Pediatric Congenital Heart DiseasePhase 4NCT02752191
Feraheme As An MRI Contrast Agent For Pediatric Congenital Heart Disease
A Phase 4 study testing ferumoxytol and gadofosveset in people with Pediatric Congenital Heart Disease, run by Paul Finn at 1 site in United States.
Trial summary
The standard clinical cardiovascular MRI practice for children with CHD frequently involves the use of gadolinium-based contrast agents (GBCA) to enhance tissue contrast. Most GBCAs are small molecules that quickly cross the capillary wall and access the interstitial space, a process which diminishes the signal… Read more about this study
- Who is it for?
- Ages 1 day to 6 years
- Conditions the study is for
- Pediatric Congenital Heart Disease
- What is being tested?
- ferumoxytol and gadofosveset
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2016to 19 Dec 2018
- Phase
- Phase 4
- 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 1 day to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female pediatric patients of all ethnicities (age newborn to 6 years) with known or suspected CHD with inconclusive echocardiographic exams and are referred for cardiovascular MRI for further evaluation of cardiac anatomy and function.
- Written informed consent obtained from subject's legal representative/guardian(s) and ability for subject to comply with the requirements of the study
Who cannot join
- Standard clinical contraindications to MRI, including subjects with cochlear implants and implanted cardiac devices
- Subjects with past or current diagnosis of iron overload due to hereditary hemochromatosis or other causes (for subjects receiving Feraheme injection only).
- Subjects with known hypersensitivity or allergy to iron oxide particles.
- Subjects with renal insufficiency defined as estimated glomerular filtration rate (eGFR) < 40 mL/min/1.73m2 (for subjects receiving Ablavar injection only).
- Subjects who are critically ill at the time of MRI and for whom the period of general anesthesia and separation from the critical care nursery or intensive care unit poses added risk as deemed by referring cardiologists, cardiac surgeons or the managing radiologist (for Part II only).
- Other medical conditions, in the judgment of the clinician investigator, that would increase the risks to the child related to participation in the study.
Trial locations and contacts
1 site in 1 country.
About this study
The standard clinical cardiovascular MRI practice for children with CHD frequently involves the use of gadolinium-based contrast agents (GBCA) to enhance tissue contrast. Most GBCAs are small molecules that quickly cross the capillary wall and access the interstitial space, a process which diminishes the signal contrast between blood vessels and surrounding tissue. Therefore, these types of GBCA are most useful for first-pass MR angiography, wherein the images are acquired quickly during the initial 15-30 seconds post-injection when the GBCA concentration is much higher in the arteries than in the interstitial space. For young children with complex CHD, the stringent requirements for high spatial resolution, and the need for cardiac gating and good blood-myocardium contrast in order to provide detailed evaluation of intracardiac structures are not compatible with conventional GBCA-based first-pass MR angiography. Even with Ablavar® (gadofosveset trisodium), an FDA approved GBCA with longer intravascular half-life than other GBCAs, cardiac-gated Ablavar®-enhanced MRI may be insufficient for young children with CHD based on our institutional experience and on data from the literature; there remains diminished blood-tissue contrast during the high-resolution cardiac-gated MRI. Furthermore, there have been safety concerns regarding gadolinium deposition in brain tissues after repeated GBCA exposure as well as concerns of nephrogenic systemic fibrosis (NSF) associated with GBCA injection in young children < 2 years old who may have immature renal function. The long-term health consequences of these effects in the pediatric population are unclear. For the above reasons, we seek to study the diagnostic imaging effectiveness of Feraheme (Feraheme®), an FDA-approved drug for parenteral iron supplementation, as an MRI contrast agent in children with CHD. Although Feraheme® has been approved for the treatment of iron deficiency anemia secondary to renal disease, Feraheme® has been used as an off-label MRI contrast agent at select medical centers.
- Conditions
- Pediatric Congenital Heart Disease
- Being tested
- ferumoxytol, gadofosveset
- Keywords
- ferumoxytol
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ferumoxytolDrug
Also called Feraheme
ferumoxytol as an MRI contrast agent infused over several minutes
gadofosvesetDrug
Also called Ablavar
gadofosveset as an MRI contrast agent injected over several seconds
| Study group | Role | What they receive |
|---|---|---|
| Ferumoxytol | Active comparator | Ferumoxytol, 4mg/kg of body weight, one time infusion of several minutes Receives: Drug: ferumoxytol |
| gadofosveset | Active comparator | gadofosveset, 0.03mmol/kg, one time bolus injection Receives: Drug: gadofosveset |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite Image Quality Score Among 7 Anatomical Structures.
1-4 image quality Likert score by an imaging expert, with 4 representing excellent image quality and 1 is non-diagnostic
Measured: day 1
Secondary outcomes (2)
Image Quality Score at Individual Anatomical Sites.
1-4 image quality Likert score by an imaging expert, with 4 representing excellent image quality and 1 is non-diagnostic.
- image quality score at the aortic root.
- image quality score at the main pulmonary artery.
- image quality score at the coronary arteries.
- image quality score a the out-flow tracts.
- image quality score at the valves.
- image quality score at the ventricular chambers.
- image quality at the atria.
Measured: day 1
Incidence of Adverse Events
Number of participants with one or more adverse events
Measured: day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2016 (estimated)
- Study startApr 2016
- Primary completion19 Dec 2018
- Study completion19 Dec 2018
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 Pediatric Congenital Heart DiseaseStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Paul Finn
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Paul Finn, University of California, Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02752191
- Sponsor's protocol ID
- 16-00016
- Status last verified
- Nov 2022
- First submitted
- 15 Apr 2016
- Last updated
- 28 Dec 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feraheme As An MRI Contrast Agent For Pediatric Congenital Heart Disease
- ClinicalTrials.gov ID
- NCT02752191
- Acronym / sponsor ID
- 16-00016
- Conditions
- Pediatric Congenital Heart Disease
- Keywords
- ferumoxytol
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Paul FinnUniversity, hospital or other
- Study start
- Apr 2016 (actual)
- Primary completion
- 19 Dec 2018 (actual)
- Study completion
- 19 Dec 2018 (actual)
- First posted
- 26 Apr 2016 (estimated)
- Results
- Posted 28 Dec 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The standard clinical cardiovascular MRI practice for children with CHD frequently involves the use of gadolinium-based contrast agents (GBCA) to enhance tissue contrast. Most GBCAs are small molecules that quickly cross the capillary wall and access the interstitial space, a process which diminishes the signal contrast between blood vessels and surrounding tissue. Therefore, these types of GBCA are most useful for first-pass MR angiography, wherein the images are acquired quickly during the initial 15-30 seconds post-injection when the GBCA concentration is much higher in the arteries than in the interstitial space. For young children with complex CHD, the stringent requirements for high spatial resolution, and the need for cardiac gating and good blood-myocardium contrast in order to provide detailed evaluation of intracardiac structures are not compatible with conventional GBCA-based first-pass MR angiography. Even with Ablavar® (gadofosveset trisodium), an FDA approved GBCA with longer intravascular half-life than other GBCAs, cardiac-gated Ablavar®-enhanced MRI may be insufficient for young children with CHD based on our institutional experience and on data from the literature; there remains diminished blood-tissue contrast during the high-resolution cardiac-gated MRI. Furthermore, there have been safety concerns regarding gadolinium deposition in brain tissues after repeated GBCA exposure as well as concerns of nephrogenic systemic fibrosis (NSF) associated with GBCA injection in young children < 2 years old who may have immature renal function. The long-term health consequences of these effects in the pediatric population are unclear. For the above reasons, we seek to study the diagnostic imaging effectiveness of Feraheme (Feraheme®), an FDA-approved drug for parenteral iron supplementation, as an MRI contrast agent in children with CHD. Although Feraheme® has been approved for the treatment of iron deficiency anemia secondary to renal disease, Feraheme® has been used as an off-label MRI contrast agent at select medical centers.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ferumoxytol | Active comparator | Drug: ferumoxytolFerumoxytol, 4mg/kg of body weight, one time infusion of several minutes |
| gadofosveset | Active comparator | Drug: gadofosvesetgadofosveset, 0.03mmol/kg, one time bolus injection |
Who may qualify
- Ages
- 1 day to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Male or female pediatric patients of all ethnicities (age newborn to 6 years) with known or suspected CHD with inconclusive echocardiographic exams and are referred for cardiovascular MRI for further evaluation of cardiac anatomy and function.
- Written informed consent obtained from subject's legal representative/guardian(s) and ability for subject to comply with the requirements of the study
Key exclusion criteria (6 listed)
- Standard clinical contraindications to MRI, including subjects with cochlear implants and implanted cardiac devices
- Subjects with past or current diagnosis of iron overload due to hereditary hemochromatosis or other causes (for subjects receiving Feraheme injection only).
- Subjects with known hypersensitivity or allergy to iron oxide particles.
- Subjects with renal insufficiency defined as estimated glomerular filtration rate (eGFR) < 40 mL/min/1.73m2 (for subjects receiving Ablavar injection only).
- Subjects who are critically ill at the time of MRI and for whom the period of general anesthesia and separation from the critical care nursery or intensive care unit poses added risk as deemed by referring cardiologists, cardiac surgeons or the managing radiologist (for Part II only).
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 |
|---|---|---|
| ferumoxytolAlso: Ferahemeferumoxytol as an MRI contrast agent infused over several minutes | Drug | Ferumoxytol |
| gadofosvesetAlso: Ablavargadofosveset as an MRI contrast agent injected over several seconds | Drug | gadofosveset |
Primary outcome
Composite Image Quality Score Among 7 Anatomical Structures.
Time frame: day 1
1-4 image quality Likert score by an imaging expert, with 4 representing excellent image quality and 1 is non-diagnostic
2 secondary outcomes
Image Quality Score at Individual Anatomical Sites.
Time frame: day 1
Incidence of Adverse Events
Time frame: day 1
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Dec 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing ferumoxytol and gadofosveset in people with Pediatric Congenital Heart Disease, run by Paul Finn at 1 site in United States.
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 2 inclusion and 6 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 28 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Dec 2022). 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?
Composite Image Quality Score Among 7 Anatomical Structures. (time frame: day 1). 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 28 Dec 2022.