A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of emicizumab in patients with mild or moderate hemophilia A without FVIII inhibitors

Phase 1

• Conditions: Mild or moderate hemophilia A; MedDRA version: 20.0Level: LLTClassification code 10053753Term: Hemophilia A without inhibitorsSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2019-002179-32-ES
• Lead Sponsor: Roche Farma S. A. U. que realiza el ensayo en España y actúa como representante F. Hoffmann-La Roche Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-09-04
• Last Update Posted: 19 October 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

-Diagnosis of mild (FVIII level between > 5% and < 40%) or moderate (FVIII level between >= 1% and <= 5%) congenital Hemophilia A without FVIII inhibitors
- Weight >= 3 kg
- Need for prophylaxis based on investigator assessment
- A negative test for inhibitor (i.e., < 0.6 BU/mL) within 8 weeks prior to enrollment
-No documented inhibitor (i.e., < 0.6 BU/mL), FVIII half-life < 6 hours, or FVIII recovery < 66% in the last 5 years
- Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks prior to enrollment
-Adequate hematologic hepatic and renal function
- For women of childbearing potential: agreement to remain abstinent or use contraception during the treatment period and for at least 24 weeks after the final dose of study drug
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

-Inherited or acquired bleeding disorder other than mild (FVIII level between > 5% and < 40%) or moderate (FVIII level between >= 1% and <= 5%) congenital hemophilia A
- History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator’s judgment
- Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease
- Other conditions that may currently increase the risk of bleeding or thrombosis
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
- Planned surgery during the emicizumab loading dose phase. Surgeries in patients on emicizumab from Week 5 onwards are allowed
- Known HIV infection with CD4 counts < 200 cells/micro L
- Concomitant disease, condition, significant abnormality on screening evaluation or laboratory tests, or treatment that could interfere with the conduct of the study, or that would in the opinion of the investigator, pose an additional unacceptable risk in administering study drug to the patient
- Receipt of any of the following:
o An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration with the exception of prior emicizumab prophylaxis
o A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter
o Any other investigational drug currently being administered or planned to be administered
- Inability to comply with the study protocol in the opinion of the investigator
- Pregnant or breastfeeding, or intending to become pregnant during the study
- Women of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of study drug

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified