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Completed

NCT Number: NCT03519646

Eliglustat on Gaucher Disease Type IIIB

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.

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Key information

Conditions

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

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

National Taiwan University Hospital

Taipei, 10041, Taiwan

About this study

  • 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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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 .

Treatment and study plan

Eliglustat

Drug
  • 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.

Other names: Cerdelga

Primary outcomes

  1. 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.

Secondary outcomes

  1. Assessment of Gaucher related biomarkers test :CCL18 (30% decrease)

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

    Measure of Gaucher disease type I biomarkers:CCL18(ng/ml) in plasma.

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

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

    Measure of Gaucher disease type I biomarkers:Lyso GL1 (ng/mL) in plasma

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

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

    Measure of Gaucher disease type I biomarkers:Chitotriosidase(nmol/ml/h) in plasma.

  4. 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.

    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.

  5. 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.

    Eliglustat plasma concentration over time

Sponsors and collaborators

Lead sponsor

National Taiwan University Hospital

Other

Collaborators

  • Sanofi

Registry information

Official study 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.

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
May 9, 2018
Registry last updated
Sep 15, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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