- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04409080
REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
A Phase 1/2 Study of REGN7257 (Anti-Interleukin 2 Receptor Subunit Gamma [IL2RG] Monoclonal Antibody) in Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
This study is researching an experimental drug called REGN7257 (called "study drug"). The study is focused on patients who have severe aplastic anemia (SAA), a disease of the bone marrow resulting in an impairment of the production of blood cells.
The main purpose of this two-part study (Part A and Part B) is to test how safe and tolerable REGN7257 is in patients with SAA in which other Immunosuppressive therapies (ISTs) have not worked well.
The study is looking at several other research questions to better understand the following properties of REGN7257:
- Side effects that may be experienced by participants taking REGN7257
- How REGN7257 works in the body
- How much REGN7257 is present in blood after dosing
- If REGN7257 works to raise levels of certain blood counts after treatment
- How quickly REGN7257 works to raise levels of certain blood counts
- In patients for whom REGN7257 works to raise levels of certain blood counts after treatment, how many continue to show such a response throughout the study
- If REGN7257 works to lower the number of platelet and red blood cell transfusions needed
- How REGN7257 changes immune cell counts and composition
- How the body reacts to REGN7257 and if it produces proteins that bind to REGN7257 (this would be called the formation of anti-drug antibodies [ADA])
Study Overview
Study Type
Enrollment (Estimated)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Contact
- Name: Clinical Trials Administrator
- Phone Number: 844-734-6643
- Email: clinicaltrials@regeneron.com
Study Locations
-
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Ile-de-France
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Paris, Ile-de-France, France, 75010
- Recruiting
- Hopital Saint-Louis - APHP
-
Principal Investigator:
- Regis Peffault de Latour
-
-
-
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Gyeonggi
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Incheon, Gyeonggi, Korea, Republic of, 21565
- Recruiting
- Gachon University Gil Hospital
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Principal Investigator:
- Hawk Kim
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Seoul Capital Area
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Seoul, Seoul Capital Area, Korea, Republic of, 03080
- Recruiting
- Seoul National University Hospital
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Principal Investigator:
- Sung Soo Yoon
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Seoul, Seoul Capital Area, Korea, Republic of, 06351
- Recruiting
- Samsung Medical Center
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Principal Investigator:
- Jun Ho Jang
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Seoul, Seoul Capital Area, Korea, Republic of, 06591
- Recruiting
- The Catholic University of Korea, Seoul St. Marys Hospital
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Principal Investigator:
- Jong Wook Lee
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Seoul, Seoul Capital Area, Korea, Republic of, 07985
- Completed
- Ewha Womans University Medical Centre
-
-
-
-
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London, United Kingdom, SE5 9RS
- Recruiting
- King's College Hospital, London
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Principal Investigator:
- Austin Kulasekararaj
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West Yorkshire
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Leeds, West Yorkshire, United Kingdom, LS97TF
- Recruiting
- St James's University Hospital
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Principal Investigator:
- Morag Griffin
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-
-
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Ohio
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Cleveland, Ohio, United States, 44195
- Active, not recruiting
- Cleveland Clinic Foundation
-
-
Texas
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Houston, Texas, United States, 77030
- Recruiting
- The University of Texas MD Anderson Cancer Center
-
Principal Investigator:
- Tapan Kadia
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Key Inclusion Criteria:
- Part A: SAA that is IST-refractory or IST-relapsed, as defined in the protocol
- Part B: SAA that is IST-relapsed, as defined in the protocol
- Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient
- Adequate hepatic and renal function as defined in the protocol
Key Exclusion Criteria:
- Diagnosis of Fanconi anemia or other congenital bone marrow failure syndrome as defined in the protocol
- Evidence of myelodysplastic syndrome as defined in the protocol
- Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of clinically significant hemolysis (eg, treatment indicated) or history of PNH-associated thrombosis
- Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior to dosing
- Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to dosing for patients enrolled in Part A
- Treatment with eltrombopag or investigational thrombopoietin receptor agonist, Granulocyte Colony-Stimulating Factor (G-CSF), or an androgen (eg, danazol), within 2 weeks prior to dosing
- HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit as defined in the protocol
- Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI
- Active infection as defined in the protocol
Note: Other protocol-defined inclusion/ exclusion criteria apply
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Part A
Part A: Single ascending dose (SAD) escalation cohorts
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Administered by intravenous (IV) infusion, in Part A and B.
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Experimental: Part B
Part B: Multiple REGN7257 dosages.
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Administered by intravenous (IV) infusion, in Part A and B.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of adverse events (AEs)
Time Frame: 12 months post-treatment, approximately 52 weeks
|
Part A
|
12 months post-treatment, approximately 52 weeks
|
Incidence of serious adverse events (SAEs)
Time Frame: 12 months post-treatment, approximately 52 weeks
|
Part A
|
12 months post-treatment, approximately 52 weeks
|
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time Frame: 12 months post-treatment, approximately 52 weeks
|
Part A
|
12 months post-treatment, approximately 52 weeks
|
Incidence of serious adverse events (SAEs)
Time Frame: Through the end of study visit, approximately 78 weeks
|
Part B
|
Through the end of study visit, approximately 78 weeks
|
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time Frame: Through the end of study visit, approximately 78 weeks
|
Part B
|
Through the end of study visit, approximately 78 weeks
|
Overall response rate (ORR)
Time Frame: At 6 months, approximately 26 weeks
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Part B
|
At 6 months, approximately 26 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
ORR
Time Frame: At 3 months, approximately 12 weeks
|
Parts A and B
|
At 3 months, approximately 12 weeks
|
Complete response (CR)
Time Frame: At 3 months, approximately 12 weeks
|
Parts A and B
|
At 3 months, approximately 12 weeks
|
Partial response (PR)
Time Frame: At 3 months, approximately 12 weeks
|
Parts A and B
|
At 3 months, approximately 12 weeks
|
Time to best response
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Time to best response
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Time to first response
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Time to first response
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Any clinical response
Time Frame: Until the end of study, approximately week 52
|
Part A
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Until the end of study, approximately week 52
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Any clinical response
Time Frame: Until the end of study, approximately week 78
|
Part B
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Until the end of study, approximately week 78
|
Platelet transfusions per month over time
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Platelet transfusions per month over time
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Red blood cell transfusions per month over time
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Red blood cell transfusions per month over time
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Changes in lymphocyte cell counts
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Changes in lymphocyte cell counts
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Changes in neutrophil cell counts
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Changes in neutrophil cell counts
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Changes in hemoglobin cell counts
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Changes in hemoglobin cell counts
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Changes in reticulocyte cell counts
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Changes in reticulocyte cell counts
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Changes in platelet cell counts
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Changes in platelet cell counts
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Changes in the whole blood immune cell subsets (T cells)
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Changes in the whole blood immune cell subsets (T cells)
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Changes in the whole blood immune cell subsets (B cells)
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Changes in the whole blood immune cell subsets (B cells)
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Changes in the whole blood immune cell subsets [Natural killer (NK) cells]
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Changes in the whole blood immune cell subsets (NK cells)
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Drug concentrations in serum over time
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Drug concentrations in serum over time
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Incidence of treatment-emergent anti-drug antibody (ADA) over time
Time Frame: Up to 12 months
|
Part A
|
Up to 12 months
|
Incidence of treatment-emergent ADA over time
Time Frame: Up to 18 months
|
Part B
|
Up to 18 months
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: Clinical Trial Management, Regeneron Pharmaceuticals
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- R7257-RAA-1947
- 2020-002031-29 (EudraCT Number)
- 2023-508601-24-00 (Other Identifier: EU CT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Time Frame
IPD Sharing Access Criteria
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
- ICF
- ANALYTIC_CODE
- CSR
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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