Severe Aplastic Anemia (SAA)
Conditions
Keywords
Immunosuppressive therapy (IST), Refractory, Relapsed
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
The trial was intended to be a Phase 1/2 trial, but no participants were enrolled in Phase 2
Interventions
DRUGREGN7257
Administered by intravenous (IV) infusion, in Part A and B.
Sponsors
Regeneron Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of adverse events (AEs) | 12 months post-treatment, approximately 52 weeks | Part A |
| Incidence of serious adverse events (SAEs) | 12 months post-treatment, approximately 52 weeks | Part A |
| Incidence and severity of treatment-emergent adverse events (TEAEs) | 12 months post-treatment, approximately 52 weeks | Part A |
| Overall response rate (ORR) | At 6 months, approximately 26 weeks | Part B |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| ORR | At 3 months, approximately 12 weeks | Parts A and B |
| Complete response (CR) | At 3 months, approximately 12 weeks | Parts A and B |
| Partial response (PR) | At 3 months, approximately 12 weeks | Parts A and B |
| Time to best response | Up to 12 months | Part A |
| Time to first response | Up to 12 months | Part A |
| Any clinical response | Until the end of study, approximately week 52 | Part A |
| Platelet transfusions per month over time | Up to 12 months | Part A |
| Red blood cell transfusions per month over time | Up to 12 months | Part A |
| Changes in lymphocyte cell counts | Up to 12 months | Part A |
| Changes in neutrophil cell counts | Up to 12 months | Part A |
| Changes in hemoglobin cell counts | Up to 12 months | Part A |
| Changes in reticulocyte cell counts | Up to 12 months | Part A |
| Changes in platelet cell counts | Up to 12 months | Part A |
| Changes in the whole blood immune cell subsets (T cells) | Up to 12 months | Part A |
| Changes in the whole blood immune cell subsets (B cells) | Up to 12 months | Part A |
| Changes in the whole blood immune cell subsets [Natural killer (NK) cells] | Up to 12 months | Part A |
| Changes in the whole blood immune cell subsets (NK cells) | Up to 18 months | Part B |
| Drug concentrations in serum over time | Up to 12 months | Part A |
| Incidence of treatment-emergent anti-drug antibody (ADA) over time | Up to 12 months | Part A |
| Incidence of treatment-emergent ADA over time | Up to 18 months | Part B |
Countries
France, South Korea, United Kingdom, United States
Contacts
STUDY_DIRECTORClinical Trial Management
Regeneron Pharmaceuticals
Outcome results
None listed