Crigler Najjar SyndromeDRAPNCT02356978
New Phototherapy Device to Treat Patients With Crigler-Najjar Disease
A study testing Arm1 homemade phototherapy treatment and DRAP in people with Crigler Najjar Syndrome and Children, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Evaluating the efficacity of a new device phototherapy by comparing it with conventional phototherapy. Jaundice occurs in many newborns, and is, in most cases benign, However, owing to the potential neurotoxicity of unconjugated bilirubin, newborns must be monitored to identify those who might develop severe… Read more about this study
- Who is it for?
- Ages Up to 6 years
- Conditions the study is for
- Crigler Najjar Syndrome, Children
- What is being tested?
- Arm1 homemade phototherapy treatment and DRAP
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteFrance
- Study dates
- Dec 2014to Dec 2017
- Phase
- Interventional
- 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 Up to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- genetic diagnosis of Crigler Najjar disease
- child whose height is between 60cm and 100cm
- patients with follow-up in reference center,
Who cannot join
- opposition of parents
- no social security insurance
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
AP-HP, Antoine Béclère Hospital
Clamart, 92141
VINCENT GAJDOS, Professor, +33 1 45 37 42 72, vincent.gajdos@abc.aphp.fr
About this study
Official title: Trial of a New Phototherapy Device for Treating Hyperbilirubinemia in Crigler-Najjar Patients : a New Concept
Evaluating the efficacity of a new device phototherapy by comparing it with conventional phototherapy. Jaundice occurs in many newborns, and is, in most cases benign, However, owing to the potential neurotoxicity of unconjugated bilirubin, newborns must be monitored to identify those who might develop severe hyperbilirubinemia an, in rare cases, acute bilirubin encephalopathy or kernicterus. Treatment of jaundice in newborn relies on phototherapy, exposing their skin to light of a specific wavelength . Fluorescent tubes or halogen lamps have been used as light sources for phototherapy for many years. Light-emitting diodes (LEDs) are more recent sources which are power efficient, have a longer life and are portable with low heat production. Several technologies and devices are developed using LEDs and specially a compact system.
Read the detailed description
Crigler-Najjar (CN) syndrome is a congenital inborn error of hepatic bilirubin metabolism caused by the deficiency of bilirubin uridinediphosphate glucuronosyltransferase activity CN patients are at permanent risk of life-threatening bilirubin encephalopathy (kernicterus). Treatment of CN disease relies on daily prolonged phototherapy (10-12 h/day). To date, liver transplantation is the only curative treatment available. To date, no phototherapy device exists for teenagers and adults. CN patients use homemade systems or add several neonatal systems together to reach a better efficacy.
A new device has been designed by weaving optical fibers into a sheet connected to LEDs. This new technology, marketed by several manufacturers of phototherapy devices, is used in pediatric wards for treating jaundiced newborns. A prototype of 80× 100cm was thus developed, which combines numerous advantages : important surface of treatment, easy use and transportation, user-friendliness.
Before going to a therapeutic trial for assessing the benefits of this new device in ambulatory current practice, it is essential to make sure of both its feasibility and efficiency on the control of bilirubinemia, under medical supervision.
Hypothesis: the new phototherapy sheet is as effective as, and better tolerated than the usual devices used in CN patients.
- Conditions
- Crigler Najjar Syndrome, Children
- Being tested
- Arm1 homemade phototherapy treatment, DRAP
- Keywords
- Rare disease, Crigler-Najjar disease, Paediatric, Experimental device
- Medical subject headings
- Crigler-Najjar Syndrome, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Arm1 homemade phototherapy treatmentDevice
session of 10 or 12 hours phototherapy treatment using the homemade device during the first night and then the "DRAP" device during the next nights.
DRAPDevice
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Each patient will be treated before using the active usual homemade device, and after using the experimental new "DRAP" device.
This new sheet was designed by weaving optical fibers connected to LEDs ( "BROCHIER" Technology). The "LIGHTEX" technology ® is a principle of weaving mill of optical fibres with side lighting connected to LEDs and allowing to realize flexible or stiff bright surfaces with very weak congestions, low consumption and high life cycle. The energy illumination of this device varies between 3 and 4 mW / cm ² ( average 3,6 mW / cm ².) Receives: Device: Arm1 homemade phototherapy treatment; Device: DRAP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Kinetic of blood Bilirubin level using the "DRAP" device (blood bilirubin concentration)
blood bilirubin concentration will be measured 2 times during phototherapy treatment (before and after phototherapy)
Measured: Before using "DRAP" device (H0), and after using (Hour12, Hour 36, Hour 48, Hour 60, Hour 72, Hour 84, Hour 96)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2015 (estimated)
- Study startDec 2014
- Primary completionDec 2017 (estimated)
- Study completionDec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Crigler Najjar SyndromeStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- VINCENT GAJDOS, Professor, Principal investigator, AP-HP, Antoine Béclère Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02356978
- Sponsor's protocol ID
- P140202
- Acronym
- DRAP
- Status last verified
- Dec 2016
- First submitted
- 2 Feb 2015
- Last updated
- 12 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Trial of a New Phototherapy Device for Treating Hyperbilirubinemia in Crigler-Najjar Patients : a New Concept
- ClinicalTrials.gov ID
- NCT02356978
- Acronym / sponsor ID
- DRAP / P140202
- Conditions
- Crigler Najjar Syndrome, Children
- Keywords
- Rare disease, Crigler-Najjar disease, Paediatric, Experimental device
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Dec 2014
- Primary completion
- Dec 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 6 Feb 2015 (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.
Evaluating the efficacity of a new device phototherapy by comparing it with conventional phototherapy. Jaundice occurs in many newborns, and is, in most cases benign, However, owing to the potential neurotoxicity of unconjugated bilirubin, newborns must be monitored to identify those who might develop severe hyperbilirubinemia an, in rare cases, acute bilirubin encephalopathy or kernicterus. Treatment of jaundice in newborn relies on phototherapy, exposing their skin to light of a specific wavelength . Fluorescent tubes or halogen lamps have been used as light sources for phototherapy for many years. Light-emitting diodes (LEDs) are more recent sources which are power efficient, have a longer life and are portable with low heat production. Several technologies and devices are developed using LEDs and specially a compact system.
Detailed description
Crigler-Najjar (CN) syndrome is a congenital inborn error of hepatic bilirubin metabolism caused by the deficiency of bilirubin uridinediphosphate glucuronosyltransferase activity CN patients are at permanent risk of life-threatening bilirubin encephalopathy (kernicterus). Treatment of CN disease relies on daily prolonged phototherapy (10-12 h/day). To date, liver transplantation is the only curative treatment available. To date, no phototherapy device exists for teenagers and adults. CN patients use homemade systems or add several neonatal systems together to reach a better efficacy.
A new device has been designed by weaving optical fibers into a sheet connected to LEDs. This new technology, marketed by several manufacturers of phototherapy devices, is used in pediatric wards for treating jaundiced newborns. A prototype of 80× 100cm was thus developed, which combines numerous advantages : important surface of treatment, easy use and transportation, user-friendliness.
Before going to a therapeutic trial for assessing the benefits of this new device in ambulatory current practice, it is essential to make sure of both its feasibility and efficiency on the control of bilirubinemia, under medical supervision.
Hypothesis: the new phototherapy sheet is as effective as, and better tolerated than the usual devices used in CN patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Device: Arm1 homemade phototherapy treatment, Device: DRAPEach patient will be treated before using the active usual homemade device, and after using the experimental new "DRAP" device. This new sheet was designed by weaving optical fibers connected to LEDs ( "BROCHIER" Technology). The "LIGHTEX" technology ® is a principle of weaving mill of optical fibres with side lighting connected to LEDs and allowing to realize flexible or stiff bright surfaces with very weak congestions, low consumption and high life cycle. The energy illumination of this device varies between 3 and 4 mW / cm ² ( average 3,6 mW / cm ².) |
Who may qualify
- Ages
- Up to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- genetic diagnosis of Crigler Najjar disease
- child whose height is between 60cm and 100cm
- patients with follow-up in reference center,
Key exclusion criteria (2 listed)
- opposition of parents
- no social security insurance
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 |
|---|---|---|
| Arm1 homemade phototherapy treatmentsession of 10 or 12 hours phototherapy treatment using the homemade device during the first night and then the "DRAP" device during the next nights. | Device | Arm 1 |
| DRAP | Device | Arm 1 |
Primary outcome
Kinetic of blood Bilirubin level using the "DRAP" device (blood bilirubin concentration)
Time frame: Before using "DRAP" device (H0), and after using (Hour12, Hour 36, Hour 48, Hour 60, Hour 72, Hour 84, Hour 96)
blood bilirubin concentration will be measured 2 times during phototherapy treatment (before and after phototherapy)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2017.
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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.
VINCENT GAJDOS, Professor principal investigator
AP-HP, Antoine Béclère Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Arm1 homemade phototherapy treatment and DRAP in people with Crigler Najjar Syndrome and Children, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 2 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: status not confirmed, as of the sponsor's last update on 12 Sep 2017. 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 France. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact VINCENT GAJDOS, Professor (+33 1 45 37 42 72, vincent.gajdos@abc.aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Kinetic of blood Bilirubin level using the "DRAP" device (blood bilirubin concentration) (time frame: Before using "DRAP" device (H0), and after using (Hour12, Hour 36, Hour 48, Hour 60, Hour 72, Hour 84, Hour 96)). 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 12 Sep 2017.