REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

Phase 1TerminatedNCT04409080

Regeneron Pharmaceuticals17 enrolled

Overview

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 \[ADA\])

The trial was intended to be a Phase 1/2 trial, but no participants were enrolled in Phase 2

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Severe Aplastic Anemia (SAA)

Interventions

REGN7257DRUG

Administered by intravenous (IV) infusion, in Part A and B.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: 1. Part A: SAA that is IST-refractory or IST-relapsed, as defined in the protocol 2. Part B: SAA that is IST-relapsed, as defined in the protocol 3. Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient 4. Adequate hepatic and renal function as defined in the protocol Key Exclusion Criteria: 1. Diagnosis of Fanconi anemia or other congenital bone marrow failure syndrome as defined in the protocol 2. Evidence of myelodysplastic syndrome as defined in the protocol 3. Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of clinically significant hemolysis (eg, treatment indicated) or history of PNH-associated thrombosis 4. Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior to dosing 5. Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to dosing for patients enrolled in Part A 6. 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 7. HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit as defined in the protocol 8. Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI 9. Active infection as defined in the protocol Note: Other protocol-defined inclusion/ exclusion criteria apply

Locations (10)

Cleveland Clinic Foundation

Cleveland, Ohio, United States

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Hopital Saint-Louis - APHP

Paris, Île-de-France Region, France

Gachon University Gil Hospital

Incheon, Gyeonggi-do, South Korea

Seoul National University Hospital

Seoul, Seoul Capital Area, South Korea

Samsung Medical Center

Seoul, Seoul Capital Area, South Korea

The Catholic University of Korea, Seoul St. Marys Hospital

Seoul, Seoul Capital Area, South Korea

Ewha Womans University Medical Centre

Seoul, Seoul Capital Area, South Korea

St James's University Hospital

Leeds, West Yorkshire, United Kingdom

King's College Hospital, London

London, United Kingdom

Outcomes

Primary Outcomes

Incidence of adverse events (AEs)

Part A

Time frame: 12 months post-treatment, approximately 52 weeks

Incidence of serious adverse events (SAEs)

Part A

Time frame: 12 months post-treatment, approximately 52 weeks

Incidence and severity of treatment-emergent adverse events (TEAEs)

Part A

Time frame: 12 months post-treatment, approximately 52 weeks

Incidence of serious adverse events (SAEs)

Part B

Time frame: Through the end of study visit, approximately 78 weeks

Incidence and severity of treatment-emergent adverse events (TEAEs)

Part B

Time frame: Through the end of study visit, approximately 78 weeks

Overall response rate (ORR)

Part B

Time frame: At 6 months, approximately 26 weeks

Secondary Outcomes

ORR

Parts A and B

Time frame: At 3 months, approximately 12 weeks

Complete response (CR)

Parts A and B

Time frame: At 3 months, approximately 12 weeks

Partial response (PR)

Parts A and B

Time frame: At 3 months, approximately 12 weeks

Time to best response

Part A

Time frame: Up to 12 months

Time to best response

Part B

Time frame: Up to 18 months

Time to first response

Part A

Time frame: Up to 12 months

Time to first response

Part B

Time frame: Up to 18 months

Any clinical response

Part A

Time frame: Until the end of study, approximately week 52

Any clinical response

Part B

Time frame: Until the end of study, approximately week 78

Platelet transfusions per month over time

Part A

Time frame: Up to 12 months

Platelet transfusions per month over time

Part B

Time frame: Up to 18 months

Red blood cell transfusions per month over time

Part A

Time frame: Up to 12 months

Red blood cell transfusions per month over time

Part B

Time frame: Up to 18 months

Changes in lymphocyte cell counts

Part A

Time frame: Up to 12 months

Changes in lymphocyte cell counts

Part B

Time frame: Up to 18 months

Changes in neutrophil cell counts

Part A

Time frame: Up to 12 months

Changes in neutrophil cell counts

Part B

Time frame: Up to 18 months

Changes in hemoglobin cell counts

Part A

Time frame: Up to 12 months

Changes in hemoglobin cell counts

Part B

Time frame: Up to 18 months

Changes in reticulocyte cell counts

Part A

Time frame: Up to 12 months

Changes in reticulocyte cell counts

Part B

Time frame: Up to 18 months

Changes in platelet cell counts

Part A

Time frame: Up to 12 months

Changes in platelet cell counts

Part B

Time frame: Up to 18 months

Changes in the whole blood immune cell subsets (T cells)

Part A

Time frame: Up to 12 months

Changes in the whole blood immune cell subsets (T cells)

Part B

Time frame: Up to 18 months

Changes in the whole blood immune cell subsets (B cells)

Part A

Time frame: Up to 12 months

Changes in the whole blood immune cell subsets (B cells)

Part B

Time frame: Up to 18 months

Changes in the whole blood immune cell subsets [Natural killer (NK) cells]

Part A

Time frame: Up to 12 months

Changes in the whole blood immune cell subsets (NK cells)

Part B

Time frame: Up to 18 months

Drug concentrations in serum over time

Part A

Time frame: Up to 12 months

Drug concentrations in serum over time

Part B

Time frame: Up to 18 months

Incidence of treatment-emergent anti-drug antibody (ADA) over time

Part A

Time frame: Up to 12 months

Incidence of treatment-emergent ADA over time

Part B

Time frame: Up to 18 months

Data from ClinicalTrials.gov

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