Congenital Antithrombin DeficiencyPhase 3NCT04918173
Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery
A Phase 3 study testing Atenativ in people with Congenital Antithrombin Deficiency, run by Octapharma at 30 sites in 14 countries.
Trial summary
The goal 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 when they receive Atenativ for surgical procedures or parturition. Read more about this study
- Who is it for?
- Ages 12 to 80 years
- Conditions the study is for
- Congenital Antithrombin Deficiency
- What is being tested?
- Atenativ
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 30 sitesin 14 countries
- Study dates
- 1 Jul 2022to 1 Dec 2026
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Adult male or female patients ≥18 and ≤80 years of age. Solely in the US, 4 male or female patients between ≥12 and <17 years of age will be enrolled into the PK phase, and subsequently in the treatment phase, if applicable
- Documented congenital antithrombin deficiency, defined by plasma activity level of antithrombin ≤60% from medical history
- Personal or family history of TEs or TEEs (except for PK patients)
- 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
- 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
- Patient has provided informed consent
Who cannot join
- Requires emergency surgery or emergency caesarean section
- Has undergone surgery within the last 6 weeks
- 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)
- Malignancies, renal failure (patients on renal replacement therapy), or severe liver disease (aspartate aminotransferase [ASAT] >5 times the upper limit of normal)
- Body mass index >40 kg/m2 (for non-pregnant patients, only)
- Known hypersensitivity or allergic reaction to antithrombin or any of the excipients in Atenativ
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- History of anaphylactic reaction(s) to blood or blood components
- Refusal to receive transfusion of blood-derived products
- Administration of any antithrombin concentrate or antithrombin-containing blood product within 14 days of either of the two phases of the study
- Prior diagnosis of heparin-induced thrombocytopenia
- TE or TEE within the last 6 months
- Female patients who are nursing at the time of screening*
- Have participated in another investigational study within the last 30 days
- Persons dependent on the sponsor, the investigator or the centre of investigation
- Persons placed in an institution by administrative or judicial order
- criterion does not include female patients who plan to breastfeed after giving birth
Trial locations and contacts
30 sites in 14 countries. Contact the study team or a site near you to ask about taking part.
30 sites in 14 countries
United States5 sites, 5 recruiting
- Recruiting
Duke University School of Medicine, Division of Hematology
Durham, North Carolina, 27708
- Recruiting
University of Miami, Miller School of Medicine
Miami, Florida, 33124
- Recruiting
Weill Cornell Medicine, New York Presbyterian Hospital
New York, New York, 10065
- Recruiting
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61615
- Recruiting
Georgetown University Hospital
Washington D.C., District of Columbia, 20057
Italy4 sites, 4 recruiting
- Recruiting
Unita Strutturale Complessa di Immunoematologia e Medicina Trasfusionale -Dipartimento Interaziendale di Medicina Trasfusionale ed Ematologia - ASST Papa Giovanni XXIII ematologia Piazza OMS, 1
Bergamo, 24127
- Recruiting
Universita degli Studi di Milano - IRCCS Ospedale Maggiore Policlinico - Centro Emofilia e Trombosi Angelo Bianchi Bonomi
Milan
- Recruiting
University of Padua Medical School
Padua
- Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma
Spain4 sites, 4 recruiting
- Recruiting
Hospital Universitario La Paz
Madrid, 28046
- Recruiting
Hospital Universitario Morales Meseguer
Murcia
- Recruiting
Ourense University Hospital
Ourense, 32005
- Recruiting
Central University Hospital of Asturias
Oviedo, Principality of Asturias, 33011
France3 sites, 3 recruiting
- Recruiting
Centre de Référence de l'Hémophilie Unité d'Hémostase Clinique Hôpital Cardiologique Louis Pradel
Bron
- Recruiting
University Hospital of Reims
Reims
- Recruiting
Centre Hospitalier Universitaire de Rouen (CHU de Rouen) - Centre de Traitement des Maladies Hemorragiques (CRTH) (Centre d'Hemophiles)
Rouen
Germany3 sites, 3 recruiting
- Recruiting
Vivantes Klinikum Friedrichshain Klinik für Angiologie Hämostaseologie
Berlin
- Recruiting
UKB Bonn Institut für Experimentelle Hämatologie und Transfusionsmedizin
Bonn
- Recruiting
Gerinnungszentrum Rhein-Ruhr
Duisburg
Israel2 sites, 2 recruiting
- Recruiting
Rabin Medical Centre, Institute of Haematology
Petah Tikva
- Recruiting
Sheba Medical Centre
Ramat Gan
United Kingdom2 sites, 2 recruiting
- Recruiting
Royal-free Hospital-Katherine Dormandy Haemophilia and Thrombosis Centre
London
- Recruiting
St. Thomas Hospital, Guy's and St. Thomas' NHS Foundation Trust
London
Armenia1 site, 1 recruiting
- Recruiting
Yeolyan Hematology and Oncology Centre
Yerevan
Austria1 site, 1 recruiting
- Recruiting
Medizinische Universität Wien. Universitätsklinik für Innere Medizin Klinische Abteilung für Hämatologie und Hämostaseologie
Vienna
Czechia1 site, 1 recruiting
- Recruiting
Centre for Thrombosis and Haemaostasis Nemocnice
Nymburk
Hungary1 site, 1 recruiting
- Recruiting
University of Debrecen, Medical and Health Science Centre
Debrecen
Romania1 site, 1 recruiting
- Recruiting
Emergency County Hospital Craiova
Craiova
About this study
Official title: A Multicenter, Prospective, Open-label, Uncontrolled Phase 3 Study to Assess the Efficacy, Safety and Pharmacokinetics of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery
The goal 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 when they receive Atenativ for surgical procedures or parturition.
- Conditions
- Congenital Antithrombin Deficiency
- Being tested
- Atenativ
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AtenativDrug
Antithrombin concentrate
| Study group | Role | What they receive |
|---|---|---|
| Atenativ treatment | Experimental | During the PK phase, patients will receive a 60 IU/kg Atenativ as a single intravenous infusion for PK analysis.
During the treatment phase, patients will receive a single intravenous loading dose followed by maintenance doses administered every 24 hours for approximately 2-7 days for surgical patients and approximately 5 days for parturients Receives: Drug: Atenativ |
What is being measured
The primary outcome is the main question the study is designed to answer.
Thrombotic event incidence
The primary objective of this study is to assess the incidence of the composite of TEs and TEEs in patients with congenital antithrombin deficiency under cover of Atenativ for surgical procedures or parturition
Measured: Up to day 30 post treatment initiation
Secondary outcomes (13)
Single dose Pharmacokinetics of Atenativ: Area under the curve (AUCnorm(0-∞))
Assess the area under the curve (AUCnorm(0-∞)) of Atenativ in patients with congenital antithrombin deficiency
Measured: Up to day 14 post PK infusion
Single dose Pharmacokinetics of Atenativ: Maximum plasma concentration (Cmax)
Assess the maximum plasma concentration (Cmax) after a single dose of Atenativ in patients with congenital antithrombin deficiency
Measured: Before first infusion, 20 minutes, 1 hour, 3 hours, 8 hrs, 1 day, 2 days, 3, days, 4 days, 5 days, 6 days, 7 days, 8 days, 10 days, 12 days and 14 days post-infusion
Single dose Pharmacokinetics of Atenativ: Half-life (t1/2)
Assess the half-life (t1/2) after a single dose of Atenativ in patients with congenital antithrombin deficiency
Measured: Before first infusion, 20 minutes, 1 hour, 3 hours, 8 hrs, 1 day, 2 days, 3, days, 4 days, 5 days, 6 days, 7 days, 8 days, 10 days, 12 days and 14 days post-infusion
Single dose Pharmacokinetics of Atenativ: Mean residence time (MRT)
Assess the mean residence time (MRT) after a single dose of Atenativ in patients with congenital antithrombin deficiency
Measured: Before first infusion, 20 minutes, 1 hour, 3 hours, 8 hrs, 1 day, 2 days, 3, days, 4 days, 5 days, 6 days, 7 days, 8 days, 10 days, 12 days and 14 days post-infusion
Single dose Pharmacokinetics of Atenativ: Clearance (CL)
Assess the clearance (CL) after a single dose of Atenativ in patients with congenital antithrombin deficiency
Measured: Before first infusion, 20 minutes, 1 hour, 3 hours, 8 hrs, 1 day, 2 days, 3, days, 4 days, 5 days, 6 days, 7 days, 8 days, 10 days, 12 days and 14 days post-infusion
Single dose Pharmacokinetics of Atenativ: Incremental in vivo recovery (IVR)
Assess the Incremental in vivo recovery after a single dose of Atenativ in patients with congenital antithrombin deficiency
Measured: Before first infusion, 20 minutes, 1 hour, 3 hours, 8 hrs, 1 day, 2 days, 3, days, 4 days, 5 days, 6 days, 7 days, 8 days, 10 days, 12 days and 14 days post-infusion
Single dose Pharmacokinetics of Atenativ: Volume of distribution at steady state (Vss)
Assess the volume of distribution at steady state (Vss) after a single dose of Atenativ in patients with congenital antithrombin deficiency
Measured: Before first infusion, 20 minutes, 1 hour, 3 hours, 8 hrs, 1 day, 2 days, 3, days, 4 days, 5 days, 6 days, 7 days, 8 days, 10 days, 12 days and 14 days post-infusion
Single dose Pharmacokinetics of Atenativ: Maximum Plasma Concentration (Tmax)
Assess the time to reach Maximum Plasma Concentration (Tmax) after a single dose of Atenativ in patients with congenital antithrombin deficiency
Measured: Before first infusion, 20 minutes, 1 hour, 3 hours, 8 hrs, 1 day, 2 days, 3, days, 4 days, 5 days, 6 days, 7 days, 8 days, 10 days, 12 days and 14 days post-infusion
10. Coagulation parameters: Activated partial thromboplastin time [aPTT]
Assess activated partial thromboplastin time [aPTT] in patients with congenital antithrombin deficiency undergoing surgical procedures or parturition
Measured: Up to day 7 post treatment initiation
Coagulation parameters: Prothrombin time [PT]
Assess prothrombin time [PT] in patients with congenital antithrombin deficiency undergoing surgical procedures or parturition
Measured: Up to day 7 post treatment initiation
Coagulation parameters: International normalised ratio [INR]
Assess international normalised ratio [INR] in patients with congenital antithrombin deficiency undergoing surgical procedures or parturition
Measured: Up to day 7 post treatment initiation
Coagulation parameters: Fibrinogen level
Assess fibrinogen in patients with congenital antithrombin deficiency undergoing surgical procedures or parturition
Measured: Up to day 7 post treatment initiation
Safety and tolerability: Number of adverse events (AEs)
Number of adverse events (AEs) following treatment with Atenativ in patients with congenital antithrombin deficiency
Measured: Up to day 30 post treatment initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2021
- Study start1 Jul 2022
- Primary completion1 Dec 2026 (estimated)
- Study completion1 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Antithrombin DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04918173
- Sponsor's protocol ID
- ATN-106
- Status last verified
- Sep 2026
- First submitted
- 18 May 2021
- Last updated
- 28 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No