NF1NF107-BMP2NCT02718131
A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1)
A study testing INFUSE Bone Graft (BMP-2) in people with NF1 and Congenital Pseudarthrosis of Tibia, run by University of Alabama at Birmingham at 16 sites in 2 countries.
Trial summary
The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA (tibial pseudarthrosis) repair in children with NF1, compared to a control group of patients treated surgically without BMP-2. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- NF1, Congenital Pseudarthrosis of Tibia
- What is being tested?
- INFUSE Bone Graft (BMP-2)
- Type of treatment
- Medical device, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 1 Mar 2016to 1 Jun 2020
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of NF1 using the NIH Consensus Conference criteria. In addition to tibial pseudarthrosis, one or more of the following diagnostic criteria for NF1 must be present:
- Six or more cafe'-au-lait spots (≥ 0.5cm prepubertal; ≥ 1.5cm postpubertal)
- Freckling in the axilla or groin Optic pathway glioma
- Two or more iris Lisch nodules
- Two or more neurofibromas or one plexiform neurofibroma
- A first-degree relative with NF1
Show all 31Show fewer
- Patients must have tibial pseudarthrosis that has the potential to cause significant morbidity. Radiographic findings (AP \& lateral leg radiographs) must support the diagnosis of tibial pseudarthrosis with chronic non-union.
- Age between 2 years and 18 years of age at time of study entry.
- Performance Level: Karnofsky ≥ 50 percent for patients > 10 years of age and Lansky ≥ 50 percent for patients or ≤ 10 years of age.
- Prior Therapy:
- Patients who have undergone 1 previous surgery for tibial pseudarthrosis repair will be eligible to enter the study if they have refracture.
- Use of BMP-2 in the prior surgery is permitted, however patients with prior exposure must be screened for antibodies to BMP-2, bovine collagen, and rhBMP-2 neutralizing antibodies.
- Prior use of BMP-2 is allowed but will be recorded as a possible compounding factor.
- Patients who have had 2 or more prior surgeries for pseudarthrosis repair are ineligible
- Absence of Tumors:
- Patients must undergo thorough physical examination of the leg undergoing surgery. If physical exam is equivocal for presence of tumors, then a normal MRI of the lower extremity will be required before eligibility is met.
- If there is evidence of plexiform neurofibroma or nodular neurofibroma of > 3 cm diameter on the ipsilateral leg, then they are ineligible for the study.
- Organ Function Requirements
- Adequate bone marrow function defined as:
- Absolute neutrophil count (ANC) > 1500/
- µl Platelet count > 100,000/
- µl Hemoglobin ≥ 10.0 gm/dL
- Adequate renal function defined as:
- maximum serum creatinine of 1.5 mg/dL OR
- a creatinine clearance≥70ml/min/1.73m2.
- Adequate renal function defined as:
- maximum serum creatinine of 1.5 mg/dL OR
- a creatinine clearance ≥ 70ml/min/1.73m2.
- Adequate liver function defined as:
- Total bilirubin < 1.5 X upper limit of normal for age, and SGPT (serum glutamic pyruvic transaminase, ALT) < 2 X upper limit of normal for age
- Serum Vitamin D level ≥ 10 ng/ml
Who cannot join
- Lack of documentation for a diagnosis of NF1
- Tibial fracture without evidence of pseudarthrosis or tibial dysplasia
- Tibial dysplasia/bowing without fracture or pseudarthrosis
- Plexiform neurofibroma of any size, or nodular neurofibroma of > 3 cm diameter involving the ipsilateral leg, including the hip
- If presence of plexiform is suspected but not certain on physical exam, MRI of the leg may be indicated to rule this out.
- History of MPNST (malignant peripheral nerve sheath tumor) or any malignancy other than asymptomatic and stable optic nerve glioma
Show all 28Show fewer
- Optic nerve glioma that has resulted in precocious puberty or visual impairment of any degree
- Visual impairment from any cause
- Precocious puberty from any cause
- Hypertension other than mild essential hypertension controlled with medication
- Metastatic disease of any kind
- Inadequate neurovascular status in the involved limb that may jeopardize healing
- Active or known prior infection at the pseudarthrosis site
- Active systemic infection
- Other injury or condition that prevents ambulation or completion of study assessments
- Two or more prior surgeries for tibial pseudarthrosis
- Bilateral tibial dysplasia
- Selection of a surgical approach that does not include prescribed surgical intervention, which must include removal of pseudarthrosis tissue, placement of an intramedullary rod using the Williams approach, and autogenous bone graft from the iliac crest distributed at the osteotomy site
- Normal ipsilateral fibula without planned fibular osteotomy at time of surgery
- Allergy to bone morphogenetic protein
- Allergy to bovine collagen products
- Positive antibody titers to BMP-2, bovine collagen, or BMP-2 neutralizing antibodies prior to surgery
- History of using any of the following medications, regardless of dose, for at least 1 month, within 3 months of enrollment: Anabolic agents, Glucocorticoids (does not include inhaled glucocorticoids), Growth hormone, Parathyroid hormone (PTH)
- Need for postoperative medications that could interfere with bone healing of the implant, such as steroids, (but not including low-dose aspirin or routine perioperative anti-inflammatory drugs)
- Untreated endocrine abnormality, such as hypothyroidism, parathyroid hormone disorder
- Severe Vitamin D deficiency with serum 25-OH (hydroxy) Vitamin D < 10 ng/ml (25 nmol/l) Patients with Vitamin D levels < 10 ng/ml may be treated with Vitamin D and reconsidered for enrollment when levels are sufficient
- Females who are sexually active without use of effective contraception
- Females who are pregnant or breastfeeding
Trial locations and contacts
16 sites in 2 countries.
16 sites in 2 countries
United States15 sites
Johns Hopkins
Baltimore, Maryland, 21287
The University of Alabama at Birmingham
Birmingham, Alabama, 35294
Children' Hospital Boston and Massachusetts General Hospital
Boston, Massachusetts, 02115
Children' Hospital Boston
Boston, Massachusetts, 02115
Children's Lurie Hospital
Chicago, Illinois, 60611
University of Chicago
Chicago, Illinois, 60637
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Texas Scottish Rite Hospital for Children
Dallas, Texas, 75235
Indiana Unversity
Indianapolis, Indiana, 46202
Children's Hospital Los Angeles
Los Angeles, California, 90027
New York University Medical Center
New York, New York, 10016
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19096
University of Utah
Salt Lake City, Utah, 84132
Washington University in St. Louis
St Louis, Missouri, 63130
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Australia1 site
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
About this study
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)
The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA (tibial pseudarthrosis) repair in children with NF1, compared to a control group of patients treated surgically without BMP-2. The following Specific Aims will be addressed: 1) to determine if use of an osteogenic agent (BMP-2) at the time of surgical repair of TPA in NF1 patients will result in improved bone healing; 2) to document safety of BMP-2 in a pediatric NF1 population; and 3) to collect, process, and preserve biologic specimens at the time of surgery for future studies.
Read the detailed description
A randomized study will be performed by a multi-center group of the NF Consortium. A total of 54 patients will be randomized for treatment with or without INFUSE Bone Graft at the time of surgical repair. For all patients, a standard surgical procedure will be used, including: resection of pseudarthrosis tissue; placement of a rigid intramedullary rod; and placement of autogenous bone graft from iliac crest. For patients in the BMP group, the INFUSE device containing BMP-2 will in addition be applied intraoperatively to the osteotomy site. Fracture union will be determined by scoring of radiographs (RUST score) for cortical bone fusion and callus formation at the osteotomy site. RUST score at 12 months post-surgery will be the primary outcome measure to determine efficacy. Secondary measures will include determination of time to healing (months); quality of life measures; functional walking measures; and incidence of refracture after surgery. This study, once successfully completed, will determine if use of INFUSE Bone Graft improves healing of tibial pseudarthrosis in NF1 and will document safety issues. Regardless of results, the better performing of the two groups (control or BMP) will be able to serve as a much-needed control arm for future studies of additional targeted therapeutic agents for NF1-related bone disease. An international working group of orthopaedic surgeons and NF specialists has been formed and is committed to successful completion of this trial.
- Conditions
- NF1, Congenital Pseudarthrosis of Tibia
- Being tested
- INFUSE Bone Graft (BMP-2), Control Group
- Keywords
- Tibial Pseudarthrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INFUSE Bone Graft (BMP-2)Device
The INFUSE bone graft, containing BMP-2 on a collagen sponge, will be wrapped around the tibia during the surgical process.
Control GroupProcedure or surgery
The control group will receive the standard surgical protocol, without addition of the INFUSE device.
| Study group | Role | What they receive |
|---|---|---|
| INFUSE Bone Graft (BMP-2) | Active comparator | 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. Receives: Device: INFUSE Bone Graft (BMP-2) |
| Control Group | Placebo comparator | 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. Receives: Procedure: Control Group |
What is being measured
The primary outcome is the main question the study is designed to answer.
RUST Score
The RUST scale (Radiographic Union Score for Tibial fractures; RUST) was developed in 2010 as a measure of tibial fracture healing and has been validated in the NF1 population. This was used as the primary outcome measure in this trial. The score is based on radiographic evaluation of the bone cortex based on AP (anterior-posterior) and lateral xrays. Four cortices of tibial bone seen in these views are each given a score of 1 to 3, with 1 representing no healing, 2 representing some callus present, and 3 representing healing of the bone cortex. The 4 scores are totaled, to give a total score range of 4 to 12. For the purpose of this study, a total RUST score of 9 to 12, with at least 2 evaluable cortices having a score of 3, is considered to be healed. Scores of 5 to 8 are considered to represent partial healing, and a score of 4 is considered to be no healing.
Measured: 12 mos post surgery. The score at 12 mos post surgery will be used to determine success of healing and is the score reported.
Secondary outcomes (11)
Average Time to Healing in Months
Time to healing is determined by RUST scores calculated at 3 month intervals after surgery. The first time point at which a score of 9 to 12 is achieved on the RUST Scale will be considered the time to complete healing. One time point is reported which is the time point of first healing.
Measured: Baseline through 12 months post-surgery.
Refracture Rate
Numbers of patients with refracture within 1 year after surgery.
Measured: 12 months post-surgery
Long-term Refracture
Numbers of patients with refracture over long-term follow up of up to 10 years.
Measured: annually, up to 10 years post-surgery
Ten Meter Timed Walk
Time (seconds) to perform the Ten Meter Timed Walk. Assessed at 6 and 12 months after surgery. Both 6 month and 12 month reported.
Measured: Assessed at 6 and 12 months after surgery. Month 12 reported.
Pain Intensity
Pain Intensity was measured using the Faces Pain Scale-Revised (FPS-R) for patients aged 4 years and older. This is a self-report pain scale, where children chose one of 6 faces to indicate their current pain. Scores range from 0 to 10, with 0 representing no pain (happy face) and 10 representing the most pain.
Measured: measured at post-op visits at 6 weeks, 3 mos, 6 mos, and 12 mos after surgery
Quality of Life Score - The Upper Extremity and Physical Function Core Scale
Quality of Life (QOL) was measured during the study using the Pediatric Outcome Data Collection Instrument (PODCI), a validated scale developed by the American Academy of Orthopaedic Surgeons to measure outcomes in orthopedic interventions. The Parent-report form, which was used for this study, consists of 86 Likert-format questions, covering 5 domains (Upper extremity and physical function; transfer and basic mobility; sports/physical functioning; pain/comfort; and happiness scales). In addition, a Global Functioning Scale is computed. Scores for each subscale range from 0 to 100, with lower scores representing better quality of life.
Measured: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Transfer and Basic Mobility Core Scale
Quality of Life (QOL) was measured during the study using the Pediatric Outcome Data Collection Instrument (PODCI), a validated scale developed by the American Academy of Orthopaedic Surgeons to measure outcomes in orthopedic interventions. The Parent-report form, which was used for this study, consists of 86 Likert-format questions, covering 5 domains (Upper extremity and physical function; transfer and basic mobility; sports/physical functioning; pain/comfort; and happiness scales). In addition, a Global Functioning Scale is computed. Scores for each subscale range from 0 to 100, with lower scores representing better quality of life.
Measured: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Sports and Physical Functioning Core Scale
Quality of Life (QOL) was measured during the study using the Pediatric Outcome Data Collection Instrument (PODCI), a validated scale developed by the American Academy of Orthopaedic Surgeons to measure outcomes in orthopedic interventions. The Parent-report form, which was used for this study, consists of 86 Likert-format questions, covering 5 domains (Upper extremity and physical function; transfer and basic mobility; sports/physical functioning; pain/comfort; and happiness scales). In addition, a Global Functioning Scale is computed. Scores for each subscale range from 0 to 100, with lower scores representing better quality of life.
Measured: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Pain/Comfort Core Scale
Quality of Life (QOL) was measured during the study using the Pediatric Outcome Data Collection Instrument (PODCI), a validated scale developed by the American Academy of Orthopaedic Surgeons to measure outcomes in orthopedic interventions. The Parent-report form, which was used for this study, consists of 86 Likert-format questions, covering 5 domains (Upper extremity and physical function; transfer and basic mobility; sports/physical functioning; pain/comfort; and happiness scales). In addition, a Global Functioning Scale is computed. Scores for each subscale range from 0 to 100, with lower scores representing better quality of life.
Measured: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Happiness Core Scale
Quality of Life (QOL) was measured during the study using the Pediatric Outcome Data Collection Instrument (PODCI), a validated scale developed by the American Academy of Orthopaedic Surgeons to measure outcomes in orthopedic interventions. The Parent-report form, which was used for this study, consists of 86 Likert-format questions, covering 5 domains (Upper extremity and physical function; transfer and basic mobility; sports/physical functioning; pain/comfort; and happiness scales). In addition, a Global Functioning Scale is computed. Scores for each subscale range from 0 to 100, with lower scores representing better quality of life.
Measured: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Global Functioning Scale
Quality of Life (QOL) was measured during the study using the Pediatric Outcome Data Collection Instrument (PODCI), a validated scale developed by the American Academy of Orthopaedic Surgeons to measure outcomes in orthopedic interventions. The Parent-report form, which was used for this study, consists of 86 Likert-format questions, covering 5 domains (Upper extremity and physical function; transfer and basic mobility; sports/physical functioning; pain/comfort; and happiness scales). In addition, a Global Functioning Scale is computed. Scores for each subscale range from 0 to 100, with lower scores representing better quality of life.
Measured: Assessed at 6 and 12 months after surgery. Month 12 reported.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2016 (estimated)
- Study start1 Mar 2016
- Primary completion1 Jun 2020
- Study completion1 Jun 2020
Why it stopped: Insufficient enrollment to meet the statistical requirements
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for NF1Studies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Collaborators
- The Children's Tumor Foundation, Medtronic
- Responsible party
- Principal investigator, Bruce Korf, MD, University of Alabama at Birmingham
- Investigators
- Bruce R. Korf, MD, PhD, Study chair, Univ. of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02718131
- Sponsor's protocol ID
- NF107
- Other IDs
- W81XWH-12-1-0155, G140004, 507821
- Acronym
- NF107-BMP2
- Status last verified
- Sep 2021
- First submitted
- 7 Mar 2016
- Last updated
- 27 Oct 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02718131
- Acronym / sponsor ID
- NF107-BMP2 / NF107
- Conditions
- NF1, Congenital Pseudarthrosis of Tibia
- Keywords
- Tibial Pseudarthrosis
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Insufficient enrollment to meet the statistical requirements
- Enrolment
- 5 participants (actual)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Collaborators
- The Children's Tumor Foundation, Medtronic
- Study start
- 1 Mar 2016 (actual)
- Primary completion
- 1 Jun 2020 (actual)
- Study completion
- 1 Jun 2020 (actual)
- First posted
- 24 Mar 2016 (estimated)
- Results
- Posted 27 Oct 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA (tibial pseudarthrosis) repair in children with NF1, compared to a control group of patients treated surgically without BMP-2. The following Specific Aims will be addressed: 1) to determine if use of an osteogenic agent (BMP-2) at the time of surgical repair of TPA in NF1 patients will result in improved bone healing; 2) to document safety of BMP-2 in a pediatric NF1 population; and 3) to collect, process, and preserve biologic specimens at the time of surgery for future studies.
Detailed description
A randomized study will be performed by a multi-center group of the NF Consortium. A total of 54 patients will be randomized for treatment with or without INFUSE Bone Graft at the time of surgical repair. For all patients, a standard surgical procedure will be used, including: resection of pseudarthrosis tissue; placement of a rigid intramedullary rod; and placement of autogenous bone graft from iliac crest. For patients in the BMP group, the INFUSE device containing BMP-2 will in addition be applied intraoperatively to the osteotomy site. Fracture union will be determined by scoring of radiographs (RUST score) for cortical bone fusion and callus formation at the osteotomy site. RUST score at 12 months post-surgery will be the primary outcome measure to determine efficacy. Secondary measures will include determination of time to healing (months); quality of life measures; functional walking measures; and incidence of refracture after surgery. This study, once successfully completed, will determine if use of INFUSE Bone Graft improves healing of tibial pseudarthrosis in NF1 and will document safety issues. Regardless of results, the better performing of the two groups (control or BMP) will be able to serve as a much-needed control arm for future studies of additional targeted therapeutic agents for NF1-related bone disease. An international working group of orthopaedic surgeons and NF specialists has been formed and is committed to successful completion of this trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| INFUSE Bone Graft (BMP-2) | Active comparator | Device: 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. |
| Control Group | Placebo comparator | Procedure: Control GroupChildren 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. |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (31 listed)
- Diagnosis of NF1 using the NIH Consensus Conference criteria. In addition to tibial pseudarthrosis, one or more of the following diagnostic criteria for NF1 must be present:
- Six or more cafe'-au-lait spots (≥ 0.5cm prepubertal; ≥ 1.5cm postpubertal)
- Freckling in the axilla or groin Optic pathway glioma
- Two or more iris Lisch nodules
- Two or more neurofibromas or one plexiform neurofibroma
Key exclusion criteria (28 listed)
- Lack of documentation for a diagnosis of NF1
- Tibial fracture without evidence of pseudarthrosis or tibial dysplasia
- Tibial dysplasia/bowing without fracture or pseudarthrosis
- Plexiform neurofibroma of any size, or nodular neurofibroma of > 3 cm diameter involving the ipsilateral leg, including the hip
- If presence of plexiform is suspected but not certain on physical exam, MRI of the leg may be indicated to rule this out.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Device | INFUSE Bone Graft (BMP-2) |
| Control GroupThe control group will receive the standard surgical protocol, without addition of the INFUSE device. | Procedure or surgery | Control Group |
Primary outcome
RUST Score
Time frame: 12 mos post surgery. The score at 12 mos post surgery will be used to determine success of healing and is the score reported.
The RUST scale (Radiographic Union Score for Tibial fractures; RUST) was developed in 2010 as a measure of tibial fracture healing and has been validated in the NF1 population. This was used as the primary outcome measure in this trial. The score is based on radiographic evaluation of the bone cortex based on AP (anterior-posterior) and lateral xrays. Four cortices of tibial bone seen in these views are each given a score of 1 to 3, with 1 representing no healing, 2 representing some callus present, and 3 representing healing of the bone cortex. The 4 scores are totaled, to give a total score range of 4 to 12. For the purpose of this study, a total RUST score of 9 to 12, with at least 2 evaluable cortices having a score of 3, is considered to be healed. Scores of 5 to 8 are considered to represent partial healing, and a score of 4 is considered to be no healing.
11 secondary outcomes
Average Time to Healing in Months
Time frame: Baseline through 12 months post-surgery.
Refracture Rate
Time frame: 12 months post-surgery
Long-term Refracture
Time frame: annually, up to 10 years post-surgery
Ten Meter Timed Walk
Time frame: Assessed at 6 and 12 months after surgery. Month 12 reported.
Pain Intensity
Time frame: measured at post-op visits at 6 weeks, 3 mos, 6 mos, and 12 mos after surgery
Quality of Life Score - The Upper Extremity and Physical Function Core Scale
Time frame: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Transfer and Basic Mobility Core Scale
Time frame: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Sports and Physical Functioning Core Scale
Time frame: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Pain/Comfort Core Scale
Time frame: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Happiness Core Scale
Time frame: Assessed at 6 and 12 months after surgery. Month 12 reported.
Quality of Life Score - The Global Functioning Scale
Time frame: Assessed at 6 and 12 months after surgery. Month 12 reported.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Bruce R. Korf, MD, PhD study chair
Univ. of Alabama at Birmingham
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing INFUSE Bone Graft (BMP-2) in people with NF1 and Congenital Pseudarthrosis of Tibia, run by University of Alabama at Birmingham at 16 sites in 2 countries.
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 31 inclusion and 28 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: stopped early, as of the sponsor's last update on 27 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 27 Oct 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
RUST Score (time frame: 12 mos post surgery. The score at 12 mos post surgery will be used to determine success of healing and is the score reported.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Oct 2021.