A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in People With Hemophilia A

Overview

Sponsor-declared trial summary

Key facts

Sponsor

F. Hoffmann-La Roche AG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Hemic and Lymphatic Diseases [C15]

Decision date (initial)

2026-06-01

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

F. Hoffmann La Roche

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Pharmacodynamic, Pharmacokinetic, Safety, Efficacy

To evaluate the efficacy of NXT007 prophylaxis compared with FVIII prophylaxis based on the superiority assessment of the primary endpoint (treated bleeds)

Secondary objectives 6

1. To evaluate the efficacy of NXT007 prophylaxis compared with FVIII prophylaxis based on the secondary endpoints
2. To evaluate the quality of life of participants treated with NXT007 prophylaxis compared with FVIII prophylaxis based on health-related quality of life questionnaire
3. To evaluate participants’ satisfaction with treatment administration as assessed through the use of the Treatment Administration Satisfaction Questionnaire (TASQ)
4. To evaluate the safety of NXT007 prophylaxis compared with FVIII prophylaxis
5. To characterize the pharmacokinetics of NXT007
6. To evaluate the immunogenicity of NXT007

Conditions and MedDRA coding

Hemophilia A Without Inhibitors

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration, Pharmaceuticals And Medical Devices Agency

EMA paediatric investigation plan (PIP)

EMEA-003550-PIP01-23

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Diagnosis of severe (FVIII:C <1 IU/dL) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital HA without inhibitors against FVIII
2. No documented inhibitor (i.e., < 0.6 BU/mL), FVIII half-life ≥6 hours, or FVIII recovery >66% in the last 3 years prior to screening
3. Documented historical negative test for FVIII inhibitor (i.e., < 0.6 BU/mL) within 12 months prior to enrollment
4. Documentation of the details of prophylactic and episodic FVIII treatment and of the number and type of bleeding episodes for at least the last 6 months prior to screening
5. Agreement to adhere to the contraception requirements (for potential participants with childbearing potential)

Exclusion criteria 6

1. Sensitivity to any of the study investigations, or components thereof, or drug or other allergy that, in the opinion of the investigator, contraindicates participation in the study
2. Use of systemic immunomodulators (e.g., interferon or rituximab) at the time of enrollment or planned use during the study, except for anti-retroviral therapy to treat HIV
3. Planned surgery (excluding minor procedures such as non-molar tooth extraction, incision and drainage) during the study
4. History or presence of an abnormal ECG that is deemed clinically significant, (e.g., complete left bundle branch block, second- or third -degree atrioventricular heart block) or ECG evidence or clinical history of prior myocardial infarction
5. Refusal to accept plasma-derived and/or blood product transfusion support in emergency scenario
6. History of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease (e.g., severe left ventricular systolic dysfunction, left ventricular hypertrophy), coronary heart disease (symptomatic or with ischemia demonstrated by diagnostic testing

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The annualized number of treated bleeds (ABR) over the main 6-month study treatment period

Secondary endpoints 19

1. Annualized number of bleeds (ABR) over the main 6-month study treatment period for All bleeds (treated and untreated)
2. Annualized number of bleeds (ABR) over the main 6-month study treatment period for treated spontaneous bleeds
3. Annualized number of bleeds (ABR) over the main 6-month study treatment period for treated joint bleeds
4. Quality of Life domain score in adult version at Month 7 visit
5. Annualized number of treated target joint bleeds (ABR) over the main 6-month study treatment period
6. Proportion of participants with zero treated bleeds over the main 6-month study treatment period
7. Number of injections and dose (per body weight) per bleed of coagulation factors administered to treat a bleed over the main 6-month study treatment period
8. Annualized FVIII injection rate and annualized FVIII consumption excluding regular FVIII prophylaxis and/or prior to procedures or surgeries in each arm over the main 6-month study treatment period
9. Quality of life domain score in adolescent version at Month 7 visit
10. Change from baseline in preoccupation, social activity impact and recreational activity impact domain scores
11. Physical impact domain score at specified timepoints
12. Incidence and severity of adverse events, with severity determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v.5.0)
13. Incidence and severity of thromboembolic events and thrombotic microangiopathy
14. Incidence and severity of injection-site reactions
15. Incidence of adverse events leading to drug discontinuation
16. Incidence of severe hypersensitivity, anaphylaxis, or anaphylactoid reactions
17. Plasma concentration of NXT007 at specified timepoints
18. Prevalence of anti-NXT007 antibodies (ADA) at baseline and incidence of ADAs during the study
19. Incidence of neutralizing ADAs

Investigational products

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

Test 4

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

F. Hoffmann-La Roche AG

Sponsor organisation

F. Hoffmann-La Roche AG

Address

Grenzacherstrasse 124

City

Basel

Postcode

4058

Country

Switzerland

Scientific contact point

Organisation

F. Hoffmann-La Roche AG

Contact name

Trial Information System - TISL

Public contact point

Organisation

F. Hoffmann-La Roche AG

Contact name

Trial Information System - TISL

Third parties 9

Locations

9 EU/EEA countries · 25 investigational sites

By country

Investigational sites

Results and documents

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

Documents 82 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