Huntington's DiseaseEarly Phase 1PEARL-HDNCT02061722
[PETDE10] Imaging of PDE10A Enzyme Levels in Huntington's Disease Gene Expansion Carriers and Healthy Controls With PET.
A Early Phase 1 study testing PET Imaging with [18F]MNI-659 in people with Huntington's Disease, run by CHDI Foundation, Inc. at 8 sites in 4 countries.
Trial summary
The aim of this study is to measure the availability of the PDE10A enzyme in Huntington disease gene expansion carriers (HDGECs) using the recently developed radioligand [18F]MNI-659. Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Huntington's Disease
- What is being tested?
- PET Imaging with [18F]MNI-659
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 8 sitesin 4 countries
- Study dates
- Jan 2013to May 2016
- Phase
- Early Phase 1
- 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 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Capacity to give full informed consent in writing, and have read and signed the informed consent
- Age 18 to 70 years, inclusive
- HCs: Healthy according to medical history, physical examination, ECG, vital signs, laboratory assessment and MRI, with a body mass index between 19 and 27 (both inclusive)
- HDGECs: Otherwise healthy according to medical history, no co-morbidity of psychotic disorders, physical examination, vital signs and laboratory assessments
- HDGECs:
- HD Stage 1 or HD Stage 2: Patients with a clinical diagnosis of HD, defined by the presence of noticeable motor disorder and 40 CAG repeats (HD stage 1: Total Functional Capacity (TFC) 11-13, HD stage 2: TFC 7-10); (B) Pre-manifest: Subjects that are carriers of the mutant Huntingtin gene with ≥40 CAG repeats, a disease burden score ≥ 275 (calculated by the equation ((CAGn-35.5) X age)), and a Total Motor Score (TMS) ≤ 5.
- Able and willing to travel to Stockholm
- Willing to comply with use of adequate contraceptive measures:
Who cannot join
- Any disease, condition or concomitant medications that significantly compromises the function of the body systems and that, in the opinion of the Investigator might interfere with the conduct of the study or the interpretation
- Regular use of any medication prohibited by this protocol with the exception for Viagra, Levitra and Cialis, which may be temporarily discontinued prior to the PET measurements
- HDGECs: History of other neurological condition (including brain surgery, intracranial haematoma, stroke/cerebrovascular disorders, epilepsy), co-morbidity of psychotic disorders
- HCs: Anamnesis/medical history of neurological conditions (Alzheimer´s disease, dementia, Parkinson´s disease, brain surgery, intracranial haematoma, stroke/cerebrovascular disorders, epilepsy) or psychiatric conditions schizophrenia, depression, bipolar disorder, attention-deficit hyperactivity disorder)
- HCs: Family history of HD
- History of or current alcohol or drug abuse or dependence
Show all 16Show fewer
- History of anaphylactoid or anaphylactic reactions to any allergen including drugs and contrast media
- Haematological or biochemical parameters that are outside the normal range and are considered clinically significant by the Investigator
- Clinical relevant findings in the 12-lead ECG as determined by the evaluating physician
- Donation of blood (450 mL) within three months prior to Visit 3
- Contraindication to MRI, such as known claustrophobia, presence of metal devises or implants (e.g. pacemaker, vascular- or heart- valves, stents, clips), metal deposited in the body (e.g. bullets or shells), or metal grains in the eyes
- Participating in a clinical trial within the past 3 months
- HCs: previous participation in another PET study
- Positive viral test result for Hepatitis B or C or HIV 1 or 2
- Female subjects: breast-feeding or positive pregnancy test at screening or at Visit 3
- Contraindication for arterial cannulation (by assessment of Allen's test)
Trial locations and contacts
8 sites in 4 countries.
8 sites in 4 countries
Sweden5 sites
Skane Universitetssjukhus Lund, Neurologiska kliniken
Lund, SE-221 85
Karolinska Universitetssjukhus, Huddinge
Stockholm, SE-141 86
Karolinska University Hospital
Stockholm, SE-171 76
KTA Karolinska Trial Alliance
Stockholm, SE-171 76
Neurologkliniken Akademiska sjukhuset, ing 85
Uppsala, SE-751 85
Netherlands1 site
Leiden University Medical Center, Department of Neurology
Leiden, K5Q112
About this study
Official title: [PETDE10] Imaging of Phosphodiesterase 10 A (PDE10A) Enzyme Levels in the Living Human Brain of Huntington´s Disease Gene Expansion Carriers and Healthy Controls With Positron Emission Tomography
The aim of this study is to measure the availability of the PDE10A enzyme in Huntington disease gene expansion carriers (HDGECs) using the recently developed radioligand [18F]MNI-659. The study will be cross-sectional, examining HDGECs at different stages of the disease (pre-manifest, stage 1 and stage 2), in comparison with Healthy Controls (HCs). The HDGECs included in this study will be recruited from the large database of the REGISTRY (NCT01590589) or ENROLL-HD (NCT01574053) studies.
Read the detailed description
The study will be organized in an "adaptive-like" mode. Initially a cohort of 5 HDGECs and 5 HCs will be studied. The data obtained in the first cohort will be analysed and depending on the variability of the data, there will be approximately 10 HDGECs and an equal number of HCs in the second cohort and the size of the third cohort may be altered to in total include approximately 45 HDGECs and an equal number of HCs.
- Conditions
- Huntington's Disease
- Being tested
- PET Imaging with [18F]MNI-659
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PET Imaging with [18F]MNI-659Radiation
The radioligands [11C]raclopride and [18F]MNI-659 will be administered at doses less than 10 micrograms, i.e. within the micro dosing concept, and no pharmacological effects are expected.
The injected radioactivity of [11C]raclopride will be 300 MBq/70 kg of body weight ± 10%.
The injected radioactivity of [18F]MNI-659 will be 185 MBq/70 kg of body weight ± 10%.
| Study group | Role | What they receive |
|---|---|---|
| PET Imaging with [18F]MNI-659 | Experimental | All subjects (both HDGECs and HCs) will receive single intravenous doses of the radioligands [11C]raclopride (non-investigational medicinal product [NIMP]) and [18F]MNI-659 (investigational medicinal product [IMP]).
The radioligands [11C]raclopride and [18F]MNI-659 will be administered at doses less than 10 micrograms, i.e. within the micro dosing concept, and no pharmacological effects are expected.
The injected radioactivity of [11C]raclopride will be 300 MBq/70 kg of body weight ± 10%.
The injected radioactivity of [18F]MNI-659 will be 185 MBq/70 kg of body weight ± 10%. Receives: Radiation: PET Imaging with [18F]MNI-659 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome measure will be the distribution volume (VT) in the striatum (caudate and putamen), globus pallidus, and ventral striatum estimated using kinetic and graphical analysis. Thalamus, cortex and cerebellum will also be examined.
Measured: Visit 3: For HD subjects, this will occur within 90 days from the Screening Visit. For Healthy Controls, this will occur within 28 days of Screening visit.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2014 (estimated)
- Study startJan 2013
- Primary completionMay 2016
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington's 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
- Early Phase 1
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- CHDI Foundation, Inc.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Bernhard Landwehrmeyer, MD, PhD, Principal investigator, Ulm University HospitalCristina Sampaio, MD, PhD, Study director, CHDI Foundation, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02061722
- Sponsor's protocol ID
- CHDIKI1201/PET-HD-PDE10A
- Acronym
- PEARL-HD
- Status last verified
- Mar 2016
- First submitted
- 3 Feb 2014
- Last updated
- 1 Jun 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- [PETDE10] Imaging of Phosphodiesterase 10 A (PDE10A) Enzyme Levels in the Living Human Brain of Huntington´s Disease Gene Expansion Carriers and Healthy Controls With Positron Emission Tomography
- ClinicalTrials.gov ID
- NCT02061722
- Acronym / sponsor ID
- PEARL-HD / CHDIKI1201/PET-HD-PDE10A
- Conditions
- Huntington's Disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- CHDI Foundation, Inc.University, hospital or other
- Study start
- Jan 2013
- Primary completion
- May 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 13 Feb 2014 (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.
The aim of this study is to measure the availability of the PDE10A enzyme in Huntington disease gene expansion carriers (HDGECs) using the recently developed radioligand [18F]MNI-659. The study will be cross-sectional, examining HDGECs at different stages of the disease (pre-manifest, stage 1 and stage 2), in comparison with Healthy Controls (HCs). The HDGECs included in this study will be recruited from the large database of the REGISTRY (NCT01590589) or ENROLL-HD (NCT01574053) studies.
Detailed description
The study will be organized in an "adaptive-like" mode. Initially a cohort of 5 HDGECs and 5 HCs will be studied. The data obtained in the first cohort will be analysed and depending on the variability of the data, there will be approximately 10 HDGECs and an equal number of HCs in the second cohort and the size of the third cohort may be altered to in total include approximately 45 HDGECs and an equal number of HCs.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PET Imaging with [18F]MNI-659 | Experimental | Radiation: PET Imaging with [18F]MNI-659All subjects (both HDGECs and HCs) will receive single intravenous doses of the radioligands [11C]raclopride (non-investigational medicinal product [NIMP]) and [18F]MNI-659 (investigational medicinal product [IMP]). The radioligands [11C]raclopride and [18F]MNI-659 will be administered at doses less than 10 micrograms, i.e. within the micro dosing concept, and no pharmacological effects are expected. The injected radioactivity of [11C]raclopride will be 300 MBq/70 kg of body weight ± 10%. The injected radioactivity of [18F]MNI-659 will be 185 MBq/70 kg of body weight ± 10%. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Capacity to give full informed consent in writing, and have read and signed the informed consent
- Age 18 to 70 years, inclusive
- HCs: Healthy according to medical history, physical examination, ECG, vital signs, laboratory assessment and MRI, with a body mass index between 19 and 27 (both inclusive)
- HDGECs: Otherwise healthy according to medical history, no co-morbidity of psychotic disorders, physical examination, vital signs and laboratory assessments
- HDGECs:
Key exclusion criteria (16 listed)
- Any disease, condition or concomitant medications that significantly compromises the function of the body systems and that, in the opinion of the Investigator might interfere with the conduct of the study or the interpretation
- Regular use of any medication prohibited by this protocol with the exception for Viagra, Levitra and Cialis, which may be temporarily discontinued prior to the PET measurements
- HDGECs: History of other neurological condition (including brain surgery, intracranial haematoma, stroke/cerebrovascular disorders, epilepsy), co-morbidity of psychotic disorders
- HCs: Anamnesis/medical history of neurological conditions (Alzheimer´s disease, dementia, Parkinson´s disease, brain surgery, intracranial haematoma, stroke/cerebrovascular disorders, epilepsy) or psychiatric conditions schizophrenia, depression, bipolar disorder, attention-deficit hyperactivity disorder)
- HCs: Family history of HD
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 |
|---|---|---|
| PET Imaging with [18F]MNI-659The radioligands [11C]raclopride and [18F]MNI-659 will be administered at doses less than 10 micrograms, i.e. within the micro dosing concept, and no pharmacological effects are expected. The injected radioactivity of [11C]raclopride will be 300 MBq/70 kg of body weight ± 10%. The injected radioactivity of [18F]MNI-659 will be 185 MBq/70 kg of body weight ± 10%. | Radiation | PET Imaging with [18F]MNI-659 |
Primary outcome
The primary outcome measure will be the distribution volume (VT) in the striatum (caudate and putamen), globus pallidus, and ventral striatum estimated using kinetic and graphical analysis. Thalamus, cortex and cerebellum will also be examined.
Time frame: Visit 3: For HD subjects, this will occur within 90 days from the Screening Visit. For Healthy Controls, this will occur within 28 days of Screening visit.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2016.
Related trials for Huntington's Disease
RecruitingPhase 2/3
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
Huntington Disease
RecruitingPhase 3
Pridopidine Phase 3 Study in Huntington's Disease
Huntington Disease
Not yet recruiting
Creatine and Beta-Hydroxy-Beta-Methylbutyrate (HMB) Supplementation Plus Home-Based Exercise for Sarcopenia in Huntington's Disease
Huntington Disease, Sarcopenia
Recruiting
Feasibility and Acceptability of an LLM-based Chatbot for Family Caregivers: Evaluation Study
Caregivers
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
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.
Bernhard Landwehrmeyer, MD, PhD principal investigator
Ulm University Hospital
Cristina Sampaio, MD, PhD study director
CHDI Foundation, Inc.
8 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing PET Imaging with [18F]MNI-659 in people with Huntington's Disease, run by CHDI Foundation, Inc. at 8 sites in 4 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 8 inclusion and 16 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 1 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Sweden, Denmark, Netherlands, Norway. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 primary outcome measure will be the distribution volume (VT) in the striatum (caudate and putamen), globus pallidus, and ventral striatum estimated using kinetic and graphical analysis. Thalamus, cortex and cerebellum will also be examined. (time frame: Visit 3: For HD subjects, this will occur within 90 days from the Screening Visit. For Healthy Controls, this will occur within 28 days of Screening visit.). 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 1 Jun 2016.