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

Primary Purpose

Severe Aplastic Anemia (SAA)

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
REGN7257
Sponsored by
Regeneron Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Severe Aplastic Anemia (SAA) focused on measuring Immunosuppressive therapy (IST), Refractory, Relapsed

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • SAA that is IST-refractory or 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 as defined in the protocol
  • 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 including COVID-19

Note: Other protocol-defined inclusion/ exclusion criteria apply

Sites / Locations

  • Cleveland Clinic FoundationRecruiting
  • The University of Texas MD Anderson Cancer CenterRecruiting
  • Hopital Saint-Louis - APHPRecruiting
  • Gachon University Gil HospitalRecruiting
  • Seoul National University HospitalRecruiting
  • Samsung Medical CenterRecruiting
  • The Catholic University of Korea, Seoul St. Marys HospitalRecruiting
  • Ewha Womans University Medical Centre
  • St James's University HospitalRecruiting
  • King's College Hospital, LondonRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Part A and Part B

Arm Description

Part A: Single ascending dose Part B: Preferred dose

Outcomes

Primary Outcome Measures

Incidence of adverse events (AEs)
Part A
Incidence of serious adverse events (SAEs)
Parts A and B
Incidence and severity of treatment-emergent adverse events (TEAEs) up
Parts A and B
Overall response rate (ORR)
Part B

Secondary Outcome Measures

ORR
Parts A and B
Complete response (CR)
Parts A and B
Partial response (PR)
Parts A and B
Time to best response
Parts A and B
Time to first response
Parts A and B
Proportion of patients who maintain any clinical response
Parts A and B
Platelet transfusions per month over time
Parts A and B
Red blood cell transfusions per month over time
Parts A and B
Changes in lymphocyte cell counts
Parts A and B
Changes in neutrophil cell counts
Parts A and B
Changes in hemoglobin cell counts
Parts A and B
Changes in reticulocyte cell counts
Parts A and B
Changes in platelet cell counts
Parts A and B
Changes in the whole blood immune cell subsets (T cells)
Parts A and B
Changes in the whole blood immune cell subsets (B cells)
Parts A and B
Changes in the whole blood immune cell subsets (NK cells)
Parts A and B
Drug concentrations in serum over time
Parts A and B
Incidence of treatment-emergent anti-drug antibody (ADA) over time
Parts A and B

Full Information

First Posted
May 26, 2020
Last Updated
July 20, 2023
Sponsor
Regeneron Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT04409080
Brief Title
REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 13, 2021 (Actual)
Primary Completion Date
January 2, 2026 (Anticipated)
Study Completion Date
January 2, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Regeneron Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with immunosuppressive therapy (IST)-refractory or IST-relapsed severe aplastic anemia (SAA). An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in patients with IST-refractory or IST-relapsed SAA. The secondary objectives of this study are to assess the following for REGN7257: Clinical response over time Maintenance of response Impact on transfusion requirements Effect on blood counts and cell populations Pharmacokinetics (PK) Immunogenicity

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Aplastic Anemia (SAA)
Keywords
Immunosuppressive therapy (IST), Refractory, Relapsed

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
57 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Part A and Part B
Arm Type
Experimental
Arm Description
Part A: Single ascending dose Part B: Preferred dose
Intervention Type
Drug
Intervention Name(s)
REGN7257
Intervention Description
Single dose will be administered by intravenous IV infusion
Primary Outcome Measure Information:
Title
Incidence of adverse events (AEs)
Description
Part A
Time Frame
12 months post-treatment, approximately 52 weeks
Title
Incidence of serious adverse events (SAEs)
Description
Parts A and B
Time Frame
12 months post-treatment, approximately 52 weeks
Title
Incidence and severity of treatment-emergent adverse events (TEAEs) up
Description
Parts A and B
Time Frame
12 months post-treatment, approximately 52 weeks
Title
Overall response rate (ORR)
Description
Part B
Time Frame
6 months post-treatment, approximately 26 weeks
Secondary Outcome Measure Information:
Title
ORR
Description
Parts A and B
Time Frame
3 months post-treatment, approximately 12 weeks
Title
Complete response (CR)
Description
Parts A and B
Time Frame
3 months post-treatment, approximately 12 weeks
Title
Partial response (PR)
Description
Parts A and B
Time Frame
3 months post-treatment, approximately 12 weeks
Title
Time to best response
Description
Parts A and B
Time Frame
Up to 12 months
Title
Time to first response
Description
Parts A and B
Time Frame
Up to 12 months
Title
Proportion of patients who maintain any clinical response
Description
Parts A and B
Time Frame
Up to 12 months
Title
Platelet transfusions per month over time
Description
Parts A and B
Time Frame
Up to 12 months
Title
Red blood cell transfusions per month over time
Description
Parts A and B
Time Frame
Up to 12 months
Title
Changes in lymphocyte cell counts
Description
Parts A and B
Time Frame
Up to 12 months
Title
Changes in neutrophil cell counts
Description
Parts A and B
Time Frame
Up to 12 months
Title
Changes in hemoglobin cell counts
Description
Parts A and B
Time Frame
Up to 12 months
Title
Changes in reticulocyte cell counts
Description
Parts A and B
Time Frame
Up to 12 months
Title
Changes in platelet cell counts
Description
Parts A and B
Time Frame
Up to 12 months
Title
Changes in the whole blood immune cell subsets (T cells)
Description
Parts A and B
Time Frame
Up to 12 months
Title
Changes in the whole blood immune cell subsets (B cells)
Description
Parts A and B
Time Frame
Up to 12 months
Title
Changes in the whole blood immune cell subsets (NK cells)
Description
Parts A and B
Time Frame
Up to 12 months
Title
Drug concentrations in serum over time
Description
Parts A and B
Time Frame
Up to 12 months
Title
Incidence of treatment-emergent anti-drug antibody (ADA) over time
Description
Parts A and B
Time Frame
Up to 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: SAA that is IST-refractory or 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 as defined in the protocol 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 including COVID-19 Note: Other protocol-defined inclusion/ exclusion criteria apply
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Clinical Trials Administrator
Phone
844-734-6643
Email
clinicaltrials@regeneron.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trial Management
Organizational Affiliation
Regeneron Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sophia Balderman
Facility Name
The University of Texas MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tapan Kadia
Facility Name
Hopital Saint-Louis - APHP
City
Paris
State/Province
Ile-de-France
ZIP/Postal Code
75010
Country
France
Individual Site Status
Recruiting
Facility Name
Gachon University Gil Hospital
City
Incheon
State/Province
Gyeonggi
ZIP/Postal Code
21565
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hawk Kim
Facility Name
Seoul National University Hospital
City
Seoul
State/Province
Seoul Capital Area
ZIP/Postal Code
03080
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yoon Sung Soo
Facility Name
Samsung Medical Center
City
Seoul
State/Province
Seoul Capital Area
ZIP/Postal Code
06351
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jun Ho Yang
Facility Name
The Catholic University of Korea, Seoul St. Marys Hospital
City
Seoul
State/Province
Seoul Capital Area
ZIP/Postal Code
06591
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jong Wook Lee
Facility Name
Ewha Womans University Medical Centre
City
Seoul
State/Province
Seoul Capital Area
ZIP/Postal Code
07985
Country
Korea, Republic of
Individual Site Status
Completed
Facility Name
St James's University Hospital
City
Leeds
State/Province
West Yorkshire
ZIP/Postal Code
LS97TF
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Morag Griffin
Facility Name
King's College Hospital, London
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Austin Kulasekararaj

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
IPD Sharing Time Frame
Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.
IPD Sharing Access Criteria
Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency [EMA], Pharmaceuticals and Medical Devices Agency [PMDA], etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).
IPD Sharing URL
https://vivli.org/

Learn more about this trial

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

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