Eliglustat on Gaucher Disease Type IIIB

NCT03519646 | Completed

• 1 other identifier: 201612250MIPB
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Gaucher Disease, Type III

Keywords

Enzyme Replacement Therapy; Eliglustat (Cerdelga®)

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  Number of adverse events in patients.

  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)

  Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.

• Assessment of Gaucher related biomarkers test :Lyso GL1(30% decrease)

  Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.

• Assessment of Gaucher related biomarkers test:Chitotriosidase(30% decrease)

  Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga.

• Change in lymphadenopathy manifestations.

  From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first.

• Pharmacokinetics

  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.

Study Arms (1)

Experimental Case_Eiglustat

EXPERIMENTAL

Besides regular ERT, patients also need to take Eiglustat for 24 months.

Drug: Eliglustat

Interventions

Eliglustat DRUG

1. 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.

Also known as: Cerdelga

Experimental Case_Eiglustat

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 .

Sponsors & Collaborators

• National Taiwan University Hospital: lead
• Sanofi: collaborator

Study Sites (1)

National Taiwan University Hospital

Taipei, 10041, Taiwan

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

eliglustat

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Ni-Chung Lee, M.D., Ph.D

  National Taiwan University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 20, 2018
• First Posted: May 9, 2018
• Study Start: April 23, 2018
• Primary Completion: September 11, 2020
• Study Completion: September 11, 2020
• Last Updated: September 15, 2022

Record last verified: 2021-01

Locations

TW (1)