Combination treatment with DDAVP and factor VIII clotting factor concentrates in patients with mild haemophilia A.

DDAVP treatment combined with FVIII clotting factor concentrates in patients with mild haemophilia A. - DAVID

Status

Not yet recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-005435-14-NL

Enrollment

Unknown

Registered

2015-12-10

Start date

2016-05-19

Completion date

Unknown

Last updated

2016-05-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mild hemophilia A patients with a FVIII plasma levels above 0.05 IU/mL. MedDRA version: 18.1 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850 MedDRA version: 18.1 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850

Interventions

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Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: - Mild hemophilia A patients (FVIII > 0.05 IU/mL) - In need of surgery - Age between 12 and 70 years at study inclusion date - Need for perioperative clotting factor concentrates - Treatment duration with FVIII-concentrates of at least 48 hours - Results of FVIII levels after a DDAVP test dose, or if test results are not admissible, willingness to undergo a DDAVP test - Male gender - (Parental) informed consent Are the trial subjects under 18? yes Number of subjects for this age range: 6 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4

Exclusion criteria

Exclusion criteria: - Patients with other congenital or acquired hemostatic abnormalities - Very low response to DDAVP after 1 hour – absolute increase in FVIII 0.2 BU) in medical history or preoperatively, unless successfully treated with immunotolerance therapy - Contraindications for DDAVP, e.g. cardiovascular disease (see appendix IV) - Use of co-medication that has an interaction with DDAVP (see appendix IV) - Intolerance to previous DDAVP administrations - DDAVP not advisable due to the type of surgery according to the hematologist and/or surgeon

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the proportion of mild hemophilia A patients within FVIII target levels with the DDAVP and FVIII concentrate combination treatment in the first 72 hours postoperatively, without adding off-protocol FVIII concentrate. ;Secondary Objective: 1. To acquire data to improve the population based PK-model for perioperative DDAVP and FVIII concentrate combination treatment in mild hemophilia A patients. 2. To establish (possible) adverse events of combination treatment; e,g, side effects of DDAVP, bleeding episodes, development of neutralizing antibodies, thrombotic events. 3. To establish the proportion of mild hemophilia A patients that reaches FVIII target levels with the DDAVP and FVIII concentrate combination treatment preoperatively. 4. To establish the amount of off-protocol FVIII concentrates required. 5. To evaluate the intra-individual reproducibility of DDAVP response. 6. To evaluate DDAVP tachyphylaxis and its extent. 7. To perform an economical evaluation to quantify the potential cost reduction of the combination treatment. 8. To evaluate the experienced quality of care in participating patients ;Primary end point(s): The main endpoint will be the proportion of patients within FVIII target levels with DDAVP and FVIII concentrate combination treatment in the first 72 hours after surgery, without adding off-protocol FVIII concentrates. Off-protocol FVIII concentrates are defined as every extra dosage that is necessary to reach the target level, but was not in the original dosing advice. A patient is considered to meet the main endpoint if all pre-DDAVP measurements in the first 72 hours after surgery are within the target levels. The total number of measurements used for this purpose are 2 in patients with a treatment duration of 48 hours or 3 if treatment duration is more than 48 hours. The measurements for the primary endpoint are marked in appendix VII. If one measurement is below or above the limits of the target range, this pati	—

Measure

Time frame

Main Objective: To assess the proportion of mild hemophilia A patients within FVIII target levels with the DDAVP and FVIII concentrate combination treatment in the first 72 hours postoperatively, without adding off-protocol FVIII concentrate. ;Secondary Objective: 1. To acquire data to improve the population based PK-model for perioperative DDAVP and FVIII concentrate combination treatment in mild hemophilia A patients. 2. To establish (possible) adverse events of combination treatment; e,g, side effects of DDAVP, bleeding episodes, development of neutralizing antibodies, thrombotic events. 3. To establish the proportion of mild hemophilia A patients that reaches FVIII target levels with the DDAVP and FVIII concentrate combination treatment preoperatively. 4. To establish the amount of off-protocol FVIII concentrates required. 5. To evaluate the intra-individual reproducibility of DDAVP response. 6. To evaluate DDAVP tachyphylaxis and its extent. 7. To perform an economical evaluation to quantify the potential cost reduction of the combination treatment. 8. To evaluate the experienced quality of care in participating patients ;Primary end point(s): The main endpoint will be the proportion of patients within FVIII target levels with DDAVP and FVIII concentrate combination treatment in the first 72 hours after surgery, without adding off-protocol FVIII concentrates. Off-protocol FVIII concentrates are defined as every extra dosage that is necessary to reach the target level, but was not in the original dosing advice. A patient is considered to meet the main endpoint if all pre-DDAVP measurements in the first 72 hours after surgery are within the target levels. The total number of measurements used for this purpose are 2 in patients with a treatment duration of 48 hours or 3 if treatment duration is more than 48 hours. The measurements for the primary endpoint are marked in appendix VII. If one measurement is below or above the limits of the target range, this pati

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Secondary

Measure	Time frame
Secondary end point(s): 1. A population based pharmacokinetic model 2. Number and nature of adverse events during combined treatment 3. Incidence & severity of bleeding 4. Incidence of thrombosis 5. Incidence and extent of tachyphylaxis 6. Economical evaluation 7. Experienced quality of care ;Timepoint(s) of evaluation of this end point: 1,2,5: During hospital admission 3,4,6: Until 90 days after surgery 7: before and day 3 after surgery	—

Measure

Time frame

Secondary end point(s): 1. A population based pharmacokinetic model 2. Number and nature of adverse events during combined treatment 3. Incidence & severity of bleeding 4. Incidence of thrombosis 5. Incidence and extent of tachyphylaxis 6. Economical evaluation 7. Experienced quality of care ;Timepoint(s) of evaluation of this end point: 1,2,5: During hospital admission 3,4,6: Until 90 days after surgery 7: before and day 3 after surgery

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Countries

Netherlands

Contacts

Public ContactDepartment of Hematology

Erasmus University Medical Centre

m.kruip@erasmusmc.nl+31107033123

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026