REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
- Registration Number
- NCT04409080
- Lead Sponsor
- Regeneron Pharmaceuticals
- Brief Summary
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 \[ADA\])
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- SUSPENDED
- Sex
- All
- Target Recruitment
- 33
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A REGN7257 Part A: Single ascending dose (SAD) escalation cohorts Part B REGN7257 Part B: Multiple REGN7257 dosages.
- Primary Outcome Measures
Name Time Method Incidence of serious adverse events (SAEs) Through the end of study visit, approximately 78 weeks Part B
Incidence and severity of treatment-emergent adverse events (TEAEs) Through the end of study visit, approximately 78 weeks Part B
Overall response rate (ORR) At 6 months, approximately 26 weeks Part B
Incidence of adverse events (AEs) 12 months post-treatment, approximately 52 weeks Part A
- Secondary Outcome Measures
Name Time Method Time to best response Up to 18 months Part B
Any clinical response Until the end of study, approximately week 78 Part B
Changes in platelet cell counts Up to 18 months Part B
Incidence of treatment-emergent anti-drug antibody (ADA) over time Up to 12 months Part A
ORR At 3 months, approximately 12 weeks Parts A and B
Platelet transfusions per month over time Up to 18 months Part B
Changes in the whole blood immune cell subsets (T cells) Up to 18 months Part B
Changes in the whole blood immune cell subsets (B cells) Up to 18 months Part B
Changes in the whole blood immune cell subsets (NK cells) Up to 18 months Part B
Partial response (PR) At 3 months, approximately 12 weeks Parts A and B
Time to first response Up to 18 months Part B
Red blood cell transfusions per month over time Up to 18 months Part B
Changes in reticulocyte cell counts Up to 18 months Part B
Changes in the whole blood immune cell subsets [Natural killer (NK) cells] Up to 12 months Part A
Complete response (CR) At 3 months, approximately 12 weeks Parts A and B
Changes in neutrophil cell counts Up to 18 months Part B
Changes in hemoglobin cell counts Up to 18 months Part B
Changes in lymphocyte cell counts Up to 18 months Part B
Drug concentrations in serum over time Up to 18 months Part B
Incidence of treatment-emergent ADA over time Up to 18 months Part B
Trial Locations
- 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, Ile-de-France, France
Gachon University Gil Hospital
🇰🇷Incheon, Gyeonggi, Korea, Republic of
Seoul National University Hospital
🇰🇷Seoul, Seoul Capital Area, Korea, Republic of
Samsung Medical Center
🇰🇷Seoul, Seoul Capital Area, Korea, Republic of
The Catholic University of Korea, Seoul St. Marys Hospital
🇰🇷Seoul, Seoul Capital Area, Korea, Republic of
Ewha Womans University Medical Centre
🇰🇷Seoul, Seoul Capital Area, Korea, Republic of
St James's University Hospital
🇬🇧Leeds, West Yorkshire, United Kingdom
King's College Hospital, London
🇬🇧London, United Kingdom