Clinical trial • Phase II • Neurology|Other

SODIUM OXYBATE for Cerebral amyloid angiopathy

Phase II trial of SODIUM OXYBATE for Cerebral amyloid angiopathy.

Overview

Trial Therapeutic Area
Neurology|Other
Trial Disease
Cerebral amyloid angiopathy
Trial Stage
Phase II
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
08-12-2023
First CTIS Authorization Date
08-04-2024

Trial design

Within-subject pre-treatment (baseline) comparison; no external drug comparator arm specified.-controlled Phase II trial across 1 site in Netherlands.

Comparator
Within-subject pre-treatment (baseline) comparison; no external drug comparator arm specified.
Target Sample Size
60
Trial Duration For Participant
183

Eligibility

Recruits 60 No vulnerable population selected. Written informed consent is required (documents listed include 'L1_SIS and ICF adults' and other adult information material). No assent procedures or minor-specific consent described..

Pregnancy Exclusion
Pregnancy/breast feeding
Vulnerable Population
No vulnerable population selected. Written informed consent is required (documents listed include 'L1_SIS and ICF adults' and other adult information material). No assent procedures or minor-specific consent described.

Inclusion criteria

  • {"criterion_text":"- Patients with D-CAA with a proven APP mutation or a history of ≥1 lobar ICH and a positive family history for D-CAA in ≥1 first degree relative • Age ≥30 years old • ≤ 2 symptomatic ICH (occurrence of ICH at least > 1 year ago) or presence of ≥ 1 haemorrhagic marker (cortical superficial siderosis, cortical microbleeds) or non-haemorrhagic marker (white matter hyperintensities, microinfarcts, enlarged perivascular spaces, lobar lacunes). • When presymptomatic, patients are aware that they have D-CAA"}
  • {"criterion_text":"- Probable sCAA according to the Modified Boston criteria 2.0 • Age ≥50 years old • ≤ 2 symptomatic ICH (occurrence of ICH at least > 1 year ago)"}
  • {"criterion_text":"- Provisional CAA when the criteria for probable sCAA are not met due to presence of deep haemorrhagic lesions but there are mostly lobar MB and cSS present or a ratio of 10 times more lobar MB than deep MB without cSS. • Age ≥50 years old • ≤ 2 symptomatic ICH (occurrence of ICH at least > 1 year ago)"}
  • {"criterion_text":"- Written informed consent"}

Exclusion criteria

  • {"criterion_text":"- Modified Rankin Score ≥ 4"}
  • {"criterion_text":"- A life expectancy of less than six months"}
  • {"criterion_text":"- Pregnancy/breast feeding"}
  • {"criterion_text":"- Contraindications for lumbar puncture"}
  • {"criterion_text":"- Unwillingness to refrain from consuming ≤ 1 alcohol unit per day and not later than 8 pm."}
  • {"criterion_text":"- Contraindications for LXB and nVNS"}
  • {"criterion_text":"- Contraindications for MRI 7T"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- The primary endpoint will be the morning Aβ 40 and 42 levels in CSF before and after treatment. CSF will be obtained through a lumbar puncture before the intervention at 3 months and after the intervention at 6 months.","definition_or_measurement_approach":"Morning Aβ40 and Aβ42 levels measured in cerebrospinal fluid (CSF) obtained by lumbar puncture before intervention (at 3 months) and after intervention (at 6 months)."}

Recruitment

Planned Sample Size
60
Recruitment Window Months
36
Consent Approach
Written informed consent required from participants (adult ICF documented). Subject information and ICF for adults are listed among study documents (e.g., 'L1_SIS and ICF adults', other subject information materials). Study materials/translations available in Dutch (translations of title/objectives present). No assent process for minors described.

Geography

Total Number Of Sites
1
Total Number Of Participants
60

Netherlands

Earliest CTIS Part Ii Submission Date
12-03-2024
Latest Decision Or Authorization Date
30-01-2026
Processing Time Days
689
Number Of Sites
1
Number Of Participants
60

Sites

Site Name
Academisch Ziekenhuis Leiden
Department Name
Neurology
Contact Person Name
Rolf Fronczek

Sponsor

Primary sponsor

Full Name
Academisch Ziekenhuis Leiden
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Netherlands

Investigational products

Investigational Product Name
SODIUM OXYBATE
Active Substance
SODIUM OXYBATE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
prodAuthStatus 2
Maximum Dose
9.00
Combination Treatment
Yes

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