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Clinical Trials/EUCTR2014-002550-39-DE
EUCTR2014-002550-39-DEActive, not recruitingPhase 1

4-part, open-label, multicenter, multinational study of thesafety, tolerability, pharmacokinetics, pharmacodynamic, and exploratoryefficacy of venglustat in combination with Cerezyme in adult patients withGaucher disease Type 3 with venglustat monotherapy extension - LEAP

Genzyme Corporation0 sites18 target enrollmentStarted: April 25, 2016Last updated:
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Conditions

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
18
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional clinical trial of medicinal product

Eligibility Criteria

Sex
All

Inclusion Criteria

  • •-The patient must provide written informed consent prior to any studyrelated procedures being performed.
  • •-Has a clinical diagnosis of Gaucher Disease Type 1 (GD1) or Gaucher Disease Type 3 GD3 and documented deficiency of acid beta-glucosidase activity.
  • •-The patient has received treatment with enzyme repalcement therapy for at least 3 years. For at least 6 months prior to enrollment, the Patient has received Cerezyme at a stable monthly dose and must continue at the same monthly dose during the study.
  • •-The patient has reached Gaucher disease therapeutic goals defined as all of the following to be eligible for this study:
  • •-Hemoglobin level of =11.0 g/dL for females and =12.0 g/dL for males.
  • •-Platelet count =100 000/mm^3.
  • •-Spleen volume <10 multiples of normal (MN)MN), or total splenectomy (provided the splenectomy occurred >3 years prior to randomization).
  • •-Liver volume <1.5 MN.
  • •-No bone crisis and free of symptomatic bone disease such as bone pain attributable to osteonecrosis and/or pathological fractures within 3 months prior to screening.
  • •- The patient has maintained GD therapeutic goals defined as all of the following to be eligible for entering Part 4 of this study:
  • •- Hemoglobin level of =11.0 g/dL for females and =12.0 g/dL for males
  • •- Platelet count =100,000/mm3
  • •- Spleen volume <10 multiples of normal (MN), or total splenectomy
  • •- Liver volume <1.5 MN
  • •- No bone crisis and free of symptomatic bone disease such as bone pain attributable to osteonecrosis and/or pathological fractures within 3 months prior to entering Part 4
  • •- The patient, if female and of childbearing potential must have a negative pregnancy test (urine beta-human chorionic gonadotropin [ß-hCG]) at baseline.
  • •-If the patient has a history of seizures, except for myoclonic seizures, they are well controlled under appropriate medication not identified as a strong or moderate inducer or inhibitor of CYP3A.
  • •Adult GD1 cohort only:
  • •-GD1 patient is =18 and =40 years of age.
  • •Adult GD3 cohort only:
  • •-GD3 patient is =18 years of age.
  • •-The patient is willing to abstain from consumption of grapefruit, grapefruit juice, or grapefruit containing products for 72 hours prior to administration of the first dose of GZ/SAR402671 and for the duration of the 156 week treatment period.
  • •-Oculomotor apraxia characterized by a horizontal saccade abnormality.
  • •-Cerezyme treatment every 2 weeks (minimum dose 30 U/kg every 2 weeks).
  • •-Females patients of childbearing potential and males patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use 2 acceptable effective methods of contraception for the duration of the study and for at least 6 weeks for females and 90 days for males following their last dose of study drug.
  • •Are the trial subjects under 18? no
  • •Number of subjects for this age range:
  • •F.1.2 Adults (18-64 years) yes
  • •F.1.2.1 Number of subjects for this age range 18
  • •F.1.3 Elderly (>=65 years) no
  • •F.1.3.1 Number of subjects for this age range

Exclusion Criteria

  • •-Substrate reduction therapy or chaperone therapy for GD within 6 months prior to enrollment.
  • •-The patient has had a partial or total splenectomy within 3 years Prior to randomization.
  • •-The patient is blood transfusion-dependent.
  • •-Prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) or total bilirubin >2 times the upper limit of normal, unless the patient has a diagnosis of Gilbert Syndrome.
  • •-Clinically significant congenital cardiac defect, coronary artery disease, valve disease or left sided heart failure; clinically significant arrhythmias or conduction defect.
  • •-The patient has any clinically significant disease, other than GD, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation.
  • •-The patient has received an investigational product within 30 days Prior to enrollment.
  • •-The patient has a history of cancer, with the exception of basal cell carcinoma.
  • •-The patient has myoclonic seizures.
  • •-The patient is pregnant or lactating.
  • •-The patient has, according to World Health Organization (WHO) Grading, a cortical cataract >onequarter of the lens circumference (Grade cortical cataract-2) or a posterior subcapsular cataract >2 mm (Grade posterior subcapsular cataract-2). Patients with nuclear cataracts will not be excluded.
  • •-The patient requires use of invasive ventilatory support.
  • •-The patient requires use of noninvasive ventilator support while awake for longer than 12 hours daily.
  • •-The patient is unable to receive treatment with Cerezyme due to a known hypersensitivity or is unwilling to receive Cerezyme Treatment every 2 weeks.
  • •-The patient is currently receiving potentially cataractogenic medications as listed in Section 8.8.2.
  • •-The patient has received strong or moderate inducers or inhibitors of Cytochrome p450 Isoform 3A within 30 days or 5 half-lives from screening, whichever is longer, prior to enrolment in Part 2. This also includes the consumption of grapefruit, grapefruit juice, or Grapefruit containing products within 72 hours of starting GZ/SAR402671 administration in Parts 2 and 3.
  • •-The patient is scheduled for in-patient hospitalization including elective surgery, during the study.
  • •-Has had a major organ transplant (eg, bone marrow or liver).
  • •-The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study or unable to undergo study assessments (e.g., contraindications for magnetic resonance imaging).

Investigators

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