A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
- Registration Number
- NCT05529992
- Lead Sponsor
- Takeda
- Brief Summary
The main purpose of this study is to observe the side effects of VPRIV in participants with type 1 Gaucher disease who are either treatment-naïve (newly diagnosed) or who are currently being treated with enzyme replacement therapy (ERT).
Participants will receive VPRIV intravenously during the treatment period (up to 51 weeks), followed by the end-of-treatment (EOT) visit after 2 weeks.
- Detailed Description
The drug being tested in this study is called Velaglucerase Alfa (VPRIV). VPRIV will be tested to treat participants with type 1 Gaucher disease. This study will look at the safety, efficacy and pharmacokinetics of VPRIV in the treatment of type 1 Gaucher disease.
The study will enroll approximately 20 participants with Gaucher disease. The study comprises a screening period (Day -21 through Day -4), baseline period (Day -3 through Day 0), treatment period (Week 1 to Week 51), and safety follow-up period. Participants will be assigned to the following drug administration:
• Velaglucerase Alfa (VPRIV)
Participants will receive VPRIV as intravenous (IV) infusion every other week from Week 1 through Week 51 during the Treatment Period. Percentage of participants with at least one serious treatment-emergent adverse event (TEAE) will be evaluated throughout the study.
This multi-center trial will be conducted in China. The overall time to participate in this study is approximately 59 weeks. Participants will make a safety follow-up telephone call or visit to the site after 30 (±7) days of the last infusion of the study drug.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 20
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Velaglucerase Alfa (VPRIV) Velaglucerase Alfa Participants received VPRIV IV infusion at 60 units per kilogram (U/kg) body weight once every other week (EOW) for 60 (+10) minutes for up to 51 weeks.
- Primary Outcome Measures
Name Time Method Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE) Up to 56.2 weeks Adverse event(AE)=any untoward medical occurrence in clinical investigation participant administered a drug;it does not necessarily have to have causal relationship with this treatment. AE can therefore be any unfavorable\&unintended sign (example,clinically significant abnormal laboratory value),symptom/disease temporally associated with use of drug whether or not it is considered related to drug. TEAE=any event emerging or manifesting at or after initiation of investigational product or any existing event that worsens in either intensity or frequency following exposure to investigational product. SAE=any untoward clinical manifestation of signs, symptoms, or outcomes(whether considered related to investigational product or not)\&at any dose: results in death,is life-threatening,requires in-patient hospitalization/prolongation of hospitalization,results in persistent/significant disability/incapacity,results in congenital abnormality/birth defect,or is an important medical event.
- Secondary Outcome Measures
Name Time Method Percentage of Participants With TEAEs Up to 56.2 weeks An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A TEAE is defined as any event emerging or manifesting at or after the initiation of the investigational product or any existing event that worsens in either intensity or frequency following exposure to the investigational product.
Percentage of Participants With Infusion-related Reactions Reported as an Adverse Event Up to 56.2 weeks An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. An infusion-related AE was defined as an AE that 1) began either during or within 12 hours after the start of the infusion and 2) was judged as possibly or probably related to the study treatment.
Percentage of Participants With Development of Anti-VPRIV Antibodies and Neutralizing Antibodies at Week 53 Week 53 Percentages were rounded off to the nearest single decimal place.
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53 Week 53 Clinical laboratory assessments included hematology and serum chemistry. Any clinically significant changes in the clinical laboratory assessment values based on the investigator's interpretation were reported. Only categories with at least one participant with event are presented.
Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis Week 53 Any abnormal changes in the urinalysis assessment values based on the investigator's interpretation were reported. Urinalysis comprised of the following parameters: bilirubin, ketones, glucose, nitrite, occult blood, protein, and urobilinogen.
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53 Week 53 Participants with atleast 1 abnormal change (above or below normal) in an infusion vital sign parameter of pulse, temperature, respiratory rate,\& blood pressure were reported. Normal ranges for each vital sign parameter were, pulse (beats per minutes \[bpm\]): 40-100(≥12 years old), 55-95(≥6 but \<12 years old), 65-110(≥2 but \<6 years old); body temperature(degree Celsius\[˚C\]): 36.5 to 37.2(all age groups),respiration rate(breaths/minutes):12-24(≥12 years old),12-22 (≥6 but \<12 years old),20-30(≥2 but \<6 years old);systolic blood pressure(BP) \[millimeters of mercury{mm Hg}\]: high: ≥140 (≥18 years old), ≥20+ 80+ 2\*age(\<18 years old), low: \<90 (≥18 years old), \< -20+ 80+ 2\*age(\<18 years old); diastolic BP(mm Hg):high:≥90 (≥18 years old), ≥20+ (80+2\*age)\*(2/3) (\<18 years old) low: \<50 (≥18 years old), \< -20+ (80+2\*age)\*(2/3) (\<18 years old). As per planned analysis,data was collected in a combined manner for all participants irrespective of age. Only non-zero categories are presented.
Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Measurements at Week 53 Week 53 Participants with clinically significant changes in any ECG measurement, such as PR, QRS, QT, corrected QT intervals, and heart rate based on the investigator's interpretation were reported.
Change From Baseline to Week 53 in Hemoglobin Concentration Baseline, Week 53 Change From Baseline to Week 53 in Platelet Count Baseline, Week 53 Change From Baseline to Week 53 in Normalized Liver Volume Baseline, Week 53 Normalized liver volume was measured by abdominal magnetic resonance imaging (MRI) or computed tomography (CT) scan. Liver volume was normalized for percent body weight.
Change From Baseline to Week 53 in Normalized Spleen Volume Baseline, Week 53 Spleen volume was normalized for percent body weight.
Change From Baseline to Week 53 in 36-item Short Form General Health Survey (SF-36) Scores Baseline, Week 53 SF-36 Version 2 is a multipurpose, participant completed, short-form health survey with 36 questions that consists of an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. Physical component summary (PCS) is mostly contributed by physical function (PF), role physical (RP), bodily pain (BP), and general health (GH). Mental component summary (MCS) is mostly contributed by mental health (MH), role emotional (RE), social function (SF), and vitality (VT). Each component on the SF-36 item health survey is scored from 0 (best) to 100 (worst). Total score ranges from 0-100 for each component summary (i.e., PCS and MCS), where higher scores are associated with less disability and better quality of life.
Change From Baseline to Week 53 in Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50) Scores Baseline, Week 53 The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The 50 questions measure 14 domains (global health, physical functioning, role/social limitations (emotional/behavioral and physical), bodily pain/discomfort, behavior, global behavior, mental health, self-esteem, general health perceptions, change in health, parental impact (emotional and time), family activities, and family cohesion) which were summarized as the physical and psychological summary scores. Each summary score was transformed and could range from 0 to 100, with higher score indicating better physical and psychosocial health. A positive change from Baseline indicates improved well-being.
Cmax: Maximum Observed Serum Concentration for VPRIV at Week 1 Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 Serum Concentration for VPRIV at Week 37 Within 3 minutes prior to the end of the 60-minute infusion at Week 37 Tmax: Time to Reach the Maximum Serum Concentration (Cmax) for VPRIV Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 AUCinf: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity for VPRIV Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 ng\*min/mL denotes nanograms\*minutes per milliliter.
Terminal Phase Elimination Half-life (T1/2) for VPRIV Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 Oral Clearance (CL) for VPRIV Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 mL/min/kg denotes milliliters per minutes per kilogram.
Apparent Steady-state Volume of Distribution (Vss) for VPRIV Pre-dose and at multiple timepoints(up to 120 minutes post-dose on Day 1 of Week 1 Percent Change From Baseline to Week 53 in Biomarker: Plasma Chemokine [C-C Motif] Ligand 18 Baseline, Week 53 Percent Change From Baseline to Week 53 in Biomarker: Glucopsychosine Baseline, Week 53
Trial Locations
- Locations (10)
Lanzhou University Second Hospital
🇨🇳Lanzhou, Gansu, China
The First Affiliated Hospital, Sun Yat-sen University
🇨🇳Guangzhou, Guangdong, China
Beijing Children's Hospital, Capital Medical University
🇨🇳Beijing, Beijing, China
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
🇨🇳Beijing, Beijing, China
The Second Hospital of Hebei Medical University
🇨🇳Shijiazhuang, Hebei, China
Guangzhou Women and Children's Medical Center
🇨🇳Guangzhou, Guangdong, China
Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology
🇨🇳Wuhan, Hubei, China
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
🇨🇳Tianjin, Tianjin, China
Chinese PLA General Hospital
🇨🇳Beijing, Beijing, China
Nanjing Children's Hospital
🇨🇳Nanjing, Jiangsu, China