| CTRI Number |
CTRI/2024/09/074511 [Registered on: 27/09/2024] Trial Registered Prospectively |
| Last Modified On: |
05/03/2026 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
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Type of Study
|
Drug |
| Study Design |
Randomized, Parallel Group, Placebo Controlled Trial |
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Public Title of Study
|
A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants with Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body |
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Scientific Title of Study
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A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NONTRANSFUSION DEPENDENT β-THALASSEMIA. |
| Trial Acronym |
NIL |
Secondary IDs if Any
Modification(s)
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| Secondary ID |
Identifier |
| 2023-508604-37-00 |
EudraCT |
| NCT06421636 |
ClinicalTrials.gov |
| R7999-BTHAL-2350, Amendment 4 dated 25 Jun 2025 |
Protocol Number |
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Details of Principal Investigator or overall Trial Coordinator (multi-center study)
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| Name |
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| Designation |
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| Affiliation |
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| Address |
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| Phone |
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| Fax |
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| Email |
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Details of Contact Person
Scientific Query
|
| Name |
Dr Annappa Kamath |
| Designation |
Executive Director Project Leadership- Biotech Client Delivery Management |
| Affiliation |
Parexel International Clinical Research Private Limited |
| Address |
CoWrks, RMZ Eco World, Ground Floor, Bay Area - Adjacent to Building 6A, Outer Ring Road, Devarabeesanahalli Village
Bangalore
KARNATAKA
560103
India |
| Phone |
918067723000 |
| Fax |
918067723001 |
| Email |
Annappa.Kamath@parexel.com |
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Details of Contact Person
Public Query
|
| Name |
Dr Annappa Kamath |
| Designation |
Executive Director Project Leadership- Biotech Client Delivery Management |
| Affiliation |
Parexel International Clinical Research Private Limited |
| Address |
CoWrks, RMZ Eco World, Ground Floor, Bay Area – Adjacent to Building 6A, Outer Ring Road, Devarabeesanahalli Village
Bangalore
KARNATAKA
560103
India |
| Phone |
918067723000 |
| Fax |
918067723001 |
| Email |
Annappa.Kamath@parexel.com |
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Source of Monetary or Material Support
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| Regeneron Pharmaceuticals, Inc.
777 Old Saw Mill River Road
Tarrytown, NY 10591
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Primary Sponsor
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| Name |
Regeneron Pharmaceuticals, Inc. |
| Address |
777 Old Saw Mill River Road, Tarrytown, NY 10591 |
| Type of Sponsor |
Pharmaceutical industry-Global |
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Details of Secondary Sponsor
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| Name |
Address |
| PAREXEL International Clinical Research Private Limited |
CoWrks, RMZ Eco World, Ground Floor, Bay Area – Adjacent to Building 6A, Outer Ring Road, Devarabeesanahalli Village, BENGALURU – 560103, Karnataka, INDIA |
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Countries of Recruitment
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Georgia
Greece
India
Italy
Malaysia
Thailand
Turkey
United Kingdom
United States of America |
Sites of Study
Modification(s)
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| No of Sites = 7 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Tulika Seth |
All India Institute of Medical Sciences |
Room No.10, Porta Cabin, 5th floor Teaching block, New Delhi 110029, India
New Delhi
DELHI |
11-26588500
11-26588663
drtulikaseth@gmail.com |
| Dr Neeraj Sidharthan |
Amrita Institute of Medical Sciences |
Department of Clinical Hematology and Stem cell transplant Unit AIMS Ponekkara Po Kochi 682041, Kerala, India
Ernakulam
KERALA |
0484-2851234
0484-2852020
neerajsidharthan@aims.amrita.edu |
| Dr Sandip Abhaykumar Shah |
Hemato Oncology Clinic Ahmedabad PVT LTD |
Vedanta Institute of medical science Ground floor and First Floor Near Samved Hospital Navrangpura Ahmedabad 380009 Gujarat India
Ahmadabad
GUJARAT |
7926402220
7940042225
sandip60@yahoo.com |
| Dr Amita Mahajan |
Indraprastha Apollo Hospitals |
Sarita Vihar, Delhi Mathura Road, New Delhi 110076, India
New Delhi
DELHI |
011-26925801
mahajanamita1@gmail.com |
| Dr Kapil Garg |
J K Lon Hospital attached to SMS Medical college |
Jawahar Lal Nehru Marg, Near Trimurti Circle, Gangawal Park, Adarsh Nagar, Jaipur 302004, Rajasthan, India
Jaipur
RAJASTHAN |
1412619827
drkapilgargjkl@gmail.com |
| Dr Shrinath Kshirsagar |
K. J. Somaiya Hospital and Research Centre |
Somaiya Ayurvihar Complex, Eastern Express Highway, Sion East, Mumbai 400022, Maharashtra, India
Mumbai
MAHARASHTRA |
2235306587
shrinath@somaiya.edu |
| Dr Amit Ravindrabhai Khurana |
Nirmal Hospital Pvt Ltd |
Ring Road, Surat 395002, Gujarat, India
Surat
GUJARAT |
261-4089999
dramitrkhurana@gmail.com |
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Details of Ethics Committee
Modification(s)
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| No of Ethics Committees= 8 |
| Name of Committee |
Approval Status |
| Ethics Committee of Care Institute of Medical Sciences (CIMS), Opp. Panchamrut Bunglows, Nr. Shukan Mall, Off Science City Road, Sola, Ahmadabad-380060, Gujarat, India. |
Approved |
| Ethics Committee, SMS Medical College & Attached Hospitals Office of Ethics Committee, 2nd Floor, New Administrative block, JLN Marg Jaipur – 302004, Rajasthan, India |
Approved |
| Institute Ethics Committee, Room no.102 ,1st floor, Old OT Block. AIIMS, New Delhi-110029, India |
Approved |
| Institution Ethics committee Amrita Institute of Medical Sciences and Research Centre, AIMS Ponekkara P O, Cochin -682041, Kerala, India |
Approved |
| Institutional Ethics Committee, Clinical Studies Indraprastha Apollo Hospitals, Sarita Vihar, Delhi – Mathura Road, New Delhi – 110076, India |
Approved |
| Institutional Ethics Committee-Clinical Trials K. J. Somaiya Medical College, Hospital & Research Centre Somaiya Ayurvihar Complex, Eastern Express Highway, Sion(East), Mumbai - 400022 , Maharashtra, India |
Approved |
| Institutional Review Board, Christian Medical College, Bagayam, Vellore- 632012, Tamil Nadu, India |
Approved |
| Nirmal Hospital Ethics Committee, Nirmal Hospital Pvt Ltd, Ring Road, Surat – 395002, Gujarat, India |
Approved |
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Regulatory Clearance Status from DCGI
Modification(s)
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Health Condition / Problems Studied
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| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D561||Beta thalassemia, |
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Intervention / Comparator Agent
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| Type |
Name |
Details |
| Comparator Agent |
Placebo |
Specified doses on specified days, per the protocol, Administered subcutaneous (SC) |
| Intervention |
REGN7999 |
Specified doses on specified days, per the protocol, Administered subcutaneous (SC) |
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Inclusion Criteria
Modification(s)
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| Age From |
18.00 Year(s) |
| Age To |
65.00 Year(s) |
| Gender |
Both |
| Details |
1. Clinical diagnosis of Non-transfusion dependent beta-thalassemia (NTDT) as described in the protocol
2. Resultant Iron overload (IOL), defined as Liver iron concentration (LIC) greater than or equal to 5 mg Fe/g DW as measured by R2 magnetic resonance imaging (MRI) at screening
3. Serum ferritin greater than or equal to 300 ng/mL as described in the protocol
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| ExclusionCriteria |
| Details |
1. Hemoglobin less than or equal to 8 g/dL at screening
2. Any RBC transfusion within 12 weeks of visit 3
3. For Part A only: Any ICT use in approximately 12 weeks prior to screening as described in the protocol
4. For Part B only: If on ICT, any change in Iron chelation therapy (ICT) dose in approximately 12 weeks prior to screening as described in the protocol
5. Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
6. Absolute contraindication to MRI
7. Diagnosis of cirrhosis of the liver
8. Diagnosis of Chronic kidney disease (CKD) stage 4 or higher
9. Other protocol-defined Inclusion/ Exclusion Criteria apply
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Method of Generating Random Sequence
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Other |
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Method of Concealment
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Centralized |
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Blinding/Masking
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Participant and Investigator Blinded |
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Primary Outcome
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| Outcome |
TimePoints |
1. Change in Liver iron concentration (LIC) by R2 magnetic resonance imaging (MRI)
2. Incidence and Severity of Treatment-emergent adverse events (TEAEs) |
1. Baseline to week 24
2. Up to week 72 |
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Secondary Outcome
Modification(s)
|
| Outcome |
TimePoints |
| Achievement of greater than or equal to 20% reduction in LIC by R2 MRI |
Baseline to week 52 |
| Change in hemoglobin |
Baseline to week 24 |
| Change in LIC by R2 MRI |
Baseline to week 52 |
| Percent change in LIC by R2 MRI |
Baseline to week 24 and week 52 |
| Achievement of greater than or equal to 20% reduction in LIC by R2 MRI. |
Baseline to week 24 |
| Change in hemoglobin over time |
Up to week 56 |
| Achievement of greater than or equal to 1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of red blood cell (RBC) transfusions |
Baseline to week 56 |
| Number of RBC transfusions required |
Baseline to week 72 |
| Achievement of transfusion independence |
Baseline to week 72 |
| Change in RBC counts over time |
Baseline to week 56 |
| Concentrations of REGN7999 in serum over time |
Up to week 56 |
| Incidence of anti-drug antibody (ADA) to REGN7999 over time |
Up to week 56 |
| Magnitude of ADA to REGN7999 over time |
Up to week 56 |
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Target Sample Size
Modification(s)
|
Total Sample Size="95"
Sample Size from India="26"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
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Phase of Trial
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Phase 2 |
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Date of First Enrollment (India)
|
10/10/2024 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
25/09/2024 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
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Estimated Duration of Trial
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Years="3"
Months="5"
Days="0" |
Recruitment Status of Trial (Global)
Modification(s)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Open to Recruitment |
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Publication Details
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N/A |
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Individual Participant Data (IPD) Sharing Statement
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Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
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Brief Summary
Modification(s)
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This study is researching an experimental drug called REGN7999 (called study drug). The study is focused on patients with non-transfusion dependent beta-thalassemia. The aim of the study is to see how safe and effective the study drug is. The study is looking at several other research questions, including: -Whether the study drug lowers extra iron levels in the body -What side effects may happen from taking the study drug -How much study drug is in the blood at different times -Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) |