NCT00319228

Brief Summary

To assess the safety, pharmacokinetics and efficacy of a plasma-derived AT-III concentrate in the treatment of subjects with congenital AT-III deficiency.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
34mo left

Started Jan 2006

Longer than P75 for phase_2

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress88%
Jan 2006Mar 2029

Study Start

First participant enrolled

January 1, 2006

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

April 26, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 27, 2006

Completed
22.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2028

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2029

Last Updated

August 25, 2023

Status Verified

August 1, 2023

Enrollment Period

22.9 years

First QC Date

April 26, 2006

Last Update Submit

August 24, 2023

Conditions

Antithrombin III Deficiency

Keywords

AT-III deficiencyAT-III concentrateBleeding disordersBlood disorders

Outcome Measures

Primary Outcomes (4)

  • The primary objectives of this clinical study are to:

    2 years

  • Assess the pharmacokinetic (PK) profile of AT-III in congenital AT-III deficient patients

    1 year

  • To measure the in vivo recovery and half-life of AT-III.

    1 year

  • To assess the clinical safety and tolerability of AT-III-DAF/DI.

    1 year

Secondary Outcomes (1)

  • To assess clinical efficacy by preventing thromboembolic or thrombotic events (prophylaxis) in individuals with congenital AT-III deficiency who are undergoing surgical procedures or who are pregnant and undergoing parturition.

    1 year

Study Arms (1)

Antithrombin III

EXPERIMENTAL
Drug: Plasma-derived AT-III concentrate

Interventions

Plasma-derived AT-III concentrateDRUG

Segment I: A single dose IV infusion of 50 IU/kg of ATIII-DAF/DI will be administered to each patient. Segment II: A single dose or multiple doses depending on the subject's ATIII plasma levels and patient's specific treatment plan.

Antithrombin III

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Congenital ATIII deficiency documented by determination of plasma levels of ATIII off all therapies. Specifically, the baseline levels of ATIII activity should be equal to or less than 60%.
  • Age \>12 years with a body weight of no less than 30 kg.
  • Have not participated in another investigational study for at least 30 days. For Segment II, enrollment requires a pregnancy/delivery or a surgical procedure (it should be a major surgery although data from a minor surgery will also be collected).
  • Documented personal history of major thromboembolic or thrombotic event.
  • Male or female
  • HIV, HBV, HCV, HAV and PARVO B19 status known prior to entry.
  • The subject is willing to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
  • The subject has signed an informed consent form (if at least 18 years old), or the subject's parent or legal guardian has signed the informed consent form. Subjects below the age of 18 years will also be asked to sign an assent form. All consent and assent forms must be approved in advance by the Institutional Review Board of the investigator's institution.
  • Patients with heparin-associated thrombocytopenia who require anticoagulation with non-heparin containing drugs will be eligible if they can be safely transitioned during the washout period for the Segment I PK study.
  • If pregnant, a woman must be Parvo B19 IgG antibody positive.

You may not qualify if:

  • Acquired deficiency of ATIII.
  • Receiving concomitant treatment for thrombophilic disorders other than ATIII deficiency.
  • Inability or unwillingness to comply with the protocol requirements.
  • History of anaphylactic reaction(s) to blood or blood components.
  • Allergies to excipients.
  • Liver function tests \>/= 2.5 X ULN
  • Serum creatinine \>1.2 X ULN.
  • Urine \>/= 2+ protein with urine dipstick test.
  • The subject is known to have abused alcohol or illicit drugs within the past 12 months.
  • The subject is unlikely to adhere to the protocol requirements of the study or is likely to be uncooperative or unable to provide a storage serum sample at the screening visit.
  • Patients on heparin-treatment who, for clinical reasons, cannot safely be discontinued from heparin therapy during the PK segment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Antithrombin III DeficiencyHemostatic DisordersHematologic Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHemic and Lymphatic DiseasesBlood Protein DisordersThrombophiliaGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesVascular DiseasesCardiovascular DiseasesHemorrhagic Disorders

Study Officials

  • Paul Pinciaro, PhD

    Grifols Biologicals, LLC

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
PREVENTION
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 26, 2006

First Posted

April 27, 2006

Study Start

January 1, 2006

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

March 1, 2029

Last Updated

August 25, 2023

Record last verified: 2023-08