Down SyndromePhase 2NCT00928304

Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.

A Phase 2 study testing Florbetaben (BAY94-9172) in people with Down Syndrome and Amyloid Beta-protein, run by Lantheus Biosciences Ltd. at 2 sites 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

Who is it for?: Ages 21 years and olderHealthy volunteers can take part
Conditions the study is for: Down Syndrome, Amyloid Beta-protein
What is being tested?: Florbetaben (BAY94-9172)
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 109 participants
Where: 2 sitesUnited States
Study dates: Jun 2009to Jan 2011
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 21 years and older
All sexes
Healthy volunteers accepted
Adult, Older adult

Who may be able to join

Study participants were individuals with DS and healthy volunteers (HVs).
Main inclusion criteria for individuals without DS
>=21 and <= 40 years of age
Mini-Mental State Examination (MMSE) >= 28
Clinical Dementia Rating (CDR) of 0
Main inclusion criteria for individuals with DS
>= 40 years of age

Who cannot join

Main exclusion criteria for both groups
Unstable medical or psychiatric condition, study specific screening procedures with the understanding that the patient has the right to withdraw from the study at any time

Trial locations and contacts

2 sites in 1 country.

United States2 sites

Study site
New Haven, Connecticut, 06510
Study site
Sun City, Arizona, 85351

Other trials in United States

About this study

Official title: An Open-Label, Non-Randomized Study to Evaluate the Efficacy and Safety of BAY94-9172 (ZK 6013443) Positron Emission Tomography (PET) for Detection of Cerebral ß-Amyloid in Individuals With Down Syndrome Compared to Individuals Without Down Syndrome

To determine the sensitivity of the visual assessment of BAY94-9172 PET images in detecting cerebral β-amyloid in individuals with Down Syndrome (DS) and specificity in individuals without DS. Given that individuals with Down Syndrome develop β-amyloid pathology over the age of 40, the clinical diagnosis of Down Syndrome will serve as the standard of truth.

Conditions: Down Syndrome, Amyloid Beta-protein
Being tested: Florbetaben (BAY94-9172)
Keywords: Amyloid beta-protein, Down Syndrome
Medical subject headings: Down Syndrome, Plaque, Amyloid

What participants receive

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

Florbetaben (BAY94-9172)Drug
300 megabecquerels (MBq) as single IV injection of 2 to 10 mL

Study groups in NCT00928304
Study group	Role	What they receive
Florbetaben (BAY94-9172)	Experimental	Not described Receives: Drug: Florbetaben (BAY94-9172)

What is being measured

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

Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome
The primary variables are sensitivity (percentage of DS subjects positive for cerebral beta-amyloid) and specificity (percentage of healthy volunteers negative for cerebral beta-amyloid) of brain uptake of florbetaben based on the majority read of the visual assessment by three independent blinded readers of PET images obtained 100 to 120 minutes post-injection of florbetaben.
Measured: 100-120 min

Secondary outcomes (3)

Sensitivity Results in the Down Syndrome Age Subgroups
The majority read sensitivity (percentage of DS subjects positive for cerebral beta-amyloid by majority read) was computed for the group of DS subjects with age equal to or below the median age (DS-young) and for DS subjects with age above the median age (DS-old). The median age was 46 yrs, with 3 subjects being exactly 46 yrs old. As defined, these subjects were assigned to the DS-young group.
Measured: 100 - 120 min
Quantitative Parameters Standard Uptake Value Ratio
The Standard Uptake Value Ratios for florbetaben signal in the frontal cortex, posterior cingulate, lateral temporal cortex, parietal cortex, and cerebellum (white matter) were determined as a quantitative measure of tracer uptake. The SUV is defined as the ratio of (1) the tissue radioactivity concentration c (in MBq/kg) at time point t, and (2) the injected activity (in MBq, extrapolated to the same time t) divided by the body weight (in kg). These SUV numbers from regions of interest were then used to derive SUV ratios (SUVR) using the SUV from the cerebellar cortex as reference.
Measured: 100 - 120 min p.i.
Consistency Between Visual and Quantitative Efficacy
For a comparison of the results of the visual assessment with the quantitative assessment, descriptive Standardized Uptake Value Ratio (SUVR) statistics were computed separately for DS subjects with an abnormal/normal majority read of the PET scan (DS-PET+/DS-PET-). The SUV is defined as the ratio of (1) the tissue radioactivity concentration c (in MBq/kg) at time point t, and (2) the injected activity (in MBq, extrapolated to the same time t) divided by the body weight (in kg). These SUV numbers from regions of interest were then used to derive SUV ratios (SUVR) using the SUV from the cerebellar cortex as reference.
Measured: 100 - 120 min

Timeline

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

Jun 2009Jan 2011

First posted25 Jun 2009 (estimated)
Study startJun 2009
Primary completionJan 2011
Study completionJan 2011

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 (1)Papers the sponsor has linked to this study.
Other trials for Down SyndromeStudies 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 2
Primary purpose: Diagnosis
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 109 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00928304
Sponsor's protocol ID: 14311
Status last verified: Jan 2014
First submitted: 24 Jun 2009
Last updated: 5 Mar 2014
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Bullich S, Roe-Vellve N, Marquie M, Landau SM, Barthel H, Villemagne VL, Sanabria A, Tartari JP, Sotolongo-Grau O, Dore V, Koglin N, Muller A, Perrotin A, Jovalekic A, De Santi S, Tarraga L, Stephens AW, Rowe CC, Sabri O, Seibyl JP, Boada M. Early detection of amyloid load using 18F-florbetaben PET. Alzheimers Res Ther. 2021 Mar 27;13(1):67. doi: 10.1186/s13195-021-00807-6.
PubMed 33773598

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?

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Study at a glance

From the registry record, last updated by the sponsor on 5 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label, Non-Randomized Study to Evaluate the Efficacy and Safety of BAY94-9172 (ZK 6013443) Positron Emission Tomography (PET) for Detection of Cerebral ß-Amyloid in Individuals With Down Syndrome Compared to Individuals Without Down Syndrome
ClinicalTrials.gov ID: NCT00928304
Acronym / sponsor ID: 14311
Conditions: Down Syndrome, Amyloid Beta-protein
Keywords: Amyloid beta-protein, Down Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 109 participants (actual)
Lead sponsor: Lantheus Biosciences Ltd.Industry (pharma, biotech, devices)
Study start: Jun 2009
Primary completion: Jan 2011 (actual)
Study completion: Jan 2011 (actual)
First posted: 25 Jun 2009 (estimated)
Results: Posted 28 Jan 2014

Clinical summary

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

Study design

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

Arm	Type	Receives
Florbetaben (BAY94-9172)	Experimental	Drug: Florbetaben (BAY94-9172)

Who may qualify

Ages: 21 years and older
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Study participants were individuals with DS and healthy volunteers (HVs).
Main inclusion criteria for individuals without DS
Main inclusion criteria for individuals with DS

Key exclusion criteria (2 listed)

Main exclusion criteria for both groups

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
Florbetaben (BAY94-9172)300 megabecquerels (MBq) as single IV injection of 2 to 10 mL	Drug	Florbetaben (BAY94-9172)

Primary outcome

Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome
Time frame: 100-120 min
The primary variables are sensitivity (percentage of DS subjects positive for cerebral beta-amyloid) and specificity (percentage of healthy volunteers negative for cerebral beta-amyloid) of brain uptake of florbetaben based on the majority read of the visual assessment by three independent blinded readers of PET images obtained 100 to 120 minutes post-injection of florbetaben.

3 secondary outcomes

Sensitivity Results in the Down Syndrome Age Subgroups
Time frame: 100 - 120 min
Quantitative Parameters Standard Uptake Value Ratio
Time frame: 100 - 120 min p.i.
Consistency Between Visual and Quantitative Efficacy
Time frame: 100 - 120 min

Results and safety

Results posted to the registry on 28 Jan 2014, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Down Syndrome Group	39	39
Healthy Volunteer Group	70	70

Primary outcome: Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome

Time frame: 100-120 min. Number (95% Confidence Interval). Unit: percentage of subjects

Measure	Majority Read n = 109
Sensitivity	46.15 (30.51 to 61.80)
Specificity	100.00 (100.00 to 100.00)

Population analysed: All subjects in the down syndrome population and healthy volunteers were included in this analysis.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: 8 days. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Down Syndrome Group	0 of 39 (0%)	2 of 39 (5.1%)	Not reported
Healthy Volunteer Group	0 of 70 (0%)	5 of 70 (7.1%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2014.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 33773598Bullich S, Roe-Vellve N, Marquie M, Landau SM, Barthel H, Villemagne VL, Sanabria A, Tartari JP, Sotolongo-Grau O, Dore V, Koglin N, Muller A, Perrotin A, Jovalekic A, De Santi S, Tarraga L, Stephens AW, Rowe CC, Sabri O, Seibyl JP, Boada M. Early detection of amyloid load using 18F-florbetaben PET. Alzheimers Res Ther. 2021 Mar 27;13(1):67. doi: 10.1186/s13195-021-00807-6.

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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.

Bayer Study Director study director

Bayer

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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Florbetaben (BAY94-9172) in people with Down Syndrome and Amyloid Beta-protein, run by Lantheus Biosciences Ltd. at 2 sites 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 7 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: finished, as of the sponsor's last update on 5 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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 28 Jan 2014). 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?

Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome (time frame: 100-120 min). 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 NCT00928304, which also lists 1 linked publication.

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 5 Mar 2014.