Severe Hemophilia APhase 3NCT03376516

Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A

A Phase 3 study testing Wilate in people with Severe Hemophilia A, run by Octapharma at 3 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

A prospective, non-controlled, international, multi-centre phase 3 study to investigate the pharmacokinetics, efficacy, safety, and immunogenicity of Wilate in previously treated children with severe haemophilia A Read more about this study

Who is it for?: Ages 1 year to 11 years, men only
Conditions the study is for: Severe Hemophilia A
What is being tested?: Wilate
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 11 participants
Where: 3 sitesin 2 countries
Study dates: 22 Nov 2017to 3 Nov 2018
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 1 year to 11 years
Men only
No healthy volunteers
Child

Who may be able to join

Severe haemophilia A (<1% FVIII:C) according to medical history
Male patients aged 1 to <12 years
Previous treatment with a FVIII concentrate for at least 50 exposure days (EDs)
Immunocompetence (CD4+ count >200/μL)
Voluntarily given, fully informed written and signed consent obtained by the patient's parent(s) or legal guardian and, depending on the children's developmental stage and intellectual capacity, informed assent by the patients before any study-related procedures are performed
The interval between the Screening Visit and the PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/μL for patients to be enrolled (i.e., inclusion criterion no. 4).

Who cannot join

Any coagulation disorders other than haemophilia A
History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory antibodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 μmol/L)
Patients receiving or scheduled to receive immunomodulating drugs (other than antiretroviral chemotherapy), such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs

Trial locations and contacts

3 sites in 2 countries.

Country

City, postcode or hospital

3 sites in 2 countries

Ukraine2 sites

"National Children's Specialized Clinic "OKHMATDYT"
Kyiv
"Western Ukrainian Specialized Children's Medical Center"
Lviv

Other trials in Ukraine

Russia1 site

Kirov SSC Hematology and Transfusiology
Kirov

Other trials in Russia

About this study

Official title: Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A

Conditions: Severe Hemophilia A
Being tested: Wilate
Medical subject headings: Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

WilateDrug
von Willebrand factor / Factor VIII (plasma derived)

Study groups in NCT03376516
Study group	Role	What they receive
All patients	Experimental	All patients will receive Wilate for prophylactic treatment. Patients will also receive Wilate for treatment of breakthrough bleeding events as required Receives: Drug: Wilate

What is being measured

The primary outcome is the main question the study is designed to answer.

Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate AUC is hours (h) x international units (IU)/decilitre (dL).
Measured: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Normalised (AUCNorm)) of FVIII:C for Wilate
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The mean area under the curve normalised for the administered dose (AUCnorm) was calculated for Wilate.
The units of measure used were AUC divided by dose.
Measured: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
Pharmacokinetic (PK) Assessment (Half-life (h)) of FVIII:C
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate half-life is hours.
Measured: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration) for FVIII:C
PK assessments of FVIII:C were determined using the one-stage (OS) assays. The maximum plasma concentration of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
Units of measure for maximum plasma concentration are international units (IU)/ decilitre (dL)
Measured: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
Pharmacokinetic (PK) Assessment (Time to Reach Maximum Plasma Concentration (Tmax)) of FVIII:C
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate Tmax is hours (h).
Measured: 48 h following a single dose of Wilate
Pharmacokinetic (PK) Assessment (Mean Residence Time (MRT)) of FVIII:C
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate MRT is hours.
Measured: 48 h following a single dose of Wilate
Pharmacokinetic (PK) Assessment (Volume of Distribution (Vd)) of FVIII:C
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate Vd is decilitre (dL)/ kilograms (kg).
Measured: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
Pharmacokinetic (PK) Assessment (Clearance) of FVIII:C
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate clearance are decilitre (dL)/ hours (h)/ kilograms (kg).
Measured: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
Incremental In Vivo Recovery (IVR) of FVIII:C
The incremental IVR was determined from all patients at baseline was determined using the one-stage (OS) assay (standardised to 50 IU/kg).
The units of measure to calculate IVR is kilograms (kg) / deciliter (dL)
Measured: 48 h following a single dose of Wilate

Secondary outcomes (10)

Total Annualized Bleeding Rate (TABR)
The total number of bleeding events (BEs) in the time period between the first dose of IMP and the study completion visit, divided by the duration (in years) between the first dose of IMP and the study completion visit.
Surgery periods, and BEs occurring within these periods, will be excluded from the calculation of TABR.
Measured: 6 months
Spontaneous Annualized Bleeding Rate (SABR)
The SABR was calculated in analogy to the TABR. The total number of spontaneous bleeding events (BEs) in the time period between the first dose of IMP and the study completion visit, divided by the duration (in years) between the first dose of IMP and the study completion visit.
Surgery periods, and BEs occurring within these periods, will be excluded from the calculation of the SABR.
Measured: 6 months
Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)
The proportion of BEs successfully treated with Wilate was assessed by the patient (together with the investigator in case of on-site treatment) in a patient diary. The treatment efficacy for all BEs was assessed using a pre-defined four-point scale: 'excellent', 'good', 'moderate', 'none'. 'Excellent' was defined as "Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection" (best outcome); 'good 'was defined as "definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection, requiring up to 2 injections for complete resolution". All efficacy ratings assessed as either 'excellent' or 'good' were considered 'successfully treated'. 'Moderate' was defined as "probable or slight beneficial effect within approximately 12 hours after the first injection" and 'none' defined as "no improvement within 12 hours, or worsening of symptoms".
Measured: 6 months
Wilate Consumption Data: Average Dose of Wilate Per Week of Study
The average consumption of Wilate per week of the study (IU/kg) for all patients receiving prophylaxis
Measured: 6 months
Incremental in Vivo Recovery (IVR) of Wilate Over Time
The rise in FVIII:C activity in IU/dl per unit dose administered in IU/kg was determined for all patients at baseline, 3 and 6 months, using the one-stage (OS) assay.
Measured: Baseline, and 3 and 6 months of treatment
Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)
Analysis of variance (ANOVA) was used in an exploratory sense to assess a possible association between the ABO blood type and the FVIII:C half-life of Wilate. This was analysed by calculating the mean square in a one-stage (OS) assay.
Measured: 6 months
Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate
Analysis of variance (ANOVA) was used in an exploratory sense to assess a possible association between VWF:Ag with the FVIII:C half-life of Wilate. This was analysed by calculating the mean square in a one-stage (OS) assay.
Measured: 6 months
Safety and Tolerability of Wilate by Monitoring The Number of Adverse Events (AEs) Throughout the Study
At each visit (whether scheduled or unscheduled) , AEs will be documented by the investigator throughout the study. In addition, the investigator will check the patient diaries for any documented event.
Measured: 6 months
Immunogenicity of Wilate: Number of Participants With FVIII Inhibitor Activity at 6 Months
FVIII inhibitor activity was determined at each study visit: screening, PK, Day 14 visit, Day 30 visit, 3 Months visit and 6 Months visit before injection.
Measured: 6 months
Virus Safety Measured by the Number With Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study
Virus safety was evaluated by taking a plasma sample for parvovirus B19 antibody testing before the first injection of Wilate at the PK visit. All patients negative at screening were tested again at the Study Completion visit. The number of Parvovirus B19 seroconversions between BL and end of study was recorded
Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Nov 20173 Nov 2018

First posted18 Dec 2017
Study start22 Nov 2017
Primary completion3 Nov 2018
Study completion3 Nov 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Severe Hemophilia AStudies of the same condition, open and under way.
More trials run by OctapharmaThe sponsor's other registered studies.
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: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 11 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03376516
Sponsor's protocol ID: WIL-30
Status last verified: Dec 2020
First submitted: 13 Dec 2017
Last updated: 19 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Severe Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
ClinicalTrials.gov ID: NCT03376516
Acronym / sponsor ID: WIL-30
Conditions: Severe Hemophilia A
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 11 participants (actual)
Lead sponsor: OctapharmaIndustry (pharma, biotech, devices)
Study start: 22 Nov 2017 (actual)
Primary completion: 3 Nov 2018 (actual)
Study completion: 3 Nov 2018 (actual)
First posted: 18 Dec 2017 (actual)
Results: Posted 24 Dec 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
All patients	Experimental	Drug: WilateAll patients will receive Wilate for prophylactic treatment. Patients will also receive Wilate for treatment of breakthrough bleeding events as required

Who may qualify

Ages: 1 year to 11 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (6 listed)

Severe haemophilia A (<1% FVIII:C) according to medical history
Male patients aged 1 to <12 years
Previous treatment with a FVIII concentrate for at least 50 exposure days (EDs)
Immunocompetence (CD4+ count >200/μL)
Voluntarily given, fully informed written and signed consent obtained by the patient's parent(s) or legal guardian and, depending on the children's developmental stage and intellectual capacity, informed assent by the patients before any study-related procedures are performed

Key exclusion criteria (4 listed)

Any coagulation disorders other than haemophilia A
History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory antibodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 μmol/L)
Patients receiving or scheduled to receive immunomodulating drugs (other than antiretroviral chemotherapy), such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Wilatevon Willebrand factor / Factor VIII (plasma derived)	Drug	All patients

Primary outcome

Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C
Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate AUC is hours (h) x international units (IU)/decilitre (dL).
Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Normalised (AUCNorm)) of FVIII:C for Wilate
Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The mean area under the curve normalised for the administered dose (AUCnorm) was calculated for Wilate. The units of measure used were AUC divided by dose.
Pharmacokinetic (PK) Assessment (Half-life (h)) of FVIII:C
Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate half-life is hours.
Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration) for FVIII:C
Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
PK assessments of FVIII:C were determined using the one-stage (OS) assays. The maximum plasma concentration of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study. Units of measure for maximum plasma concentration are international units (IU)/ decilitre (dL)
Pharmacokinetic (PK) Assessment (Time to Reach Maximum Plasma Concentration (Tmax)) of FVIII:C
Time frame: 48 h following a single dose of Wilate
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate Tmax is hours (h).
Pharmacokinetic (PK) Assessment (Mean Residence Time (MRT)) of FVIII:C
Time frame: 48 h following a single dose of Wilate
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate MRT is hours.
Pharmacokinetic (PK) Assessment (Volume of Distribution (Vd)) of FVIII:C
Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate Vd is decilitre (dL)/ kilograms (kg).
Pharmacokinetic (PK) Assessment (Clearance) of FVIII:C
Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate
PK assessments of the factor VIII coagulant activity (FVIII:C) for Wilate were determined using the one-stage (OS) assay. The units of measure to calculate clearance are decilitre (dL)/ hours (h)/ kilograms (kg).
Incremental In Vivo Recovery (IVR) of FVIII:C
Time frame: 48 h following a single dose of Wilate
The incremental IVR was determined from all patients at baseline was determined using the one-stage (OS) assay (standardised to 50 IU/kg). The units of measure to calculate IVR is kilograms (kg) / deciliter (dL)

10 secondary outcomes

Total Annualized Bleeding Rate (TABR)
Time frame: 6 months
Spontaneous Annualized Bleeding Rate (SABR)
Time frame: 6 months
Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)
Time frame: 6 months
Wilate Consumption Data: Average Dose of Wilate Per Week of Study
Time frame: 6 months
Incremental in Vivo Recovery (IVR) of Wilate Over Time
Time frame: Baseline, and 3 and 6 months of treatment
Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)
Time frame: 6 months
Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate
Time frame: 6 months
Safety and Tolerability of Wilate by Monitoring The Number of Adverse Events (AEs) Throughout the Study
Time frame: 6 months
Immunogenicity of Wilate: Number of Participants With FVIII Inhibitor Activity at 6 Months
Time frame: 6 months
Virus Safety Measured by the Number With Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study
Time frame: 6 months

Results and safety

Results posted to the registry on 24 Dec 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Wilate	11	9

Primary outcome: Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C

Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate. Mean (Standard Deviation). Unit: h*IU/dL

Measure	Wilate n = 10
1-<6 years	768.8 (288.5)
6-<12 years	671.9 (289.7)
Total	720.3 (277.3)

Population analysed: The analysis was performed in the PK population which included all patients who underwent PK assessment during the study (total: n=10). The PK population comprised 5 patients aged 1-\<6 years and 5 patients aged 6-\<12 years.

Primary outcome: Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Normalised (AUCNorm)) of FVIII:C for Wilate

Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate. Mean (Standard Deviation). Unit: h*kg/dL

Measure	Wilate n = 10
1-<6 years	15.38 (5.77)
6-<12 years	13.44 (5.79)
Total	14.41 (5.55)

Primary outcome: Pharmacokinetic (PK) Assessment (Half-life (h)) of FVIII:C

Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate. Mean (Standard Deviation). Unit: hours

Measure	Wilate n = 10
1-<6 years	8.28 (1.51)
6-<12 years	9.35 (2.40)
Total	8.82 (1.97)

Primary outcome: Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration) for FVIII:C

Time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate. Mean (Standard Deviation). Unit: IU/dL

Measure	Wilate n = 10
1-<6 years	83.0 (16.5)
6-<12 years	78.94 (26.56)
Total	80.99 (20.94)

10 secondary outcomes are reported in the full results.

Adverse events

Time frame: Between the screening visit and the follow up contact visit (30 days after the 6-month study completion visit). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Wilate	1 of 10 (10%)	3 of 10 (30%)	0 of 10

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Cristina Solomon, MD study director

Octapharma

3 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Wilate in people with Severe Hemophilia A, run by Octapharma at 3 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 19 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Ukraine, Russia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 Dec 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Pharmacokinetic (PK) Assessment (Area Under the Curve (AUC)) of FVIII:C (time frame: 0h, 0.25h, 1h, 6h, 24h and 48 h following a single dose of Wilate). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03376516.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jan 2021.