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A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Primary Purpose

Gaucher Disease

Status

Recruiting

Phase

Phase 3

Locations

China

Study Type

Interventional

Intervention

Velaglucerase Alfa

About this trial

This is an interventional treatment trial for Gaucher Disease focused on measuring VPRIV, Gaucher Disease

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion:

Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator:
1. Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or
2. Decreased GCB activity level that is >30% of normal, but with confirmation of genetic mutation test
Is at least 2 years of age, inclusive, at screening
Is naive to treatment for Gaucher disease (Has not received treatment for Gaucher disease [investigational or approved products] within the 12 months prior to screening) OR Is receiving or has recently received Imiglucerase ERT (Has received Imiglucerase treatment within the 12 months prior to screening and not within the 14 days prior to screening)
Has Gaucher disease-related hematological abnormalities, defined as
1. Hemoglobin levels of ≥1 g/dL below the lower limit of normal for their age and gender AND/OR
2. A platelet count of <90 × 10^9/L below the lower limit of normal for their age and gender
Has Gaucher disease-related viscera abnormalities, defined as the following:
- Participant has at least moderate splenomegaly, assessed by palpation (2 to 3 cm below the left costal margin), or by abdominal radiology scan (magnetic resonance imaging [MRI] or computed tomography [CT] scan, with spleen volume >5 times normal) AND/OR
- Participant has hepatomegaly, assessed by palpation or by abdominal radiology scan (MRI or CT scan); Participants who have undergone splenectomy must have satisfied these criteria for this study.

Exclusion:

Has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease as assessed by the investigator
Has had a splenectomy or an active, clinically significant spleen infarction within the 12 months prior to screening
Has received treatment with any investigational drug or device within 30 days prior to screening, or within 5 half-lives of that investigational product, whichever is greater; such treatment during the study will not be permitted
Is currently receiving red blood cell growth factor (eg, erythropoietin), chronic systemic corticosteroids, or has been on such treatment within the 6 months prior to screening
Presents with non-Gaucher disease related exacerbated anemia at screening
Has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)

Sites / Locations

Chinese PLA General HospitalRecruiting
Beijing Children's Hospital, Capital Medical UniversityRecruiting
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Lanzhou University Second Hospital
The First Affiliated Hospital, Sun Yat-sen UniversityRecruiting
Guangzhou Women and Children's Medical CenterRecruiting
The Second Hospital of Hebei Medical University
Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology
Nanjing Children's Hospital
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical SciencesRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Velaglucerase Alfa (VPRIV)

Arm Description

Participants will receive VPRIV intravenous infusion once every other week (EOW) for 60 (+10) minutes as per physician treatment plan for up to 51 weeks.

Outcomes

Primary Outcome Measures

Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Events (TEAE) Throughout the Study

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. An SAE is any untoward clinical manifestation of signs, symptoms, or outcomes (whether considered related to the investigational product or not) and at any dose: results in death, is life-threatening, requires in-patient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, or is an important medical event.

Secondary Outcome Measures

Percentage of Participants With TEAEs

Percentage of Participants With Infusion-related Reactions Throughout the Study Reported as Adverse Event

An infusion-related AE is defined as an AE that 1) begins either during or within 12 hours after the start of the infusion and 2) is judged as possibly or probably related to the study treatment.

Percentage of Participants With Development of anti-VPRIV Antibodies, Including Neutralizing Antibodies Throughout the Study

Participants with the development of anti-VPRIV antibodies, including neutralizing antibodies will be assessed.

Number of Participants With Clinically Significant Changes in Laboratory Assessments, Vital Signs Measurements, and Electrocardiogram (ECG) Measurements

Number of participants with clinically significant changes in laboratory parameters, vital signs, and 12-Lead electrocardiogram (ECG) findings will be reported.

Change from Baseline to Week 53 in Hemoglobin Concentration

Change from Baseline to Week 53 in Platelet Count

Change from Baseline to Week 53 in Normalized Liver Volume

Normalized liver volume will be measured by abdominal magnetic resonance imaging (MRI) or computed tomography (CT) scan. Liver volume will be normalized for percent (%) body weight.

Change from Baseline to Week 53 in Normalized Spleen Volume

Normalized spleen volume will be measured by abdominal MRI or CT scan. Spleen volume will be normalized for % body weight.

Change from Baseline to Week 53 in Quality of Life (QoL) Questionnaire Assessment as Measured by Short Form-36 (SF-36), Version 2

Participants greater than or equal to (≥)18 years-old will complete the SF-36 questionnaire. QoL assessment is not applicable for participants <5 years of age. The SF-36, version 2 is a self-administered, validated questionnaire designed to measure generic health-related QoL. This 36-item questionnaire measures 8 domains, including physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. Two summary scores, including the Physical Component Score and Mental Component Score, will be calculated ranging from 0 (worst) to 100 (best). Higher scores indicate better health status.

Change from Baseline to Week 53 in QoL Questionnaire Assessment as Measured by Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50)

Participants from >5 and <18 years of age will complete the CHQ-PF50. QoL assessment is not applicable for participants <5 years of age. The CHQ-PF50 will assess the parent(s) or caregiver's impression of the following scales: 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. The total score will be calculated ranging from 0 (worst) to 100 (best). An increase in the CHQ-PF50 score indicates a more favorable assessment by the proxy of the child's health and/or well-being.

Cmax: Maximum Observed Serum Concentration for VPRIV at Week 1

Cmax: Maximum Observed Serum Concentration for VPRIV at Week 37

Tmax: Time to Reach the Maximum Serum Concentration (Cmax) for VPRIV

AUCinf: Area Under the Plasma Concentration-time Curve from Time 0 to Infinity for VPRIV

Terminal Phase Elimination Half-life (T1/2) for VPRIV

Oral Clearance (CL) for VPRIV

Apparent Steady-state Volume of Distribution (Vss) for VPRIV

Percentage Change from Baseline to Week 53 in Biomarker: Plasma Chemokine [C-C motif] Ligand 18

Percentage Change from Baseline to Week 53 in Biomarker: Glucosylsphingosine

Full Information

NCT ID

NCT05529992

First Posted

September 2, 2022

Last Updated

January 10, 2023

Sponsor

Takeda

1. Study Identification

Unique Protocol Identification Number

NCT05529992

Brief Title

A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Official Title

A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease

Study Type

Interventional

2. Study Status

Record Verification Date

January 2023

Overall Recruitment Status

Recruiting

Study Start Date

January 3, 2023 (Actual)

Primary Completion Date

December 4, 2024 (Anticipated)

Study Completion Date

December 4, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Takeda

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Gaucher Disease

Keywords

VPRIV, Gaucher Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Velaglucerase Alfa (VPRIV)

Arm Type

Experimental

Arm Description

Participants will receive VPRIV intravenous infusion once every other week (EOW) for 60 (+10) minutes as per physician treatment plan for up to 51 weeks.

Intervention Type

Drug

Intervention Name(s)

Velaglucerase Alfa

Other Intervention Name(s)

VPRIV

Intervention Description

VPRIV intravenous infusion every other week for 60 minutes.

Primary Outcome Measure Information:

Title

Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Events (TEAE) Throughout the Study

Description

Time Frame

Up to 59 weeks

Secondary Outcome Measure Information:

Title

Percentage of Participants With TEAEs

Description

Time Frame

Up to 59 weeks

Title

Percentage of Participants With Infusion-related Reactions Throughout the Study Reported as Adverse Event

Description

An infusion-related AE is defined as an AE that 1) begins either during or within 12 hours after the start of the infusion and 2) is judged as possibly or probably related to the study treatment.

Time Frame

Up to 59 weeks

Title

Percentage of Participants With Development of anti-VPRIV Antibodies, Including Neutralizing Antibodies Throughout the Study

Description

Participants with the development of anti-VPRIV antibodies, including neutralizing antibodies will be assessed.

Time Frame

Up to 59 weeks

Title

Number of Participants With Clinically Significant Changes in Laboratory Assessments, Vital Signs Measurements, and Electrocardiogram (ECG) Measurements

Description

Number of participants with clinically significant changes in laboratory parameters, vital signs, and 12-Lead electrocardiogram (ECG) findings will be reported.

Time Frame

Up to 59 weeks

Title

Change from Baseline to Week 53 in Hemoglobin Concentration

Time Frame

Baseline, up to Week 53

Title

Change from Baseline to Week 53 in Platelet Count

Time Frame

Baseline, up to Week 53

Title

Change from Baseline to Week 53 in Normalized Liver Volume

Description

Normalized liver volume will be measured by abdominal magnetic resonance imaging (MRI) or computed tomography (CT) scan. Liver volume will be normalized for percent (%) body weight.

Time Frame

Baseline, up to Week 53

Title

Change from Baseline to Week 53 in Normalized Spleen Volume

Description

Normalized spleen volume will be measured by abdominal MRI or CT scan. Spleen volume will be normalized for % body weight.

Time Frame

Baseline, up to Week 53

Title

Change from Baseline to Week 53 in Quality of Life (QoL) Questionnaire Assessment as Measured by Short Form-36 (SF-36), Version 2

Description

Time Frame

Baseline, up to Week 53

Title

Change from Baseline to Week 53 in QoL Questionnaire Assessment as Measured by Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50)

Description

Time Frame

Baseline, up to Week 53

Title

Cmax: Maximum Observed Serum Concentration for VPRIV at Week 1

Time Frame

Pre-dose and at multiple timepoints (up to 120 minutes) post-dose on Day 1 of Week 1

Title

Cmax: Maximum Observed Serum Concentration for VPRIV at Week 37

Time Frame

Post-dose (up to 60 minutes) of Week 37

Title

Tmax: Time to Reach the Maximum Serum Concentration (Cmax) for VPRIV

Time Frame

Pre-dose and at multiple timepoints (up to 120 minutes) post-dose on Day 1 of Week 1

Title

AUCinf: Area Under the Plasma Concentration-time Curve from Time 0 to Infinity for VPRIV

Time Frame

Pre-dose and at multiple timepoints (up to 120 minutes) post-dose on Day 1 of Week 1

Title

Terminal Phase Elimination Half-life (T1/2) for VPRIV

Time Frame

Pre-dose and at multiple timepoints (up to 120 minutes) post-dose on Day 1 of Week 1

Title

Oral Clearance (CL) for VPRIV

Time Frame

Pre-dose and at multiple timepoints (up to 120 minutes) post-dose on Day 1 of Week 1

Title

Apparent Steady-state Volume of Distribution (Vss) for VPRIV

Time Frame

Pre-dose and at multiple timepoints (up to 120 minutes) post-dose on Day 1 of Week 1

Title

Percentage Change from Baseline to Week 53 in Biomarker: Plasma Chemokine [C-C motif] Ligand 18

Time Frame

Baseline, up to Week 53

Title

Percentage Change from Baseline to Week 53 in Biomarker: Glucosylsphingosine

Time Frame

Baseline, up to Week 53

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion: Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator: Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or Decreased GCB activity level that is >30% of normal, but with confirmation of genetic mutation test Is at least 2 years of age, inclusive, at screening Is naive to treatment for Gaucher disease (Has not received treatment for Gaucher disease [investigational or approved products] within the 12 months prior to screening) OR Is receiving or has recently received Imiglucerase ERT (Has received Imiglucerase treatment within the 12 months prior to screening and not within the 14 days prior to screening) Has Gaucher disease-related hematological abnormalities, defined as Hemoglobin levels of ≥1 g/dL below the lower limit of normal for their age and gender AND/OR A platelet count of <90 × 10^9/L below the lower limit of normal for their age and gender Has Gaucher disease-related viscera abnormalities, defined as the following: Participant has at least moderate splenomegaly, assessed by palpation (2 to 3 cm below the left costal margin), or by abdominal radiology scan (magnetic resonance imaging [MRI] or computed tomography [CT] scan, with spleen volume >5 times normal) AND/OR Participant has hepatomegaly, assessed by palpation or by abdominal radiology scan (MRI or CT scan); Participants who have undergone splenectomy must have satisfied these criteria for this study. Exclusion: Has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease as assessed by the investigator Has had a splenectomy or an active, clinically significant spleen infarction within the 12 months prior to screening Has received treatment with any investigational drug or device within 30 days prior to screening, or within 5 half-lives of that investigational product, whichever is greater; such treatment during the study will not be permitted Is currently receiving red blood cell growth factor (eg, erythropoietin), chronic systemic corticosteroids, or has been on such treatment within the 6 months prior to screening Presents with non-Gaucher disease related exacerbated anemia at screening Has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Takeda Contact

Phone

+1-877-825-3327

medinfoUS@takeda.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Study Director

Organizational Affiliation

Takeda

Official's Role

Study Director

Facility Information:

Facility Name

Chinese PLA General Hospital

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100039

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 134 26309517

mystong@163.com

First Name & Middle Initial & Last Name & Degree

Yan Meng

Facility Name

Beijing Children's Hospital, Capital Medical University

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100045

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 133 31119559

wangtianyou@bch.com.cn

First Name & Middle Initial & Last Name & Degree

Tianyou Wang

Facility Name

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100730

Country

China

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 133 71628442

Zhengqingqiu33@aliyun.com

First Name & Middle Initial & Last Name & Degree

Zhengqing Qiu

Facility Name

Lanzhou University Second Hospital

City

Lanzhou

State/Province

Gansu

ZIP/Postal Code

730030

Country

China

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 139 19209600

zhangliansheng2021@126.com

First Name & Middle Initial & Last Name & Degree

Liansheng Zhang

Facility Name

The First Affiliated Hospital, Sun Yat-sen University

City

Guangzhou

State/Province

Guangdong

ZIP/Postal Code

510080

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 189 02233573

l-xuequn@126.com

First Name & Middle Initial & Last Name & Degree

Xuequn Luo

Facility Name

Guangzhou Women and Children's Medical Center

City

Guangzhou

State/Province

Guangdong

ZIP/Postal Code

510623

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 156 02309257

zhw2001zhw@163.com

First Name & Middle Initial & Last Name & Degree

Wen Zhang

Facility Name

The Second Hospital of Hebei Medical University

City

Shijiazhuang

State/Province

Hebei

ZIP/Postal Code

050000

Country

China

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 158 03210962

zhanglihui10510@163.com

First Name & Middle Initial & Last Name & Degree

Lihui Zhang

Facility Name

Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology

City

Wuhan

State/Province

Hubei

ZIP/Postal Code

430030

Country

China

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 133 87522645

xpluo@tjh.tjmu.edu.cn

First Name & Middle Initial & Last Name & Degree

Xiaoping Luo

Facility Name

Nanjing Children's Hospital

City

Nanjing

State/Province

Jiangsu

ZIP/Postal Code

210008

Country

China

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 189 51769586

fyj322@189.cn

First Name & Middle Initial & Last Name & Degree

Yongjun Fang

Facility Name

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

City

Tianjin

State/Province

Tianjin

ZIP/Postal Code

300020

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Site Contact

Phone

86 138 21700281

fkzhang@ihcams.ac.cn

First Name & Middle Initial & Last Name & Degree

Fengkui Zhang

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

IPD Sharing Access Criteria

IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

IPD Sharing URL

https://vivli.org/ourmember/takeda/

Links:

URL

https://clinicaltrials.takeda.com/study-detail/fba4cdf09dab492e?idFilter=%5B%22TAK-669-3001%22%5D

Description

To obtain more information on the study, click here/on this link

Learn more about this trial

A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

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