Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery

Phase 1

• Conditions: Congenital antithrombin deficiency; MedDRA version: 23.0Level: LLTClassification code 10083881Term: Antithrombin deficiencySystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2021-004307-40-HU
• Lead Sponsor: Octapharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-11-23
• Last Update Posted: 7 February 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 34

Inclusion Criteria

Patients who meet all of the following criteria are eligible for the study:

1. Adult male or female patients =18 and =80 years of age

2. Documented congenital antithrombin deficiency, defined by plasma level of antithrombin =60% [16]

3. Personal or family history of TEs or TEEs

4. For the Treatment Phase: either a) non-pregnant surgical patients scheduled for elective surgical procedure(s) known to be associated with a high risk for occurrence of TEs or TEEs, or b) pregnant patients of at least 27 weeks gestational age who are scheduled for caesarean section or delivery

5. For female patients of childbearing potential entering the PK Phase who are not known to be pregnant, and for female surgical patients of childbearing potential entering the Treatment Phase for any procedure other than caesarean section or delivery, a negative urine pregnancy test at screening and at baseline

6. Patient has provided informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
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 4

Exclusion Criteria

Patients who meet any of the following criteria at the time of screening are not eligible for the study:

1. Requires emergency surgery or emergency caesarean section

2. Has undergone surgery within the last 6 weeks

3. History or suspicion of another hereditary thrombophilic disorder other than antithrombin deficiency (e.g., activated protein C [APC] resistance/Factor V Leiden, Protein S or C deficiency, prothrombin gene mutation [G20210A], or acquired [lupus anticoagulant] thrombophilic disorder)

4. Malignancies, renal failure, or severe liver disease (aspartate aminotransferase [ASAT] >5 times the upper limit of normal)

5. Body mass index >40 kg/m2

6. Known hypersensitivity or allergic reaction to antithrombin or any of the excipients in Atenativ

7. History of anaphylactic reaction(s) to blood or blood components

8. Refusal to receive transfusion of blood-derived products

9. Administration of any antithrombin concentrate or antithrombin-containing blood product other than the study medication within 14 days of either of the two phases of the study

10. Prior diagnosis with heparin-induced thrombocytopenia

11. TE or TEE within the last 6 months

12. Female patients who are nursing

13. Have participated in another investigational study within the last 30 days

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this study is to assess the incidence of the composite of thrombotic events (TEs) and thromboembolic events (TEEs) in patients with congenital antithrombin deficiency under cover of Atenativ for surgical procedures or parturition;Secondary Objective: The secondary objectives of this study are to:<br>• Assess the single-dose PK of Atenativ in patients with congenital antithrombin deficiency<br>• Assess coagulation parameters in patients with congenital antithrombin deficiency undergoing surgical procedures or parturition<br>• Assess the safety and tolerability of Atenativ in patients with congenital antithrombin deficiency;Primary end point(s): The primary endpoint is the incidence of the composite of TEs and TEEs in patients with congenital antithrombin deficiency under cover of Atenativ for surgical procedures or parturition to 30 days after treatment initiation;Timepoint(s) of evaluation of this end point: 30 days after treatment initiation