NCT01085344

Brief Summary

Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis while reducing disability to a greater degree than intermittent therapy.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
56

participants targeted

Target at P25-P50 for phase_4

Timeline
Completed

Started Jun 1997

Longer than P75 for phase_4

Geographic Reach
1 country

1 active site

Status
completed

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 26, 1997

Completed
12.7 years until next milestone

First Submitted

Initial submission to the registry

March 1, 2010

Completed
10 days until next milestone

First Posted

Study publicly available on registry

March 11, 2010

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2012

Completed
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2014

Completed
5 years until next milestone

Results Posted

Study results publicly available

December 5, 2019

Completed
Last Updated

December 5, 2019

Status Verified

November 1, 2019

Enrollment Period

15.4 years

First QC Date

March 1, 2010

Results QC Date

May 10, 2018

Last Update Submit

November 18, 2019

Conditions

Severe Hemophilia A

Keywords

hemophilia, prophylaxis, cost effectiveness

Outcome Measures

Primary Outcomes (1)

  • Number of Participants Who Developed Target Joint Bleeding

    The number of participants who developed target joint bleeding during the study, which was defined as 3 bleeds into any 1 joint within a period of 3 months.

    6 months

Secondary Outcomes (6)

  • Annualized Bleeding Rate

    6 months

  • Annualized Factor Use

    12 months

  • Number of Patients Who Developed an Inhibitor to FVIII

    6 months

  • Physical Disability as Measured by the CHAQ

    through study completion, a median of 10 years

  • Joint Damage as Determined by the Physiotherapy Score

    through study completion, a median of 10 years

  • +1 more secondary outcomes

Study Arms (1)

Factor VIII

EXPERIMENTAL

escalating dose Factor VIII

Biological: Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)

Interventions

Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)BIOLOGICAL

escalating dose prophylaxis

Also known as: Recombinant Factor VIII (antihemophilic agent)
Factor VIII

Eligibility Criteria

Age12 Months - 30 Months
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Severe hemophilia A (factor level less than 2%).
  • Age greater than 1 year and less than or equal to 2.5 years.
  • Normal joints using the World Federation of Hemophilia orthopedic scale.
  • Normal radiographs of joints in which bleeding has occurred using the World Federation of Hemophilia radiographic scale.
  • Platelet count of \> 150,000.
  • Informed consent to participate.

You may not qualify if:

  • Three or more clinically determined bleeds into any single elbow, knee or ankle.
  • Presence or past history of a circulating inhibitor (level ≥ 0.5 Bethesda Units).
  • Family judged to be non-compliant by the local hemophilia clinic director.
  • Competing risk (symptomatic HIV infection, juvenile rheumatoid arthritis, metabolic bone disease, or other diseases known to cause or mimic arthritis.)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Related Publications (1)

  • Feldman BM, Rivard GE, Babyn P, Wu JKM, Steele M, Poon MC, Card RT, Israels SJ, Laferriere N, Gill K, Chan AK, Carcao M, Klaassen RJ, Cloutier S, Price VE, Dover S, Blanchette VS. Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort. Lancet Haematol. 2018 Jun;5(6):e252-e260. doi: 10.1016/S2352-3026(18)30048-6. Epub 2018 May 3.

    PMID: 29731369DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Results Point of Contact

Title
Dr. Brian Feldman
Organization
The Hospital for Sick Children
brian.feldman@sickkids.ca
416-813-5828

Study Officials

  • Brian M Feldman, MD

    The Hospital for Sick Children

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Division Head, Rheumatology

Study Record Dates

First Submitted

March 1, 2010

First Posted

March 11, 2010

Study Start

June 26, 1997

Primary Completion

December 1, 2012

Study Completion

December 1, 2014

Last Updated

December 5, 2019

Results First Posted

December 5, 2019

Record last verified: 2019-11

Locations

CA(1)