Desmopressin and FVIII concentrate combination treatment in non-severe hemophilia A patients undergoing minor interventions

Desmopressin and FVIII concentrate combination treatment in non-severe hemophilia A patients undergoing minor interventions - Little-DAVID

Status

Not yet recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-001875-57-NL

Enrollment

Registered

2016-12-19

Start date

2017-03-13

Completion date

Unknown

Last updated

2019-12-16

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

Conditions

Hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10053753 Term: Hemophilia A without inhibitors System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT

Interventions

Trade Name: Minrin Pharmaceutical Form: Solution for injection/infusion Trade Name: Octostim Pharmaceutical Form: Solution for injection

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: In order to be eligible for this study, a subject must meet all of the following criteria: - Non-severe hemophilia A patients (FVIII 0.01-0.40 IU/mL) - In need of a minor surgical intervention - Age minimally 12 and maximally 70 years at study inclusion date - Need for perioperative FVIII concentrates for a maximum of 48 hours - Having admissible results of a desmopressin test (see paragraph 3.1) - Absolute increase in FVIII 1 hour after desmopressin administration = 0.2 IU/mL after a previous (test) dose - Male gender - (Parental) informed consent Are the trial subjects under 18? yes Number of subjects for this age range: 7 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 61 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 7

Exclusion criteria

Exclusion criteria: A potential subject who meets any of the following criteria will be excluded from participation in this study: - Patients with other congenital or acquired hemostatic abnormalities - Clinically relevant FVIII inhibiting antibodies (>0.5 BU) preoperatively, unless successfully treated with immunotolerance therapy - Needed treatment duration with FVIII concentrates longer than 48 hours - Contraindications for desmopressin, e.g. cardiovascular disease (see appendix IV) - Use of co-medication that has an interaction with desmopressin (see appendix IV) - Intolerance to previous desmopressin administrations

Design outcomes

Primary

Measure	Time frame
Main Objective: Primary Objectives: 1) To show the efficacy of desmopressin and FVIII concentrate combination treatment is equal to the efficacy of FVIII monotherapy 2) To show a reduction in FVIII concentrate consumption with desmopressin and FVIII concentrate combination treatment compared to FVIII monotherapy ; Secondary Objective: Secondary Objectives: a) To evaluate the safety of both treatment arms, i.e. occurrence of bleeding, dosing below target and other adverse events. b) To perform an economical evaluation to quantify the costs of combination treatment compared to standard treatment. c) To compare the proportion of patients with FVIII plasma levels within set target levels after the minor intervention. d) To evaluate experienced quality of care in participating patients. e) To evaluate discrepancies between one-stage and chromogenic FVIII-measurements before and after treatment with desmopressin. f) To evaluate the occurrence of FVIII inhibitors. ;Timepoint(s) of evaluation of this end point: Both endpoints will be evaluated at the end of study.; Primary end point(s): To assess our first main objective, efficacy of treatment is measured. Efficacy is defined as the average deviation of FVIII peak level to the predicted FVIII peak range in IU/mL in each treatment arm, without administering FVIII concentrate outside the dosing advice. The FVIII level for the primary endpoint is measured prior to the minor intervention, 10-15 minutes after the infusion of FVIII concentrate. The measurement for the primary endpoint is marked in appendix VIII. The deviation will be set to 0 for every FVIII measurement that falls into the predicted FVIII range. The predicted FVIII range will be set to the predicted peak level +/- 10%. Example: A patient has a target target trough FV	—

Measure

Time frame

Main Objective: Primary Objectives: 1) To show the efficacy of desmopressin and FVIII concentrate combination treatment is equal to the efficacy of FVIII monotherapy 2) To show a reduction in FVIII concentrate consumption with desmopressin and FVIII concentrate combination treatment compared to FVIII monotherapy ; Secondary Objective: Secondary Objectives: a) To evaluate the safety of both treatment arms, i.e. occurrence of bleeding, dosing below target and other adverse events. b) To perform an economical evaluation to quantify the costs of combination treatment compared to standard treatment. c) To compare the proportion of patients with FVIII plasma levels within set target levels after the minor intervention. d) To evaluate experienced quality of care in participating patients. e) To evaluate discrepancies between one-stage and chromogenic FVIII-measurements before and after treatment with desmopressin. f) To evaluate the occurrence of FVIII inhibitors. ;Timepoint(s) of evaluation of this end point: Both endpoints will be evaluated at the end of study.; Primary end point(s): To assess our first main objective, efficacy of treatment is measured. Efficacy is defined as the average deviation of FVIII peak level to the predicted FVIII peak range in IU/mL in each treatment arm, without administering FVIII concentrate outside the dosing advice. The FVIII level for the primary endpoint is measured prior to the minor intervention, 10-15 minutes after the infusion of FVIII concentrate. The measurement for the primary endpoint is marked in appendix VIII. The deviation will be set to 0 for every FVIII measurement that falls into the predicted FVIII range. The predicted FVIII range will be set to the predicted peak level +/- 10%. Example: A patient has a target target trough FV

—

Secondary

Measure	Time frame
Secondary end point(s): a) Number and nature of bleeding during the first 14 days after the minor intervention (appendix VI) a) Other adverse events during the first 14 days after the minor intervention b) Treatment costs in both treatment arms c) The proportion of patients with FVIII plasma levels within set target levels after the minor intervention d) Experienced quality of care in participating patients e) Discrepancies between one-stage and chromogenic FVIII-measurements before and after desmopressin administration f) Inhibitor development 4-6 weeks after the minor intervention ;Timepoint(s) of evaluation of this end point: Every endpoint will be evaluated at the end of study	—

Measure

Time frame

Secondary end point(s): a) Number and nature of bleeding during the first 14 days after the minor intervention (appendix VI) a) Other adverse events during the first 14 days after the minor intervention b) Treatment costs in both treatment arms c) The proportion of patients with FVIII plasma levels within set target levels after the minor intervention d) Experienced quality of care in participating patients e) Discrepancies between one-stage and chromogenic FVIII-measurements before and after desmopressin administration f) Inhibitor development 4-6 weeks after the minor intervention ;Timepoint(s) of evaluation of this end point: Every endpoint will be evaluated at the end of study

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Countries

Netherlands

Contacts

Public ContactDepartment of Hematology

Erasmus University Medical Center Rotterdam

david@erasmusmc.nl+31107033123

Outcome results

None listed

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