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Angelman SyndromePhase 1NCT04863794

A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants

A Phase 1 study testing RO7248824 in people with Angelman Syndrome, run by Hoffmann-La Roche at 1 site in Netherlands.

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

The aim of Study BP41660 is to quantify the amount and concentration of [89Zr]DFO-RO7248824 in the brain with positron emission tomography (PET) following a single sub-pharmacological dose of RO7248824 and [89Zr]DFO-RO7248824 administered via IT injection to healthy participants. Read more about this study

Who is it for?
Ages 25 to 55 years, men onlyHealthy volunteers can take part
Conditions the study is for
Angelman Syndrome
What is being tested?
RO7248824
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
1 siteNetherlands
Study dates
29 Apr 2021to 1 Aug 2022
Phase
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 25 to 55 years
  • Men only
  • Healthy volunteers accepted
  • Adult

Who may be able to join

  • Informed Consent
  • Able and willing to provide written informed consent and to comply with the study protocol according to ICH and local regulations
  • Age
  • Aged from 25 to to 55 years at the time of dosing
  • Type of Participants and Disease Characteristics
  • Overtly healthy (defined by absence of evidence of any active or chronic disease) as determined by medical evaluation including:
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  • A detailed medical and surgical history
  • A complete physical and neurological examination
  • Vital signs
  • 12-lead ECG
  • Hematology
  • Coagulation
  • Blood chemistry
  • Serology and urinalysis
  • Fluent in the language of the Investigator and study staff, and able to communicate with the study staff
  • Weight
  • Body mass index (BMI) of ≥ 18 to ≤ 30 kg/m2 at screening
  • Sex
  • Male participants only who, for 3 months after the dosing of RO7248824, agree to:
  • Remain abstinent (refrain from heterosexual intercourse) or use contraceptive barrier measures such as a condom, with a female partner of childbearing potential, or pregnant female partner, to avoid exposing the embryo
  • Refrain from donating sperm

Who cannot join

  • Medical Conditions
  • Any condition or disease detected during the medical interview/physical examination that would render the participant unsuitable for the study, place the participant at undue risk or interfere with the ability of the participant to complete the study, as determined by the Investigator
  • History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study treatment; or interfering with the interpretation of data
  • History or presence of clinically significant cardiovascular disease in the opinion of the Investigator
  • History or presence of an abnormal ECG that is clinically significant in the Investigator's opinion
  • Uncontrolled arrhythmias or history of clinically significant arrhythmias
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  • Confirmed abnormal blood pressure
  • Abnormal pulse rate
  • Abnormalities in brain and
  • Evidence or history of clinically significant back pain, back pathology and/or back injury
  • Evidence or history of significant active bleeding or coagulation disorder
  • Allergy to lidocaine (Xylocaine) or its derivatives
  • Medical or surgical conditions for which LP or associated procedures is contraindicated
  • Alanine transaminase (ALT) and bilirubin > 1.5 x upper limit of normal (ULN)
  • Current or chronic history of liver disease, or known hepatic or biliary abnormalities
  • History of convulsions or history of loss of consciousness
  • Sensitivity to any of the study treatments, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates the participation in the study
  • Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first study drug administration
  • Clinically significant abnormalities in laboratory test results
  • Prior/Concomitant Therapy
  • Used or intends to use any prohibited medications
  • Likely to need concomitant medication during the study period
  • Prior/Concurrent Clinical Study Experience
  • Participating in an investigational drug or device study within 60 days prior to screening, as calculated from the day of follow-up from the previous study, or more than 4 participations in an investigational drug or device study within a year prior to dosing
  • Previously (within the past 12 months from dosing) included in medical research and/or a medical protocol involving PET or radiological investigations, or other exposure to ionizing radiation, which combined with this study would result in an effective dose of 10 mSv or more
  • Diagnostic Assessments
  • Positive test for drugs of abuse or alcohol
  • Presence of hepatitis B surface antigen (HBsAg), positive hepatitis C antibody test result at screening or within 3 months prior to first dose of study treatment)
  • Evidence of HIV infection and/or positive human HIV antibodies
  • Other Exclusions
  • Any suspicion or history of alcohol abuse and/or suspicion of regular consumption of drug of abuse or previous history of or treatment for a dependence disorder
  • Regularly smoking more than 5 cigarettes daily or equivalent and unable or unwilling not to smoke or not to use other nicotine containing products during the in-house period
  • Donated over 500 mL of blood or blood products or had significant blood loss within 3 months prior to screening
  • Under judicial supervision, guardianship or curatorship
  • Not able to undergo PET, CT, or MRI scans
  • Previous lumbar surgery that is likely, in the opinion of the Investigator or surgical team, to make IT injection unduly difficult or hazardous
  • Scoliosis or spinal deformity preventing IT injection

Trial locations and contacts

1 site in 1 country.

Netherlands1 site

  • Pra International Group B.V

    Groningen, 9728 NZ

Other trials in Netherlands

About this study

Official title: A Non-Randomized, Open-Label, Adaptive, Single Center, Positron Emission Tomography (Pet) Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants

The aim of Study BP41660 is to quantify the amount and concentration of [89Zr]DFO-RO7248824 in the brain with positron emission tomography (PET) following a single sub-pharmacological dose of RO7248824 and [89Zr]DFO-RO7248824 administered via IT injection to healthy participants.

Being tested
RO7248824
Medical subject headings
Angelman Syndrome

What participants receive

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

  • RO7248824Drug

    A single dose of 10 mg RO7248824 will be used for this PET study in healthy participants.

Study groups in NCT04863794
Study groupRoleWhat they receive
RO7248824ExperimentalIn Part 1 of the study RO7248824 and [89Zr]-labeled RO7248824 will be administered as a single bolus IT injection following a standard IT Administration procedure. In Part 2 of the study, it is planned to test up to 3 additional IT procedures. This part is tentative with regard to its conduct and the number of procedures that may be tested.
Receives: Drug: RO7248824

What is being measured

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

  1. Quantification of [89Zr]DFO-RO7248824 distribution within the central nervous system (CNS)

    Measured: Baseline up to 6 weeks

Secondary outcomes (1)

  1. Percentage of participants with adverse events (AEs)

    Measured: Baseline up to 6 weeks

Timeline

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

  1. First posted28 Apr 2021
  2. Study start29 Apr 2021
  3. Primary completion1 Aug 2022
  4. Study completion1 Aug 2022

Resources

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04863794
Sponsor's protocol ID
BP41660
Status last verified
Sep 2022
First submitted
26 Apr 2021
Last updated
8 Sep 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing RO7248824 in people with Angelman Syndrome, run by Hoffmann-La Roche at 1 site in Netherlands.

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 21 inclusion and 37 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 8 Sep 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 Netherlands. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Quantification of [89Zr]DFO-RO7248824 distribution within the central nervous system (CNS) (time frame: Baseline up to 6 weeks). 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 NCT04863794.

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 8 Sep 2022.

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