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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Regeneron Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

13 Sep 2021 → 17 Jun 2025

Decision date (initial)

2024-06-14

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Regeneron Pharmaceuticals Inc.

External identifiers

EU CT number

2023-508601-24-00

EudraCT number

2020-002031-29

ClinicalTrials.gov

NCT04409080

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacogenomic, Others, Efficacy, Pharmacokinetic, Safety, Pharmacogenetic, Pharmacodynamic

The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with IST-refractory or IST-relapsed SAA. An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in patients with IST-relapsed SAA as proof of concept

Secondary objectives 6

1. Clinical response over time
2. Maintenance of response
3. Impact on transfusion requirements
4. Effect on blood counts and cell populations
5. Pharmacokinetics (PK)
6. Immunogenicity

Conditions and MedDRA coding

Severe aplastic anemia (SAA)

Regulatory references

Scientific advice from competent authorities

Medicines And Healthcare Products Regulatory Agency

Plan to share IPD

Yes

IPD plan description

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

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
5. Other protocol-defined inclusion criteria apply

Exclusion criteria 10

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
10. Other protocol-defined exclusion criteria apply

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Part A: Incidence of adverse events (AEs), incidence of serious adverse events (SAEs), and incidence and severity of treatment-emergent adverse events (TEAEs);
2. Part B: Incidence of serious adverse events (SAEs), incidence and severity of treatment-emergent adverse events (TEAEs);
3. Part B: Overall response rate (ORR);

Secondary endpoints 33

1. Overall response rate (ORR); Parts A & B
2. Complete response (CR); Parts A and B
3. Partial response (PR); Parts A and B
4. Time to best response; Part A
5. Time to best response; Part B
6. Time to first response; Part A
7. Time to first response; Part B
8. Any clinical response; Part A
9. Any clinical response; Part B
10. Platelet transfusions per month over time; Part A
11. Platelet transfusions per month over time; Part B
12. Red blood cell transfusions per month over time; Part A
13. Red blood cell transfusions per month over time; Part B
14. Changes in lymphocyte cell counts; Part A
15. Changes in lymphocyte cell counts; Part B
16. Changes in neutrophil cell counts; Part A
17. Changes in neutrophil cell counts; Part B
18. Changes in hemoglobin cell counts; Part A
19. Changes in hemoglobin cell counts; Part B
20. Changes in reticulocyte cell counts; Part A
21. Changes in reticulocyte cell counts; Part B
22. Changes in platelet cell counts; Part A
23. Changes in platelet cell counts; Part B
24. Changes in the whole blood immune cell subsets (T cells); Part A
25. Changes in the whole blood immune cell subsets (T cells); Part B
26. Changes in the whole blood immune cell subsets (B cells); Part A
27. Changes in the whole blood immune cell subsets (B cells); Part B
28. Changes in the whole blood immune cell subsets [Natural killer (NK) cells]; Part A
29. Changes in the whole blood immune cell subsets (NK cells); Part B
30. Drug concentrations in serum over time; Part A
31. Drug concentrations in serum over time; Part B
32. Incidence of treatment-emergent anti-drug antibody (ADA) over time; Part A
33. Incidence of treatment-emergent ADA over time; Part B

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Regeneron Pharmaceuticals Inc.

Sponsor organisation

Regeneron Pharmaceuticals Inc.

Address

777 Old Saw Mill River Road

City

Tarrytown

Postcode

10591-6717

Country

United States

Scientific contact point

Organisation

Regeneron Pharmaceuticals Inc.

Contact name

Medical Affairs

Public contact point

Organisation

Regeneron Pharmaceuticals Inc.

Contact name

Medical Affairs

Third parties 5

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 13 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications