Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia

NCT02585635 | Completed

• 1 other identifier: H15-263263
• Study Type: observational
• Target: 9
• Locations: 0 countries
• Sites: N/A

Brief Summary

The MOrPH study is designed to identify optimal prophylaxis schedules for children with haemophilia. This involves development of combined pharmacokinetic and pharmacodynamic models. Interpretation of model outputs will be informed by two surveys. The first will survey families of children with haemophilia to ascertain families' values and preferences concerning prophylaxis schedules. The second will survey haemophilia physicians to ascertain the criteria physicians use to prescribe prophylaxis schedules.

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (3)

• Acceptability of injection frequencies

  Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different frequencies of prophylactic factor injections.

  Day 1 (i.e. on the day of response to the survey)

• Acceptability of time of injections

  Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different injection times.

  Day 1 (i.e. on the day of response to the survey)

• Importance of factors influencing prescription of prophylaxis schedules

  Physicians will be asked to rank the importance of factors (including cost, tolerability for families, venous access, physical activity and sport, pharmacokinetics, inhibitor development and age) that influence their prescription of prophylaxis schedules.

  Day 1 (i.e. on the day of response to the survey)

Study Arms (2)

Families

Families of children with haemophilia

Clinicians

Haemophilia physicians

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

There will be two study populations: 1. Families of children with haemophilia 2. Haemophilia physicians

You may qualify if:

• People will be eligible to participate in the family survey if they have haemophilia A or B and are aged between 14 and 17 years, or if they are the parent of a child (\< 18 years) with haemophilia
• People will be eligible to participate in the physician survey if they are physicians currently practising in paediatric haemophilia treatment centres.

You may not qualify if:

• Unable to communicate effectively in English.

Sponsors & Collaborators

• Neuroscience Research Australia: lead
• Sydney Children's Hospitals Network: collaborator
• The George Institute: collaborator
• Royal Children's Hospital: collaborator

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Rob Herbert, PhD

  Neuroscience Research Australia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 14, 2015
• First Posted: October 23, 2015
• Study Start: March 1, 2016
• Primary Completion: February 1, 2018
• Study Completion: February 1, 2018
• Last Updated: March 29, 2018

Record last verified: 2018-03

Data Sharing

• IPD Sharing: Will not share