any clinically significant disease other than GD, including cardiovascular (especially arrhythmia), renal, liver, pulmonary, endocrinopathy, hypokalemia, or hypomagnesemia that may confound the study result.
- The participant is pregnant or lactating.
- The participant is known to be allergy to Cerdelga.
- The participant use drugs that will strongly inhibit CYP2D6 or CYP3A activity .
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.
Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases.
Read the detailed description
- This is a 3-year study and the enrollment time of this study is 24 months.
- The participants have to receive the investigational agent:Cerdelga.(Cerdelga have 21 mg、42 mg and 84 mg capsule.)
- The participants have to go back to the hospital and receive the investigational agent and take the Gaucher related biomarkers test before receiving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga.
- The participants have to inform if any adverse events happened.
- The investigators will follow up by phone if adverse events happened in the participants after one months start the treatment.
- Conditions
- Gaucher Disease, Type III
- Being tested
- Eliglustat
- Keywords
- Enzyme Replacement Therapy, Eliglustat (Cerdelga®)
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EliglustatDrug
Also called Cerdelga
- This is a 3-year study and the enrollment time of this study is 24 months.
- The participants have to receive the investigational agent, Cerdelga
- The participants have to go back to the hospital and receive the investigational agent and take the test before receving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga.
- IMP Administration Method: Cerdelga have 21 mg、42 mg and 84 mg capsule.
- No need for fasting before use, but can't take with grapefruit juice.
- Five ml Blood and 10 ml urine shoud be taken before receiving Cerdelga, and 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga for Gaucher related biomarkers test.
| Study group | Role | What they receive |
|---|---|---|
| Experimental Case_Eiglustat | Experimental | Besides regular ERT, patients also need to take Eiglustat for 24 months. Receives: Drug: Eliglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of adverse events in patients.
Measured: From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Secondary outcomes (5)
Assessment of Gaucher related biomarkers test :CCL18 (30% decrease)
Measure of Gaucher disease type I biomarkers:CCL18(ng/ml) in plasma.
Measured: Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Assessment of Gaucher related biomarkers test :Lyso GL1(30% decrease)
Measure of Gaucher disease type I biomarkers:Lyso GL1 (ng/mL) in plasma
Measured: Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Assessment of Gaucher related biomarkers test:Chitotriosidase(30% decrease)
Measure of Gaucher disease type I biomarkers:Chitotriosidase(nmol/ml/h) in plasma.
Measured: Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Change in lymphadenopathy manifestations.
Physician will pay close attention to the lymphadenopathy including size, location, and number of enlarged lymph nodes, evaluate by palpation and radiological (MRI examination). The total size of the lympadenopathy will be combined into one report as "Total size" cm^3.
Measured: From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Pharmacokinetics
Eliglustat plasma concentration over time
Measured: Plasma concentration-time data will be obtained pre-dose (within 30 minutes prior to dosing) and at 1, 2, 6, 12, 26, 36 hours after 1st dosing, and 1,3,6,12,18 and 24 months thereafter.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2018
- Study start23 Apr 2018
- Primary completion11 Sep 2020
- Study completion11 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease, Type IIIStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Sanofi
- Responsible party
- Sponsor
- Investigators
- Ni-Chung Lee, M.D., Ph.D, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03519646
- Sponsor's protocol ID
- 201612250MIPB
- Status last verified
- Jan 2021
- First submitted
- 20 Mar 2018
- Last updated
- 15 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.
- ClinicalTrials.gov ID
- NCT03519646
- Acronym / sponsor ID
- 201612250MIPB
- Conditions
- Gaucher Disease, Type III
- Keywords
- Enzyme Replacement Therapy, Eliglustat (Cerdelga®)
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Collaborators
- Sanofi
- Study start
- 23 Apr 2018 (actual)
- Primary completion
- 11 Sep 2020 (actual)
- Study completion
- 11 Sep 2020 (actual)
- First posted
- 9 May 2018 (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.
Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases.
Detailed description
- This is a 3-year study and the enrollment time of this study is 24 months.
- The participants have to receive the investigational agent:Cerdelga.(Cerdelga have 21 mg、42 mg and 84 mg capsule.)
- The participants have to go back to the hospital and receive the investigational agent and take the Gaucher related biomarkers test before receiving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga.
- The participants have to inform if any adverse events happened.
- The investigators will follow up by phone if adverse events happened in the participants after one months start the treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental Case_Eiglustat | Experimental | Drug: EliglustatBesides regular ERT, patients also need to take Eiglustat for 24 months. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Gaucher disease patients diagnosed by low B-glucocerebrosidase deficiency and GBA mutation .
- The participant is at least 6 years old at time of enrollment.
- Under stable Cerezyme dosage for at least for 3 months.
- Presence of lymphadenopathy.
- Patient (and/or their parent/legal guardian) is willing to participate and able to provide signed informed consent.
Key exclusion criteria (6 listed)
- The participant is CYP2D6 ultra-rapid metabolizer.
- The participant had received substrate reduction therapy for Gaucher disease within 3 months of enrollment.
- The participant had any clinically significant disease other than GD, including cardiovascular (especially arrhythmia), renal, liver, pulmonary, endocrinopathy, hypokalemia, or hypomagnesemia that may confound the study result.
- The participant is pregnant or lactating.
- The participant is known to be allergy to Cerdelga.
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 |
|---|---|---|
| EliglustatAlso: Cerdelga1. This is a 3-year study and the enrollment time of this study is 24 months. 2. The participants have to receive the investigational agent, Cerdelga 3. The participants have to go back to the hospital and receive the investigational agent and take the test before receving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga. 4. IMP Administration Method: Cerdelga have 21 mg、42 mg and 84 mg capsule. 5. No need for fasting before use, but can't take with grapefruit juice. 6. Five ml Blood and 10 ml urine shoud be taken before receiving Cerdelga, and 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga for Gaucher related biomarkers test. | Drug | Experimental Case_Eiglustat |
Primary outcome
Adverse Events
Time frame: From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Number of adverse events in patients.
5 secondary outcomes
Assessment of Gaucher related biomarkers test :CCL18 (30% decrease)
Time frame: Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Assessment of Gaucher related biomarkers test :Lyso GL1(30% decrease)
Time frame: Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Assessment of Gaucher related biomarkers test:Chitotriosidase(30% decrease)
Time frame: Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.
Change in lymphadenopathy manifestations.
Time frame: From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.
Pharmacokinetics
Time frame: Plasma concentration-time data will be obtained pre-dose (within 30 minutes prior to dosing) and at 1, 2, 6, 12, 26, 36 hours after 1st dosing, and 1,3,6,12,18 and 24 months thereafter.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2022.
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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.
Ni-Chung Lee, M.D., Ph.D principal investigator
National Taiwan University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Eliglustat in people with Gaucher Disease, Type III, run by National Taiwan University Hospital at 1 site in Taiwan.
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 5 inclusion and 6 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: finished, as of the sponsor's last update on 15 Sep 2022. 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 Taiwan. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Adverse Events (time frame: From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Sep 2022.