Trial Outcomes & Findings for Pilot Dose Finding and Pharmacokinetic Study of Fondaparinux in Children With Thrombosis (NCT NCT00412464)

Results Overview

The primary outcome measure was assessment of safety by reporting the number of abnormal lab results resulting in adverse events. Safety laboratory assessments are as follows: Liver and kidney toxicity will be determined by serial measurements of AST, ALT, total bilirubin, BUN and creatinine. Hematologic toxicity will be assessed by serial CBCs.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

24 participants

Primary outcome timeframe

Study period which was up to 21 days of fondaparinux

Results posted on

2015-04-15

Participant Flow

Subjects were recruited between September, 2006 and June, 2009 at 4 sites (Children's Hospital Los Angeles, Children's Hospital of Orange County, Texas Children's Hospital and Nationwide Children's Hospital). All subjects were in the inpatient units of these hospitals for the duration of their study participation.

All recruited subjects entered and completed the study.

Participant milestones

Participant milestones
Measure	Fondaparinux Group Single arm group receiving fondaparinux 0.1 mg/kg.
Overall Study STARTED	24
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Dose Finding and Pharmacokinetic Study of Fondaparinux in Children With Thrombosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fondaparinux Group n=24 Participants Single arm group receiving fondaparinux 0.1 mg/kg.
Age, Categorical <=18 years	24 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	12 years STANDARD_DEVIATION 4 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Region of Enrollment United States	24 participants n=5 Participants

PRIMARY outcome

Timeframe: Study period which was up to 21 days of fondaparinux

The primary outcome measure was assessment of safety by reporting the number of abnormal lab results resulting in adverse events. Safety laboratory assessments are as follows: Liver and kidney toxicity will be determined by serial measurements of AST, ALT, total bilirubin, BUN and creatinine. Hematologic toxicity will be assessed by serial CBCs.

Outcome measures

Outcome measures
Measure	Fondaparinux Group n=24 Participants Single arm group receiving fondaparinux 0.1 mg/kg.
Number of Abnormal Lab Results Resulting in Adverse Events.	0 Adverse Events

PRIMARY outcome

Timeframe: 21 days

Subjects all had detailed pharmacokinetic measurements done which were subsequently analyzed in a population pharmacokinetic model. This model then informed the dosing recommendations that were published as a result of the study.

Outcome measures

Outcome measures
Measure	Fondaparinux Group n=24 Participants Single arm group receiving fondaparinux 0.1 mg/kg.
Therapeutic Plasma Concentration of Fondaparinux at 21 Days	24 mg/dL Standard Deviation 0.001

PRIMARY outcome

Timeframe: Study period which was up to 21 days of fondaparinux

Bleeding assessment Patients will be monitored for bleeding symptoms by physician and nursing assessment. Major bleeding will be defined as bleeding which is in a critical space (intracranial, retroperitoneal, or visceral) or leads to the need for blood transfusion. Minor bleeding will be all other bleeding and will be classified as clinically significant (i.e. If the physician has to take action to treat the minor bleed) or clinically insignificant (i.e. if the physician does not need to intervene to treat the minor bleed).

Outcome measures

Outcome measures
Measure	Fondaparinux Group n=24 Participants Single arm group receiving fondaparinux 0.1 mg/kg.
Bleeding Events	2 Adverse Events

PRIMARY outcome

Timeframe: Study period which was up to 21 days of fondaparinux

Adverse events will be defined as any untoward or unexpected event which can be a symptom, physical exam sign or laboratory abnormality. Adverse events will be classified as serious if they lead to prolonged hospitalization, re-hospitalization, transfer to an intensive care unit, or death. Adverse events will be categorized in terms of their likely association with fondaparinux as probably related, possibly related, unrelated, or unknown, and will be recorded according to standard adverse reporting guidelines for clinical trials.

Outcome measures

Outcome measures
Measure	Fondaparinux Group n=24 Participants Single arm group receiving fondaparinux 0.1 mg/kg.
Adverse Events	0 Adverse Events

PRIMARY outcome

Timeframe: Study period which was up to 21 days of fondaparinux

Patient's platelet counts should be kept above 50 x 10\^9/L while on study with platelet transfusions as needed with the exception of patients enrolled under the HIT/ suspicion of HIT inclusion (platelet transfusions are contraindicated in HIT). With regards to study patients who experience progressive decreases in platelet count to below 50 x 10\^9/L while receiving fondaparinux (excluding patients being treated for HIT or suspicion of HIT), fondaparinux will be discontinued.

Outcome measures

Outcome measures
Measure	Fondaparinux Group n=24 Participants Single arm group receiving fondaparinux 0.1 mg/kg.
Thrombocytopenic Events	0 Adverse Events

Adverse Events

Fondaparinux Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Guy Young

Children's Hospital Los Angeles

Phone: 323-361-5507

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place