HemophiliaJOSCNCT01000844
Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency
An observational study of Hemophilia, run by University of Colorado, Denver.
Trial summary
The original Joint Outcome Study (JOS) enrolled 65 boys with hemophilia from 16 sites nationally. The subjects were randomized to one of two arms (prophylaxis or an enhanced episode-based treatment)and were followed prospectively until the age of six. Read more about this study
- Who is it for?
- Ages 8 to 18 years, men only
- Conditions the study is for
- Hemophilia
- How many people
- 11 participants
- Where
- No sites listed yet
- Study dates
- Nov 2009to Oct 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
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 8 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Study population will be invited based on their enrollment in the original Joint Outcome Study (JOS). This population
Who may be able to join
- Enrolled in the original JOS study, "A Randomized Prospective Study for the Prevention of Joint Disease in Children with Factor VIII Deficiency"
- Written, informed consent of parent or guardian for the proposed study
- The local hemophilia treatment center staff must evaluate the family's participation in the original treatment protocol and determine that the family is capable of complying with the continuation protocol
Who cannot join
- Unable or unwilling to record the study information
About this study
Official title: Continuation of Children Enrolled in Protocol #95-011, 'A Randomized Prospective Study for the Prevention of Joint Disease in Children With Severe Factor VIII Deficiency'
The original Joint Outcome Study (JOS) enrolled 65 boys with hemophilia from 16 sites nationally. The subjects were randomized to one of two arms (prophylaxis or an enhanced episode-based treatment)and were followed prospectively until the age of six. At the age of six, the proportion of children on each treatment arm who developed bone or cartilage damage as determined by X-Ray or MRI was assessed. In addition, the function and structure of the index joints (defined as knees, ankles, and elbows)were evaluated using a physical assessment scale specially designed for preschool children.
The specific aim of the Joint Outcome Study Continuation (JOSC) is to extend observations of the children participating in the original JOS until the subjects reach the age of 18 years in order to determine the natural history of joint development in hemophilia and the impact of primary or secondary prophylaxis on the prevention, limitation, or reversal of hemophilic arthropathy. In addition, plasma and DNA will be collected and banked yearly for current and future studies of biomarkers and predictors of hemophilia outcomes.
- Conditions
- Hemophilia
- Keywords
- Hemophilia, Joint, Cartilage, Damage, Prophylaxis, Factor VIII, FVIII, Disease, Arthropathy
- Medical subject headings
- Hemophilia A, Disease, Joint Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the natuaral history of joint development in hemophilia and the impact of primary or secondary prophylaxis on the prevention, limitation, or reversal of hemophilic arthropathy.
Measured: Enrollment, age 14, and study exit at age 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2009 (estimated)
- Study startNov 2009
- Primary completionOct 2017
- Study completionOct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Collaborators
- Bayer, Rush University Medical Center, Phoenix Children's Hospital, The University of Texas Health Science Center, Houston, Oregon Health and Science University, Emory University, University of Texas Southwestern Medical Center, Prisma Health-Midlands, Indiana University, Intermountain Health Care, Inc., Ann & Robert H Lurie Children's Hospital of Chicago
- Responsible party
- Sponsor
- Investigators
- Marilyn Manco-Johnson, MD, Principal investigator, University of Colorado at Denver Health and Sciences Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01000844
- Sponsor's protocol ID
- 01-436
- Acronym
- JOSC
- Status last verified
- Oct 2019
- First submitted
- 22 Oct 2009
- Last updated
- 31 Oct 2019
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Warren BB, Thornhill D, Stein J, Fadell M, Ingram JD, Funk S, Norton KL, Lane HD, Bennett CM, Dunn A, Recht M, Shapiro A, Manco-Johnson MJ. Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study. Blood Adv. 2020 Jun 9;4(11):2451-2459. doi: 10.1182/bloodadvances.2019001311.
PubMed 32492157
Study at a glance
From the registry record, last updated by the sponsor on 31 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Continuation of Children Enrolled in Protocol #95-011, 'A Randomized Prospective Study for the Prevention of Joint Disease in Children With Severe Factor VIII Deficiency'
- ClinicalTrials.gov ID
- NCT01000844
- Acronym / sponsor ID
- JOSC / 01-436
- Conditions
- Hemophilia
- Keywords
- Hemophilia, Joint, Cartilage, Damage, Prophylaxis, Factor VIII, FVIII, Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Collaborators
- Bayer, Rush University Medical Center, Phoenix Children's Hospital, The University of Texas Health Science Center, Houston, Oregon Health and Science University, Emory University, University of Texas Southwestern Medical Center, Prisma Health-Midlands, Indiana University, Intermountain Health Care, Inc., Ann & Robert H Lurie Children's Hospital of Chicago
- Study start
- Nov 2009
- Primary completion
- Oct 2017 (actual)
- Study completion
- Oct 2017 (actual)
- First posted
- 23 Oct 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The original Joint Outcome Study (JOS) enrolled 65 boys with hemophilia from 16 sites nationally. The subjects were randomized to one of two arms (prophylaxis or an enhanced episode-based treatment)and were followed prospectively until the age of six. At the age of six, the proportion of children on each treatment arm who developed bone or cartilage damage as determined by X-Ray or MRI was assessed. In addition, the function and structure of the index joints (defined as knees, ankles, and elbows)were evaluated using a physical assessment scale specially designed for preschool children.
The specific aim of the Joint Outcome Study Continuation (JOSC) is to extend observations of the children participating in the original JOS until the subjects reach the age of 18 years in order to determine the natural history of joint development in hemophilia and the impact of primary or secondary prophylaxis on the prevention, limitation, or reversal of hemophilic arthropathy. In addition, plasma and DNA will be collected and banked yearly for current and future studies of biomarkers and predictors of hemophilia outcomes.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 8 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Study population will be invited based on their enrollment in the original Joint Outcome Study (JOS). This population
Key inclusion criteria (3 listed)
- Enrolled in the original JOS study, "A Randomized Prospective Study for the Prevention of Joint Disease in Children with Factor VIII Deficiency"
- Written, informed consent of parent or guardian for the proposed study
- The local hemophilia treatment center staff must evaluate the family's participation in the original treatment protocol and determine that the family is capable of complying with the continuation protocol
Key exclusion criteria (1 listed)
- Unable or unwilling to record the study information
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Determine the natuaral history of joint development in hemophilia and the impact of primary or secondary prophylaxis on the prevention, limitation, or reversal of hemophilic arthropathy.
Time frame: Enrollment, age 14, and study exit at age 18
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Oct 2019.
- Derived from this study: PubMed 32492157Warren BB, Thornhill D, Stein J, Fadell M, Ingram JD, Funk S, Norton KL, Lane HD, Bennett CM, Dunn A, Recht M, Shapiro A, Manco-Johnson MJ. Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study. Blood Adv. 2020 Jun 9;4(11):2451-2459. doi: 10.1182/bloodadvances.2019001311.
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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.
Marilyn Manco-Johnson, MD principal investigator
University of Colorado at Denver Health and Sciences Center
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by University of Colorado, Denver.
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 3 inclusion and 1 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 31 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Determine the natuaral history of joint development in hemophilia and the impact of primary or secondary prophylaxis on the prevention, limitation, or reversal of hemophilic arthropathy. (time frame: Enrollment, age 14, and study exit at age 18). 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 NCT01000844, which also lists 1 linked publication.
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 31 Oct 2019.