Congenital HyperinsulinismPhase 2NCT00674440

Utility of [F-18] fluoroDOPA for Neonatal Hyperinsulinism

A Phase 2 study testing F-DOPA and PET scan in people with Congenital Hyperinsulinism and Hyperinsulinism, run by Children's Hospital of Philadelphia at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Children with congenital hyperinsulinism (CHI) have low blood sugar, and some of these children may require surgery. In this study, researchers affiliated with the University of Pennsylvania will test how well a radioactive drug (called F-DOPA) can detect a form of hyperinsulinism that may be cured by surgery. Read more about this study

Who is it for?: Ages Up to 18 years
Conditions the study is for: Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy
What is being tested?: F-DOPA and PET scan
Type of treatment: Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 106 participants
Where: 1 siteUnited States
Study dates: Dec 2004to Jan 2009
Phase: Phase 2
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 Up to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Any age, but primarily infants 0-6 months.
Children with confirmed diagnosis of congenital hyperinsulinism.

Who cannot join

Cases in which surgery will not be considered by parents or guardians.

Trial locations and contacts

1 site in 1 country.

United States1 site

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: Localization of Focal Forms of Hyperinsulinism of Infancy With 18F-labeled L-fluoro-DOPA PET Scan

Read the detailed description

For children with congenital hyperinsulinism (CHI), low blood sugar is caused by cells in the pancreas that release too much insulin. Some children with CHI have these cells throughout their pancreas; others have them located in specific areas of the pancreas. Children who have them located in specific areas of the pancreas may be cured with surgery. F-DOPA is a radioactive drug that may go to these very cells. F-DOPA can also be used for positron emission tomography (or PET), an imaging technique used in nuclear medicine departments. In this study, researchers will test the possibility of using PET with F-DOPA in the diagnosis of children with hyperinsulinism.

Conditions: Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy, CHI, PHHI
Being tested: F-DOPA, PET scan
Keywords: Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy, CHI, PHHI, F-DOPA, L-fluoro-dihydroxyphenylalanine
Medical subject headings: Congenital Hyperinsulinism, Hyperinsulinism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

F-DOPADrug
Also called 18F-labeled L-fluorodeoxyphenylalanine, 18F-DOPA
0.08-0.16 mCi/kg once
PET scanRadiation

Study groups in NCT00674440
Study group	Role	What they receive
Subjects who had PET and surgery	Experimental	Children diagnosed with hyperinsulinism who have failed other non-surgical interventions and are candidates to be scheduled for surgery for partial pancreatectomy. Eligible children will undergo PET imaging with F-DOPA prior to surgery. Receives: Drug: F-DOPA; Radiation: PET scan

What is being measured

The primary outcome is the main question the study is designed to answer.

The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism
Comparison of PET scan results with outcome of surgery and histopathology results confirmed as focal or diffuse
Measured: up to 1 month post surgical intervention

Secondary outcomes (1)

Safety of F-DOPA PET
Number of Participants with Adverse Events
Measured: 72 hours maximum or prior to pancreatic surgery

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2004Jan 2009

First posted7 May 2008 (estimated)
Study startDec 2004
Primary completionDec 2008
Study completionJan 2009

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.
Publications (8)Papers the sponsor has linked to this study.
Other trials for Congenital HyperinsulinismStudies of the same condition, open and under way.
More trials run by Children's Hospital of PhiladelphiaThe 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 2
Primary purpose: Diagnosis
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 106 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00674440
Sponsor's protocol ID: 04-003683
Other IDs: FD-R-003457-01
Status last verified: Oct 2022
First submitted: 5 May 2008
Last updated: 9 Nov 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Hardy OT, Litman RS. Congenital hyperinsulinism - a review of the disorder and a discussion of the anesthesia management. Paediatr Anaesth. 2007 Jul;17(7):616-21. doi: 10.1111/j.1460-9592.2007.02192.x.
PubMed 17564642
Otonkoski T, Nanto-Salonen K, Seppanen M, Veijola R, Huopio H, Hussain K, Tapanainen P, Eskola O, Parkkola R, Ekstrom K, Guiot Y, Rahier J, Laakso M, Rintala R, Nuutila P, Minn H. Noninvasive diagnosis of focal hyperinsulinism of infancy with [18F]-DOPA positron emission tomography. Diabetes. 2006 Jan;55(1):13-8.
PubMed 16380471
Ribeiro MJ, Boddaert N, Delzescaux T, Valayannopoulos V, Bellanne-Chantelot C, Jaubert F, Verkarre V, Nihoul-Fekete C, Brunelle F, Lonlay P. Functional imaging of the pancreas: the role of [18F]fluoro-L-DOPA PET in the diagnosis of hyperinsulinism of infancy. Endocr Dev. 2007;12:55-66. doi: 10.1159/000109605.
PubMed 17923769
Subramaniam RM, Karantanis D, Peller PJ. [18F]Fluoro-L-dopa PET/CT in congenital hyperinsulinism. J Comput Assist Tomogr. 2007 Sep-Oct;31(5):770-2. doi: 10.1097/RCT.0b013e318031f55c.
PubMed 17895790
Ribeiro MJ, Boddaert N, Bellanne-Chantelot C, Bourgeois S, Valayannopoulos V, Delzescaux T, Jaubert F, Nihoul-Fekete C, Brunelle F, De Lonlay P. The added value of [18F]fluoro-L-DOPA PET in the diagnosis of hyperinsulinism of infancy: a retrospective study involving 49 children. Eur J Nucl Med Mol Imaging. 2007 Dec;34(12):2120-8. doi: 10.1007/s00259-007-0498-y. Epub 2007 Jul 28.
PubMed 17661030
de Lonlay P, Simon-Carre A, Ribeiro MJ, Boddaert N, Giurgea I, Laborde K, Bellanne-Chantelot C, Verkarre V, Polak M, Rahier J, Syrota A, Seidenwurm D, Nihoul-Fekete C, Robert JJ, Brunelle F, Jaubert F. Congenital hyperinsulinism: pancreatic [18F]fluoro-L-dihydroxyphenylalanine (DOPA) positron emission tomography and immunohistochemistry study of DOPA decarboxylase and insulin secretion. J Clin Endocrinol Metab. 2006 Mar;91(3):933-40. doi: 10.1210/jc.2005-1713. Epub 2006 Jan 10.
PubMed 16403819
Hardy OT, Hernandez-Pampaloni M, Saffer JR, Scheuermann JS, Ernst LM, Freifelder R, Zhuang H, MacMullen C, Becker S, Adzick NS, Divgi C, Alavi A, Stanley CA. Accuracy of [18F]fluorodopa positron emission tomography for diagnosing and localizing focal congenital hyperinsulinism. J Clin Endocrinol Metab. 2007 Dec;92(12):4706-11. doi: 10.1210/jc.2007-1637. Epub 2007 Sep 25.
PubMed 17895314
Hardy OT, Hernandez-Pampaloni M, Saffer JR, Suchi M, Ruchelli E, Zhuang H, Ganguly A, Freifelder R, Adzick NS, Alavi A, Stanley CA. Diagnosis and localization of focal congenital hyperinsulinism by 18F-fluorodopa PET scan. J Pediatr. 2007 Feb;150(2):140-5. doi: 10.1016/j.jpeds.2006.08.028.
PubMed 17236890

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Umbilical Cord Glucose
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HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)
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Early Check: Expanded Screening in Newborns
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RecruitingPhase 2
18FluoroLDOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism
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RecruitingPhase 3
18F-DOPA II - PET Imaging Optimization
Congenital Hyperinsulinism, Neuroblastoma

All Congenital Hyperinsulinism trials

Study at a glance

From the registry record, last updated by the sponsor on 9 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Localization of Focal Forms of Hyperinsulinism of Infancy With 18F-labeled L-fluoro-DOPA PET Scan
ClinicalTrials.gov ID: NCT00674440
Acronym / sponsor ID: 04-003683
Conditions: Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy, CHI, PHHI
Keywords: Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy, CHI, PHHI, F-DOPA, L-fluoro-dihydroxyphenylalanine
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 106 participants (actual)
Lead sponsor: Children's Hospital of PhiladelphiaUniversity, hospital or other
Collaborators: University of Pennsylvania
Study start: Dec 2004
Primary completion: Dec 2008 (actual)
Study completion: Jan 2009 (actual)
First posted: 7 May 2008 (estimated)
Results: Posted 9 Nov 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Diagnosis
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Subjects who had PET and surgery	Experimental	Drug: F-DOPA, Radiation: PET scanChildren diagnosed with hyperinsulinism who have failed other non-surgical interventions and are candidates to be scheduled for surgery for partial pancreatectomy. Eligible children will undergo PET imaging with F-DOPA prior to surgery.

Who may qualify

Ages: Up to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (2 listed)

Any age, but primarily infants 0-6 months.
Children with confirmed diagnosis of congenital hyperinsulinism.

Key exclusion criteria (1 listed)

Cases in which surgery will not be considered by parents or guardians.

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
F-DOPAAlso: 18F-labeled L-fluorodeoxyphenylalanine, 18F-DOPA0.08-0.16 mCi/kg once	Drug	Subjects who had PET and surgery
PET scan	Radiation	Subjects who had PET and surgery

Primary outcome

The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism
Time frame: up to 1 month post surgical intervention
Comparison of PET scan results with outcome of surgery and histopathology results confirmed as focal or diffuse

1 secondary outcome

Safety of F-DOPA PET
Time frame: 72 hours maximum or prior to pancreatic surgery

Results and safety

Results posted to the registry on 9 Nov 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
All Subjects Consented	106	95

Primary outcome: The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism

Time frame: up to 1 month post surgical intervention. Count of participants. Unit: Participants

Measure	Subjects Who Had PET and Surgery n = 95
True Negatives (PET diffuse/surgery diffuse)	40
False negatives (PET diffuse/surgery focal):	6
False positives (PET focal/surgery diffuse):	3
True positives (PET focal/surgery focal):	46

Subjects Who Had PET and Surgery: Sensitivity: 93.0 (95% CI 80.9 to 98.5)
Subjects Who Had PET and Surgery: Specifity: 88.5 (95% CI 76.6 to 95.6)

Population analysed: An F-DOPA PET/CT imaging study was performed and reported prior to surgery. This data includes surgical histopathology confirmed as focal or diffuse. 95 of the 106 original consented participants had surgical results that were evaluable. (1 consented but never scanned, 7 did not have surgery, 3 had surgery but were excluded for atypical histology that did not fit classic focal or diffuse or had second PET scan after initial surgery therefore not counted twice).

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Reviewed within 72 hours post PET or prior to pancreatic surgery (which ever was first).. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
All Subjects Who Had PET	0 of 105 (0%)	0 of 105 (0%)	0 of 105

Limitations and caveats (from the sponsor): Some subjects have atypical disease that is neither focal or diffuse. Rapid genetic test results were not available at the beginning of the study.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Nov 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 17564642Hardy OT, Litman RS. Congenital hyperinsulinism - a review of the disorder and a discussion of the anesthesia management. Paediatr Anaesth. 2007 Jul;17(7):616-21. doi: 10.1111/j.1460-9592.2007.02192.x.
Background: PubMed 16380471Otonkoski T, Nanto-Salonen K, Seppanen M, Veijola R, Huopio H, Hussain K, Tapanainen P, Eskola O, Parkkola R, Ekstrom K, Guiot Y, Rahier J, Laakso M, Rintala R, Nuutila P, Minn H. Noninvasive diagnosis of focal hyperinsulinism of infancy with [18F]-DOPA positron emission tomography. Diabetes. 2006 Jan;55(1):13-8.
Background: PubMed 17923769Ribeiro MJ, Boddaert N, Delzescaux T, Valayannopoulos V, Bellanne-Chantelot C, Jaubert F, Verkarre V, Nihoul-Fekete C, Brunelle F, Lonlay P. Functional imaging of the pancreas: the role of [18F]fluoro-L-DOPA PET in the diagnosis of hyperinsulinism of infancy. Endocr Dev. 2007;12:55-66. doi: 10.1159/000109605.
Background: PubMed 17895790Subramaniam RM, Karantanis D, Peller PJ. [18F]Fluoro-L-dopa PET/CT in congenital hyperinsulinism. J Comput Assist Tomogr. 2007 Sep-Oct;31(5):770-2. doi: 10.1097/RCT.0b013e318031f55c.
Background: PubMed 17661030Ribeiro MJ, Boddaert N, Bellanne-Chantelot C, Bourgeois S, Valayannopoulos V, Delzescaux T, Jaubert F, Nihoul-Fekete C, Brunelle F, De Lonlay P. The added value of [18F]fluoro-L-DOPA PET in the diagnosis of hyperinsulinism of infancy: a retrospective study involving 49 children. Eur J Nucl Med Mol Imaging. 2007 Dec;34(12):2120-8. doi: 10.1007/s00259-007-0498-y. Epub 2007 Jul 28.
Background: PubMed 16403819de Lonlay P, Simon-Carre A, Ribeiro MJ, Boddaert N, Giurgea I, Laborde K, Bellanne-Chantelot C, Verkarre V, Polak M, Rahier J, Syrota A, Seidenwurm D, Nihoul-Fekete C, Robert JJ, Brunelle F, Jaubert F. Congenital hyperinsulinism: pancreatic [18F]fluoro-L-dihydroxyphenylalanine (DOPA) positron emission tomography and immunohistochemistry study of DOPA decarboxylase and insulin secretion. J Clin Endocrinol Metab. 2006 Mar;91(3):933-40. doi: 10.1210/jc.2005-1713. Epub 2006 Jan 10.
Results publication: PubMed 17895314Hardy OT, Hernandez-Pampaloni M, Saffer JR, Scheuermann JS, Ernst LM, Freifelder R, Zhuang H, MacMullen C, Becker S, Adzick NS, Divgi C, Alavi A, Stanley CA. Accuracy of [18F]fluorodopa positron emission tomography for diagnosing and localizing focal congenital hyperinsulinism. J Clin Endocrinol Metab. 2007 Dec;92(12):4706-11. doi: 10.1210/jc.2007-1637. Epub 2007 Sep 25.
Results publication: PubMed 17236890Hardy OT, Hernandez-Pampaloni M, Saffer JR, Suchi M, Ruchelli E, Zhuang H, Ganguly A, Freifelder R, Adzick NS, Alavi A, Stanley CA. Diagnosis and localization of focal congenital hyperinsulinism by 18F-fluorodopa PET scan. J Pediatr. 2007 Feb;150(2):140-5. doi: 10.1016/j.jpeds.2006.08.028.
The Congenital Hyperinsulinism Center at The Children's Hospital of PhiladelphiaLink provided by the sponsor.
International non-profit organization created by parents/families based in the United StatesLink provided by the sponsor.

Related trials for Congenital Hyperinsulinism

Not yet recruiting
Umbilical Cord Glucose
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Active, not recruitingPhase 2
HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)
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RecruitingPhase 3
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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.

Lisa J States, MD principal investigator

Children's Hospital of Philadelphia

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing F-DOPA and PET scan in people with Congenital Hyperinsulinism and Hyperinsulinism, run by Children's Hospital of Philadelphia 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 1 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 9 Nov 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 9 Nov 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?

The Accuracy of FDOPA PET to Identify Focal Forms of Hyperinsulinism (time frame: up to 1 month post surgical intervention). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00674440, which also lists 8 linked publications.

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 9 Nov 2022.