CTRI/2024/08/072953
Recruiting
Phase 3
A Multicenter, Open-Label, Single-Arm, Three-Cohort, Phase 3, Clinical Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Eliglustat Sublingual Film in Patients with Gaucher Disease Type 1 and Type 3.
Amneal Pharmaceuticals Pvt Ltd10 sites in 1 country30 target enrollmentStarted: September 2, 2024Last updated:
Overview
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Amneal Pharmaceuticals Pvt Ltd
- Enrollment
- 30
- Locations
- 10
- Primary Endpoint
- Percentage change in spleen volume (in MN)
Overview
Brief Summary
This study is a phase 3, non-randomized, multicenter, open-label, single-arm, two-cohort study to evaluate the efficacy, safety, and pharmacokinetics (PK) of eliglustat in patients with Gaucher disease Type 1 and Type 3. The primary outcome measure will be the percentage change in spleen volume (in MN) at Week 39 vs. Baseline. The secondary outcomes will include the percentage change in liver volume (in MN) at Weeks 13, 26, and 39 vs. Baseline; absolute change in hemoglobin level at Weeks 13, 26, and 39 vs. Baseline; percentage change in platelet count at Weeks 13, 26, and 39 vs. Baseline; percentage change in spleen volume (in MN) at Weeks 13 and 26 vs. Baseline; comparison of Gaucher disease assessments at Weeks 13, 26, and 39 vs. Baseline; change in Gaucher disease severity score (GD-DS3) at Week 39 vs. Baseline; biomarker assessment (chitotriosidase) at Weeks 4, 13, 26, and 39 vs. Baseline; type, incidence, severity, seriousness, and relatedness of adverse events (AEs); assessment of Cmax, Tmax, AUC0-tau, Vd, CL, and t1/2 on Day 1 for eliglustat; assessment of AUC0-tau, Cmax,ss, Tmax,ss, Vss, CL, and t1/2 at steady state on Day 28 (Week 4), Week 13, and Week 39 for eliglustat; and Cmin,ss (Ctrough,ss) prior to dose on Day 14 (Week 2), Day 28 (Week 4), Week 13, and Week 39 for eliglustat. Percentage change in GL-1 (Glucosylceramide) & Lyso GL-1 (Glucosylsphingosine.) at week 4, 13,26 and 39 vs baseline.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Masking
- None
Eligibility Criteria
- Ages
- 6.00 Year(s) to 64.00 Year(s) (—)
- Sex
- All
Inclusion Criteria
- •The patient (and/or their parent/legal guardian) is willing and able to provide signed informed consent (assent for 12 to 17 years age group) (written assent for 12-17 years age group and oral assent for 6-11 years age group) prior to any study-related procedures to be performed.
- •Male and female patients aged greater than and equal to 18years (cohort I) or 12 to 17 (cohort II) or more than 6 years (Cohort III) completed years at the time of screening.
- •The patient has a diagnosis of Gaucher disease type 1 and Type 3 confirmed by a documented deficiency of acid beta-glucosidase activity by enzyme assay or Glucocerebrosidase (GBA) genotype.
- •The patient has the following symptoms of Gaucher disease during the Screening period: i.
- •At least one of the following laboratory abnormalities: Hemoglobin level of 8.0 to 11.0 g/dL if female or 8.0 to 12.0 g/dL if male Platelet count of 50,000 to 130,000/mm3 ii.
- •Splenomegaly (spleen volume of more than 3 MN).
- •If hepatomegaly is present, the liver volume must be less than 3MN.
- •Consents to provide a blood sample for genotyping for GD, chitotriosidase, and CYP2D6 (to categorize the patients predicted rate of metabolism), unless the patients genotypes for GD, chitotriosidase, and CYP2D6 are already available.
- •Female patients of childbearing potential must have a documented negative pregnancy test prior to enrollment.
- •In addition, all female patients of childbearing potential must use a medically accepted form of contraception throughout the study (either a barrier method or hormonal contraceptive with ethinyl estradiol and norethindrone or similar active components).
Exclusion Criteria
- •The patient has had a partial or total splenectomy.
- •The patient has received substrate reduction therapies for Gaucher disease within 3 months prior to enrollment.
- •The patient has any evidence of moderate to severe uncontrolled neurologic (e.g., peripheral neuropathy, tremor, seizures, Parkinsonism, or cognitive impairment) or pulmonary involvement (e.g., pulmonary hypertension, interstitial lung disease) as related to Gaucher disease.
- •The patient has current symptomatic bone disease such as bone pain attributable to osteonecrosis and/or pathologic fracture, or has had a bone crisis in the 12 months prior to enrollment.
- •The patient is transfusion-dependent.
- •The patient has the following laboratory abnormalities during the Screening period: Hemoglobin level less than 8 g/dL and Platelet count of less than 50,000/mm3
- •The patient has documented anemia due to causes other than Gaucher disease that requires treatment not yet initiated or not yet stable under treatment for at least 3 months (e.g., iron, vitamin B12, and/or folate deficiency) prior to enrollment.
- •The patient has documented thalassemia minor or sickle cell trait with a platelet count of less than 50,000 or greater than 130,000/mm
- •The patient has ever had any radiation treatment in the abdominal region.
- •The patient has documented prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase [ALT]/ aspartate aminotransferase [AST]) or total bilirubin greater than 2 times the upper limit of normal (ULN), unless the patient has a diagnosis of Gilbert Syndrome.
Outcomes
Primary Outcomes
Percentage change in spleen volume (in MN)
Time Frame: Week 39 vs Baseline (day 0)
Secondary Outcomes
- Percentage change in liver volume (in MN)(Week 13, 26 and 39 vs Baseline (day 0))
- Absolute change in Hemoglobin level(Week 13, 26 and 39 vs Baseline (day 0))
- Percentage change in platelet count(Week 13, 26 and 39 vs Baseline (day 0))
- Percentage change in spleen volume (in MN)(Week 13 and Week 26 vs Baseline (day 0))
- Comparison of Gaucher disease assessments(Week 13, Week 26 and Week 39 vs Baseline (day 0))
- Change in Gaucher disease severity score (GD-DS3)(Week 39 and vs Baseline (day 0))
- Biomarkers assessment (chitotriosidase)(Week 4, 13, 26 and 39 vs Baseline (day 0))
- Type, incidence, severity, seriousness, and relatedness of AEs(From initiation (day 0) to closure of study (week 39))
- Assessment of Cmax, Tmax, AUC0-tau, Vd, CL & t1/2(day 1 for (cohort 1)eliglustat)
- Assessment of AUC0-tau, Cmax,ss, Tmax,ss, Vss, CL and t1/2(steady state at day 28 (week 4), Week 13 and Week 39 for eliglustat)
- Cmin,ss (Ctrough,ss) prior to dose(day 14 (Week 2), day 28 (week 4), Week 13 and 39 for eliglustat)
- Cmin,ss (Ctrough,ss) prior to dose & Cpost 1.5h(Day 14 (Week 2), day 28 (week 4), Week 13, Week 26 & 39 for eliglustat for Cohort 3 patients)
- Percentage change in GL-1((glucosylceramide) & lyso-GL-1)
Investigators
Dr Prayag Shah
Amneal Pharmaceuticals Pvt. Ltd.
Study Sites (10)
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