A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1) (NF107-BMP2)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02718131|
Recruitment Status : Active, not recruiting
First Posted : March 24, 2016
Last Update Posted : July 23, 2020
|Condition or disease||Intervention/treatment||Phase|
|NF1 Congenital Pseudarthrosis of Tibia||Device: INFUSE Bone Graft (BMP-2) Procedure: Control Group||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||54 participants|
|Intervention Model:||Parallel Assignment|
|Official Title:||A Study of INFUSE Bone Graft (Recombinant Human Bone Morphogenetic Protein-2/Absorbable Collagen Sponge) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis 1 (NF1)|
|Study Start Date :||March 2016|
|Estimated Primary Completion Date :||May 2021|
|Estimated Study Completion Date :||December 2021|
Active Comparator: INFUSE Bone Graft (BMP-2)
Children with NF1 and tibial pseudarthrosis who require surgery will have the INFUSE bone graft added to their surgical protocol. After a standard surgical approach of resection of abnormal pseudarthrotic tissue, placement of a rigid intramedullary rod (of the surgeon's choice) and placement of autogenous bone graft from the iliac crest; in addition, the INFUSE bone graft in the form of a collagen sponge will be wrapped around the tibia during the surgical process.
Device: INFUSE Bone Graft (BMP-2)
The INFUSE bone graft, containing BMP-2 on a collagen sponge, will be wrapped around the tibia during the surgical process.
Placebo Comparator: Control Group
Children with NF1 and tibial pseudarthrosis who require surgery will receive the standard surgical protocol only. This includes resection of abnormal pseudarthrotic tissue, placement of a rigid intramedullary rod (of the surgeon's choice) and placement of autogenous bone graft from the iliac crest.
Procedure: Control Group
The control group will receive the standard surgical protocol, without addition of the INFUSE device.
- RUST score [ Time Frame: 12 mos post surgery. The score at 12 mos post surgery will be used to determine success of healing ]Radiographic Union Score of Tibia (RUST) calculated from X-rays.
- Average time to healing in months [ Time Frame: X-rays obtained at 3, 6, 9, and 12 mos. post-surgery ]Time to healing is determined by RUST scores calculated at 3 month intervals after surgery. A score of 9/12 is considered to be complete healing.
- Refracture rate [ Time Frame: 12 months post-surgery ]Numbers of patients with refracture within 1 year after surgery.
- Long-term refracture [ Time Frame: annually, up to 10 years post-surgery ]Numbers of patients with refracture over long-term follow up of up to 10 years.
- Ten Meter Timed Walk [ Time Frame: 6 and 12 mos. after surgery. ]Time (seconds) to perform the Ten Meter Timed Walk.
- Pain intensity [ Time Frame: measured at post-op visits at 6 weeks, 3 mos, 6 mos, and 12 mos after surgery ]Pain intensity as assessed by the patient with the Faces Pain Scale-Revised (FPS-R).
- Quality of life score [ Time Frame: 6 and 12 mos. after surgery ]QOL measured by the Pediatric Outcome Data Collection Instrument (PODCI), filled out by patient and parent.
- Adverse Events [ Time Frame: measured at post-op visits at 6 weeks, 3 mos, 6 mos, and 12 mos after surgery ]Numbers of participants with treatment-related adverse events as assessed by CTCAE v4.0.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02718131
|United States, Alabama|
|The University of Alabama at Birmingham|
|Birmingham, Alabama, United States, 35294|
|United States, California|
|Children's Hospital Los Angeles|
|Los Angeles, California, United States, 90027|
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|United States, Illinois|
|Children's Lurie Hospital|
|Chicago, Illinois, United States, 60611|
|University of Chicago|
|Chicago, Illinois, United States, 60637|
|United States, Indiana|
|Indianapolis, Indiana, United States, 46202|
|United States, Maryland|
|Baltimore, Maryland, United States, 21287|
|United States, Massachusetts|
|Children' Hospital Boston and Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02115|
|Children' Hospital Boston|
|Boston, Massachusetts, United States, 02115|
|United States, Missouri|
|Washington University in St. Louis|
|Saint Louis, Missouri, United States, 63130|
|United States, New York|
|New York University Medical Center|
|New York, New York, United States, 10016|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19096|
|United States, Texas|
|Texas Scottish Rite Hospital for Children|
|Dallas, Texas, United States, 75235|
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84132|
|Australia, New South Wales|
|The Children's Hospital at Westmead|
|Westmead, New South Wales, Australia, 2145|
|Study Chair:||Bruce R. Korf, MD, PhD||Univ. of Alabama at Birmingham|