Non-Transfusion Dependent Beta-Thalassemia (NTDT)Phase 2FERVENT-1NCT06421636
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
A Phase 2 study testing REGN7999 in people with Non-Transfusion Dependent Beta-Thalassemia (NTDT), run by Regeneron Pharmaceuticals at 26 sites in 9 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Non-Transfusion Dependent Beta-Thalassemia (NTDT)
- What is being tested?
- REGN7999
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 19 participants
- Where
- 26 sitesin 9 countries
- Study dates
- 30 Sep 2024to 3 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinical diagnosis of NTDT as described in the protocol
- IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2* MRI at screening
- Serum ferritin ≥ 300 ng/mL as described in the protocol
Who cannot join
- Hemoglobin ≤ 8 g/dL at screening
- Any RBC transfusion within 12 weeks of visit 3
- For Part A only: Any Iron Chelation Therapy (ICT) use in approximately 12 weeks prior to screening as described in the protocol
- For Part B only: If on ICT, any change in ICT dose in approximately 12 weeks prior to screening as described in the protocol
- Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
- Absolute contraindication to MRI
Show all 9Show fewer
- Diagnosis of cirrhosis of the liver
- Diagnosis of Chronic kidney disease (CKD) stage 4 or higher
- Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Trial locations and contacts
26 sites in 9 countries.
26 sites in 9 countries
India7 sites
Hemato-Oncology Clinic Ahmedabad PVT. LTD.
Ahmedabad, Gujarat, 380009
JK Lon Hospital
Jaipur, Rajasthan, 302004
Amrita Institute of Medical Sciences and Research Centre (AIMS)
Kochi, Kerala, 682041
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai, Maharashtra, 400022
All India Institute of Medical Sciences, New Delhi
New Delhi, National Capital Territory of Delhi, 110029
Indraprastha Apollo Hospitals
New Delhi, National Capital Territory of Delhi, 110048
Nirmal Hospital Pvt. Ltd - Surat
Surat, Gujarat, 395002
Thailand5 sites
Chulalongkorn University
Bangkok, 10330
Phramongkutklao Hospital
Bangkok, 10400
Chiang Mai University
Chiang Mai, 50200
Songklanagarind Hospital, Prince of Songkla University
Hat Yai, Changwat Songkhla, 90110
Srinagarind Hospital
Khon Kaen, 40000
Malaysia4 sites
Hospital Ampang
Ampang, Selangor, 68000
Hospital Queen Elizabeth
Kota Kinabalu, Sabah, 88586
Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM)
Kuala Lumpur, 56000
Sarawak General Hospital
Kuching, Sarawak, 93586
Greece3 sites
Children's Hospital Agia Sophia
Athens, Attica, 11521
Laiko General Hospital of Athens
Athens, Attica, 11527
University Hospital of Ioannina
Ioannina, 45110
Georgia2 sites
K Eristavi National Center of Experimental and Clinical Surgery
Tbilisi, 0159
Medinvest Institute of Hematology and Transfusiology Limited
Tbilisi, 0160
United Kingdom2 sites
University College London Hospitals NHS Trust
London, NW1 2PG
Imperial College Healthcare NHS Trust
London, W12 0HS
Italy1 site
Day Hospital Thalassemia and Haemoglobinopathies (DHTE); A.O.U S.Anna
Ferrara, 44124
About this study
Official title: 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 NON-TRANSFUSION DEPENDENT β-THALASSEMIA
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)
- Being tested
- REGN7999, Placebo
- Keywords
- Beta-Thalassemia, Resultant Iron Overload (IOL)
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
REGN7999Drug
Administered subcutaneous (SC)
PlaceboDrug
Administered SC
| Study group | Role | What they receive |
|---|---|---|
| Part A High Dose | Experimental | Randomized 2:2:1 Receives: Drug: REGN7999 |
| Part A Low Dose | Experimental | Randomized 2:2:1 Receives: Drug: REGN7999 |
| Part A Placebo | Placebo comparator | Randomized 2:2:1 Receives: Drug: Placebo |
| Part B High Dose | Experimental | Randomized 2:2:1 Receives: Drug: REGN7999 |
| Part B Low Dose | Experimental | Randomized 2:2:1 Receives: Drug: REGN7999 |
| Part B Placebo | Placebo comparator | Randomized 2:2:1 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI)
Measured: At week 24
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Measured: Up to week 72
Severity of TEAEs
Measured: Up to week 72
Secondary outcomes (13)
Achievement of ≥20% reduction from baseline in LIC by R2* MRI
Measured: Through week 52
Change from baseline in hemoglobin
Measured: To week 24
Change from baseline in LIC by R2* MRI
Measured: At week 52
Percent change from baseline in LIC by R2* MRI
Measured: To week 24 and week 52
Achievement of ≥20% reduction from baseline in LIC by R2* MRI
Measured: Through week 24
Change in hemoglobin over time
Measured: Up to week 56
Achievement of ≥1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of Red Blood Cell (RBC) transfusions
Measured: Baseline to week 56
Number of RBC transfusions required
Measured: Baseline to week 72
Achievement of transfusion independence
Measured: Baseline to week 72
Change in RBC counts over time
Measured: Baseline to week 56
Concentrations of REGN7999 in serum over time
Measured: Up to week 56
Incidence of Anti-Drug Antibody (ADA) to REGN7999 over time
Measured: Up to week 56
Magnitude of ADA to REGN7999 over time
Measured: Up to week 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2024
- Study start30 Sep 2024
- Primary completion3 Dec 2026 (estimated)
- Study completion3 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non-Transfusion Dependent Beta-Thalassemia (NTDT)Studies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06421636
- Sponsor's protocol ID
- R7999-BThal-2350
- Other IDs
- 2023-508604-37-00, 2023
- Acronym
- FERVENT-1
- Status last verified
- Jun 2026
- First submitted
- 14 May 2024
- Last updated
- 1 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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 NON-TRANSFUSION DEPENDENT β-THALASSEMIA
- ClinicalTrials.gov ID
- NCT06421636
- Acronym / sponsor ID
- FERVENT-1 / R7999-BThal-2350
- Keywords
- Beta-Thalassemia, Resultant Iron Overload (IOL)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2024 (actual)
- Primary completion
- 3 Dec 2026 (estimated)
- Study completion
- 3 Dec 2026 (estimated)
- First posted
- 20 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Part A High Dose | Experimental | Drug: REGN7999Randomized 2:2:1 |
| Part A Low Dose | Experimental | Drug: REGN7999Randomized 2:2:1 |
| Part A Placebo | Placebo comparator | Drug: PlaceboRandomized 2:2:1 |
| Part B High Dose | Experimental | Drug: REGN7999Randomized 2:2:1 |
| Part B Low Dose | Experimental | Drug: REGN7999Randomized 2:2:1 |
| Part B Placebo | Placebo comparator | Drug: PlaceboRandomized 2:2:1 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Clinical diagnosis of NTDT as described in the protocol
- IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2* MRI at screening
- Serum ferritin ≥ 300 ng/mL as described in the protocol
Key exclusion criteria (9 listed)
- Hemoglobin ≤ 8 g/dL at screening
- Any RBC transfusion within 12 weeks of visit 3
- For Part A only: Any Iron Chelation Therapy (ICT) use in approximately 12 weeks prior to screening as described in the protocol
- For Part B only: If on ICT, any change in ICT dose in approximately 12 weeks prior to screening as described in the protocol
- Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| REGN7999Administered subcutaneous (SC) | Drug | Part A High Dose, Part A Low Dose, Part B High Dose, Part B Low Dose |
| PlaceboAdministered SC | Drug | Part A Placebo, Part B Placebo |
Primary outcome
Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI)
Time frame: At week 24
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to week 72
Severity of TEAEs
Time frame: Up to week 72
13 secondary outcomes
Achievement of ≥20% reduction from baseline in LIC by R2* MRI
Time frame: Through week 52
Change from baseline in hemoglobin
Time frame: To week 24
Change from baseline in LIC by R2* MRI
Time frame: At week 52
Percent change from baseline in LIC by R2* MRI
Time frame: To week 24 and week 52
Achievement of ≥20% reduction from baseline in LIC by R2* MRI
Time frame: Through week 24
Change in hemoglobin over time
Time frame: Up to week 56
Achievement of ≥1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of Red Blood Cell (RBC) transfusions
Time frame: Baseline to week 56
Number of RBC transfusions required
Time frame: Baseline to week 72
Achievement of transfusion independence
Time frame: Baseline to week 72
Change in RBC counts over time
Time frame: Baseline to week 56
Concentrations of REGN7999 in serum over time
Time frame: Up to week 56
Incidence of Anti-Drug Antibody (ADA) to REGN7999 over time
Time frame: Up to week 56
Magnitude of ADA to REGN7999 over time
Time frame: Up to week 56
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2026.
- Individual participant dataSharing planned: Yes. All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Management study director
Regeneron Pharmaceuticals
26 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing REGN7999 in people with Non-Transfusion Dependent Beta-Thalassemia (NTDT), run by Regeneron Pharmaceuticals at 26 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 1 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in India, Thailand, Malaysia, Greece, Georgia and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI) (time frame: At week 24). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2026; AzTrials last refreshed its copy on 1 Nov 2026.