Legg-Calve Perthes Disease
Conditions
Brief summary
Legg-Calve-Perthes disease (LCP) represents the loss of blood flow to the head of the femur, resulting in significant hip pain and potential long term disability. This study represents a single center, randomized, prospective, controlled study comparing the treatment of LCP with an intraarticular corticosteroid (triamcinolone hexacetonide) injection with traditional nonoperative treatment.
Detailed description
Legg-Calve-Perthes disease (LCP) represents the loss of blood flow to the head of the femur, resulting in significant hip pain and potential long term disability. The cause of LCP is still unknown, but tends to occur predominantly in boys from ages 4-12. For the majority of those affected, observation and symptomatic treatment with oral antiinflammatories, such as ibuprofen, is indicated. This study represents a single center, randomized, prospective, controlled study comparing the treatment of LCP with an intraarticular corticosteroid (triamcinolone hexacetonide) injection with traditional nonoperative treatment. The investigators will test for improved outcomes by measuring functional outcomes (PODCI and ASKp questionnaires, StepWatch activity monitor), hip range of motion and visual-analog pain scales. The investigators hypothesize that injections of corticosteroids (potent, injectable antiinflammatories) will result in improved overall function through decreased pain and increased hip range of motion in this patient population.Additional biological research will be performed. There is no human information on the inflammatory response that occurs in the hip joint of children with Perthes disease, and there are no true animal models of Perthes disease. To better understand the pathobiology of Perthes disease, the collection of joint fluid from both hips may provide insight into the treatment of a disease for which we currently have no explanation of cause, and consequently no therapies
Interventions
DRUGAristospan 20mg
Triamcinolone hexacetonide injectable suspension, USP, 20mg/mL Parenteral. One single dose.
Sponsors
Seattle Children's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
4 Years to 13 Years
Healthy volunteers
No
Inclusion criteria
* age of 4 to 12 years * diagnosed with Idiopathic osteonecrosis of the femoral head * symptoms less than 12 months old.
Exclusion criteria
* Symptoms for more than 12 months * Previous treatment other than anti-inflammatories, crutches or bed rest * Subjects more than 8 years old or have a hand bone age greater than 6 years old and lateral pillar B or B/C disease. * Bilateral hip disease * Personal or family history of problems with general anesthesia * Prior steroid treatment * Previous diagnosis of: * Asthma * Identifiable rheumatologic condition * Metabolic Diseases (including but not limited to Gaucher's disease or congenital hypothyroidism) * Sickle cell disease * Known pain syndrome * Hip sepsis * Prior malignancy (solid organ or bone marrow transplant) * Blood Clotting disorder
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Primary Outcome Variable: Function. The Pediatric Outcomes Data Collection Instrument (PODCI) Will be the Primary Endpoint as a Measure of Function and Health Related Quality of Life at 12 Months Post Injection. | This exam is to be administered at time of enrollment and at 4 and 12 months follow up visits. | The Pediatric Outcomes Data Collection Instrument (PODCI) is designed to be completed by the parent/guardian of a child ten years of age or younger who has knowledge of the child's conditions. The eight scales generated from these instruments are: Upper Extremity and Physical Function Scale; Transfer and Basic Mobility Scale; Sports/Physical Functioning Scale; Pain/Comfort; Treatment Expectations Scale; Happiness Scale; Satisfaction with Symptoms Scale and Global Functioning Scale. The results of each scale are standardized into a scale of 0-100 where 0 indicates the worst outcome and 100 the best. We decided to report Global Functioning only due to space limitations. Also the Global Functioning scale encompasses items from other scales. |
Secondary
| Measure | Time frame |
|---|---|
| Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | StepWatch monitor will be used 7 days prior to treatment and 4 weeks, 4 months and 12 months follow up visits |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Injection Aristospan 20mg: Triamcinolone hexacetonide injectable suspension, USP, 20mg/mL Parenteral. One single dose. | 1 |
| Control observation | 2 |
| Total | 3 |
Baseline characteristics
| Characteristic | Injection | Control | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 1 Participants | 2 Participants | 3 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 9 years STANDARD_DEVIATION 0 | 7.33 years STANDARD_DEVIATION 2.08 | 7.33 years STANDARD_DEVIATION 2.08 |
| Region of Enrollment United States | 1 participants | 2 participants | 3 participants |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 0 Participants | 2 Participants | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 1 | 0 / 2 |
| serious Total, serious adverse events | 0 / 1 | 0 / 2 |
Outcome results
Primary
Primary Outcome Variable: Function. The Pediatric Outcomes Data Collection Instrument (PODCI) Will be the Primary Endpoint as a Measure of Function and Health Related Quality of Life at 12 Months Post Injection.
The Pediatric Outcomes Data Collection Instrument (PODCI) is designed to be completed by the parent/guardian of a child ten years of age or younger who has knowledge of the child's conditions. The eight scales generated from these instruments are: Upper Extremity and Physical Function Scale; Transfer and Basic Mobility Scale; Sports/Physical Functioning Scale; Pain/Comfort; Treatment Expectations Scale; Happiness Scale; Satisfaction with Symptoms Scale and Global Functioning Scale. The results of each scale are standardized into a scale of 0-100 where 0 indicates the worst outcome and 100 the best. We decided to report Global Functioning only due to space limitations. Also the Global Functioning scale encompasses items from other scales.
Time frame: This exam is to be administered at time of enrollment and at 4 and 12 months follow up visits.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Injection | Primary Outcome Variable: Function. The Pediatric Outcomes Data Collection Instrument (PODCI) Will be the Primary Endpoint as a Measure of Function and Health Related Quality of Life at 12 Months Post Injection. | After Treatment (12 months) Global Functioning Sca | 97 units on a scale | — |
| Injection | Primary Outcome Variable: Function. The Pediatric Outcomes Data Collection Instrument (PODCI) Will be the Primary Endpoint as a Measure of Function and Health Related Quality of Life at 12 Months Post Injection. | Before treatment - Global Functioning Scale | 64 units on a scale | — |
| Control | Primary Outcome Variable: Function. The Pediatric Outcomes Data Collection Instrument (PODCI) Will be the Primary Endpoint as a Measure of Function and Health Related Quality of Life at 12 Months Post Injection. | Before treatment - Global Functioning Scale | 79 units on a scale | Standard Deviation 2.83 |
| Control | Primary Outcome Variable: Function. The Pediatric Outcomes Data Collection Instrument (PODCI) Will be the Primary Endpoint as a Measure of Function and Health Related Quality of Life at 12 Months Post Injection. | After Treatment (12 months) Global Functioning Sca | 97.5 units on a scale | Standard Deviation 3.53 |
Secondary
Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample.
Time frame: StepWatch monitor will be used 7 days prior to treatment and 4 weeks, 4 months and 12 months follow up visits
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Injection | Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | Baseline- Average Number of steps per day | 6287 steps/day | — |
| Injection | Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | 4 weeks Follow Up- Average Number of steps per d | 1782.19 steps/day | — |
| Injection | Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | 4 months follow up- average # of steps per day | 6985.89 steps/day | — |
| Injection | Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | 12 months follow up- average # of steps per day | 6990 steps/day | — |
| Control | Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | 12 months follow up- average # of steps per day | 2158.7 steps/day | Standard Deviation 865.9 |
| Control | Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | Baseline- Average Number of steps per day | 1448.04 steps/day | Standard Deviation 1873.78 |
| Control | Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | 4 months follow up- average # of steps per day | 2687.57 steps/day | Standard Deviation 1999.4 |
| Control | Second Outcome Variable: Ambulatory Activity. It Will be Defined as Average Steps/Day as Measured by the StepWatch Activity Monitor for a 7 Day Sample. | 4 weeks Follow Up- Average Number of steps per d | 2521.91 steps/day | Standard Deviation 1046.12 |