FULL DETAILS (Read-only)  -> Click Here to Create PDF for Current Dataset of Trial
CTRI Number  CTRI/2024/04/065934 [Registered on: 18/04/2024] Trial Registered Prospectively
Last Modified On: 16/04/2026
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Other 
Public Title of Study   Phase 1a study of VGA039 in healthy volunteers and patients with von Willebrand disease  
Scientific Title of Study   A Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of VGA039 Following Intravenous or Subcutaneous Administration of Single Ascending Doses in Healthy Adults and Adult Patients with von Willebrand Disease  
Trial Acronym  VEGA 
Secondary IDs if Any  
Secondary ID  Identifier 
2022-002504-20  EudraCT 
NCT05776069  ClinicalTrials.gov 
VGA039-CP001,Version 4.0, 05 April 2023  Protocol Number 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name  Dr Shrinath Manikrao Kshirsagar 
Designation  Director-Clinical Trial Research Unit, Consultant Hematologist 
Affiliation  K J Somaiya Super Speciality Hospital & Research Centre 
Address  Advanced Centre for Oncology Haematology and Rare Diseases (ACOHRD) CTRU Third floor Super speciality (SS) building Somaiya Ayurvihar Complex, Eastern Express Highway, Sion (East),Mumbai

Mumbai
MAHARASHTRA
400022
India 
Phone  9821556030  
Fax    
Email  shrinathk2000@gmail.com  
 
Details of Contact Person
Scientific Query  
Name  Sushma Srikanth 
Designation  Manager, Clinical Operations, Project Management Admin 
Affiliation  inVentiv International Pharma Services Private Limited  
Address  4th Floor, Block – 2, DLF Downtown, Commercial Site Block V, DLF City Phase III, Sector 25A, Gurugram, Haryana 122002, India

Gurgaon
HARYANA
12202
India 
Phone  9880665889  
Fax    
Email  sushma.srikanth@syneoshealth.com  
 
Details of Contact Person
Public Query  
Name  Ataur Rahman 
Designation  Mgr,Clinical Operations,Country SSU 
Affiliation  inVentiv International Pharma Services Private Limited  
Address  4th Floor, Block – 2, DLF Downtown, Commercial Site Block V, DLF City Phase III, Sector 25A, Gurugram, Haryana 122002, India

Gurgaon
HARYANA
122002
India 
Phone  8532011805  
Fax    
Email  ataur.rahman@syneoshealth.com  
 
Source of Monetary or Material Support  
Vega Therapeutics, Inc 201 Haskins Way, 5th Floor South San Francisco, CA 94080  
 
Primary Sponsor  
Name  Vega Therapeutics, Inc 
Address  201 Haskins Way, 5th Floor South San Francisco, CA 94080  
Type of Sponsor  Pharmaceutical industry-Global 
 
Details of Secondary Sponsor  
Name  Address 
Ms Inventiv International Pharma Services Private Limited  M/s Inventiv International Pharma Services Private Limited, 4th Floor, Block 2, DLF Downtown, Commercial Site Block V, DLF City Phase III, Sector 25A, Gurugram, Haryana122002, India, Haryana (India) – 122002  
 
Countries of Recruitment     Australia
Austria
Brazil
Canada
India
South Africa
United Kingdom
United States of America  
Sites of Study  
No of Sites = 1  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
DrShrinath Manikrao Kshirsagar   K J Somaiya Super Speciality Hospital & Research Centre  Advanced Centre for Oncology Haematology and Rare Diseases (ACOHRD) CTRU Third floor Super speciality (SS) building Somaiya Ayurvihar Complex, Eastern Express Highway, Sion (East), Mumbai- 400022, Maharashtra, India.
Mumbai
MAHARASHTRA 		 09821556030

shrinathk2000@gmail.com 
 
Details of Ethics Committee
Modification(s)  
No of Ethics Committees= 1  
Name of Committee  Approval Status 
InstitutionalEthicsCommitteeClinicalTrialsKJSomaiyaMedicalCollegeHospitalandResearch Centre   Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Approved/Obtained 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  (1) ICD-10 Condition: D680||Von Willebrands disease,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Intervention  VGA039  part 2A Intravenous Administration, 106 days including End of study visit part 2B Subcutaneous Administration, 57 days including End of study visit. Total duration will be 24 weeks. Part2A 16weeks and Part2B--8weeks  
 
Inclusion Criteria  
Age From  18.00 Year(s)
Age To  60.00 Year(s)
Gender  Both 
Details  Subjects are eligible for the study only if all the following criteria are met, as applicable:
1. Adult male and female subjects, 18 to 60 years of age, inclusive.
2. Female subjects of childbearing potential must have a negative serum pregnancy test during Screening, a negative urine pregnancy test pre-dose on Day 1, and be willing to use a highly effective method of contraception or 2 other methods of contraception, excluding hormonal contraceptives, unless where abstaining from sexual intercourse is in line with the preferred and usual lifestyle of the subject, from the time of administration of the study drug until 3 months post-dose. Note: for the purposes of this study, a female subject of non-childbearing potential is defined as the subject being amenorrhoeic for at least 12 consecutive months or at least 4 months post-surgical sterilization (including bilateral fallopian tube ligation or bilateral oophorectomy with or without hysterectomy) confirmed by follicle-stimulating hormone (FSH) level > 40 mIU/mL at Screening.
3. Male subjects (and partners of childbearing potential) must be willing to use a highly effective method of contraception or 2 other effective methods of contraception, unless anatomically sterile or where abstaining from sexual intercourse is in line with the preferred and usual lifestyle of the subject, from the time of administration of the study drug until 3 months post-dose.
4. No clinically significant history of previous allergy / sensitivity to VGA039 or any of the excipients contained within the investigational medicinal product (IMP).
5. Subjects with negative human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and hepatitis C virus antibody (HCV Ab) test results at Screening. Subjects who are HCV Ab-positive with a HCV RNA test result that is negative/below the limit of detection will be eligible.
6. No clinically significant abnormalities in 12-lead ECG determined at Screening.
7. No clinically significant abnormalities in vital signs (blood pressure, pulse rate,
respiration rate, oral temperature) determined at Screening.
8. Subjects must be available to complete the study (including all follow-up visits).
9. Subjects must provide written informed consent to participate in the study.
10. Subjects must have been vaccinated for COVID-19 (as defined by being at least 2 weeks after receiving a second dose of a COVID-19 vaccine requiring 2 doses for initial immunization, or at least 2 weeks after receiving the only dose of a COVID-19 vaccine requiring a single dose for initial immunization), or have documentation in their medical record of prior COVID-19 infection that started at least 2 weeks prior to Screening and now confers natural immunity.
11. Subjects must have a negative COVID-19 test result within 24 hours before
administration of the study drug.
Additional Inclusion Criteria (for Subjects in Part 1 Only)
12. Healthy volunteers with a body mass index (BMI) of 18-32 kg/m2, inclusive.
13. Hemoglobin level ≥ 12 g/dL and platelet count ≥ 150 × 109/L at Screening.
14. No clinically significant abnormal test results for serum biochemistry, hematology (except hemoglobin level, as above), coagulation, and/or urine analyses at Screening.
15. Subjects with negative urinary drugs of abuse (DOA) screen test results, determined at Screening. A positive test result may be repeated at the Investigator’s discretion.
Additional Inclusion Criteria (for Subjects in Part 2 Only)
16. Documented diagnosis of VWD. Only participants with VWD who are symptomatic, as defined by having a history of bleeding or bruising, are eligible.
17. Hemoglobin level ≥ 8 g/dL and platelet count ≥ 150 × 109/L at Screening.
 
 
ExclusionCriteria 
Details  Subjects meeting ANY of the following exclusion criteria are NOT eligible to be enrolled into the study:
1. Use of prescription or non-prescription drugs, vitamins, herbal and dietary supplements within 28 days or 5 half-lives (whichever is longer) prior to administration of the study drug, that, in the opinion of the Investigator and Sponsor Medical Monitor, will interfere with the study procedures, affect platelet or coagulation function, or compromise subject safety.
2. Use of hormonal contraceptives within 56 days or 5 half-lives (whichever is longer) prior to administration of the study drug.
3. Detection of FV Leiden or Prothrombin G20210A mutation, or laboratory results consistent with protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at Screening.
4. Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation.
5. History of arterial or venous thrombosis, including superficial thrombophlebitis, or embolism.
6. Evidence of renal, hepatic, central nervous system, respiratory, cardiovascular disease, cerebrovascular disease, peripheral vascular disease, or metabolic dysfunction.
7. A clinically significant history of drug or alcohol abuse (defined as the consumption of more than 14 units for male and female subjects of alcohol a week) within the past 2 years.
8. Current tobacco smoker and/or nicotine user, given that these are independent risk factors for thromboembolism.
9. Inability to communicate well with the Investigators (i.e., language problem, poor mental development, or impaired cerebral function).
10. Participation in a new chemical or biological entity clinical study within the previous 3 months or a marketed drug clinical study within the 30 days before the administration of the study drug. (Washout period between studies is defined as the period of time elapsed between the last dose of study drug in the previous study and the first dose of study drug in the next study).
11. Subjects who have received investigational vaccines (except for COVID-19 vaccines only approved for emergency use) or vaccines contraindicated in the
immunocompromised within 28 days before administration of the study drug.
12. Donation of 450 mL or more blood within the month before the administration of the study drug.
13. Any condition in the opinion of the Investigator that may preclude full trial participation and/or completion of assessments for safety, PK, or clinical effect.
14. Subjects with presence or history of malignant tumors within the last 3 years, within the exception of basal cell carcinoma of the skin, if medically controlled.
Additional Exclusion Criteron (Subjects in Part 1 Only)
15. Baseline FVIII activity > 150 IU/dL.
Additional Exclusion Criteria (Subjects in Part 2 Only)
16. Baseline FVIII activity > 50 IU/dL.
17. Any acute, clinically significant bleeding event requiring surgical or procedural
intervention within 7 days prior to receiving study drug.
18. Refusal to receive blood products if clinically indicated. 
 
Method of Generating Random Sequence   Computer generated randomization 
Method of Concealment   Centralized 
Blinding/Masking   Participant and Investigator Blinded 
Primary Outcome  
Outcome  TimePoints 
Incidence of Treatment-Emergent Adverse Events [Safety & tolerability]

Incidence, nature & severity of adverse events (AEs) and serious adverse events (SAEs), including dose-limiting toxicities (DLTs)
 		 From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively  
 
Secondary Outcome  
Outcome  TimePoints 
Plasma Concentrations of single IV & SC doses of VGA039  From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively 
Pharmacodynamics of single IV & SC doses of VGA039  From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively 
Incidence of Anti-drug antibodies to VGA039   From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively 
 
Target Sample Size   Total Sample Size="64"
Sample Size from India="10" 
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" 
Phase of Trial   Phase 1 
Date of First Enrollment (India)   15/05/2024 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  01/04/2024 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="0"
Months="6"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Open to Recruitment 
Recruitment Status of Trial (India)  Open to Recruitment 
Publication Details   N/A 
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Response - NO
Brief Summary  

This is a multi-center, Phase 1a study to assess the safety, tolerability, PK, and PD of VGA039 following single IV or SC dose administration in healthy subjects and Von Willebrand disease patients.

This study consists of 2 parts based on the subject population: Part 1 and Part 2.

Part 1 is a randomized, double-blind, placebo-controlled, single ascending dose (SAD) evaluation of IV or SC VGA039 or placebo in up to 8 cohorts in normal healthy volunteers. This part of the study will not be conducted in India.

 Part 2 is an open-label, SAD of SC and IV VGA039 in up to 8 cohorts of von Willebrand patients. All participants will be enrolled, treated, and followed up for 15 weeks (IV SAD) or 8 weeks(SC SAD).

In total, approximately 64 subjects are planned for Parts 1 and 2.


 
Close