A Phase 1/2 Open-Label, Ascending Dose, Multicentre Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

Conditions

Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

Brief summary

1.1. Over a 26-week initial and 5-year total follow-up period: - Number and incidence of AEs, SAEs, and clinically meaningful laboratory test abnormalities;, 1.2. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in vital signs, ECG parameters, and physical and neurological examinations, 1.3. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in the MMSE;, 1.4. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in biochemistry and hematology safety laboratory tests, 1.5. Over a 26-week initial and 5-year total follow-up period: - Incidence of treatment-emergent suicidal ideation or behavior as measured on the C-SSRS;, 1.6. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in MRI results including edema, inflammation, asymptomatic/symptomatic hemorrhage, and other structural changes.

Detailed description

1.1. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in PGRN protein levels in CSF and blood., 1.2. Over a 5-year follow-up period: - Change from baseline in CDR + NACC FTLD-SB score; and - Change in CGI-C, PGI-C, and CaGI-C

Related papers

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Interventions

DRUGAVB-101

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
1.1. Over a 26-week initial and 5-year total follow-up period: - Number and incidence of AEs, SAEs, and clinically meaningful laboratory test abnormalities;, 1.2. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in vital signs, ECG parameters, and physical and neurological examinations, 1.3. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in the MMSE;, 1.4. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in biochemistry and hematology safety laboratory tests, 1.5. Over a 26-week initial and 5-year total follow-up period: - Incidence of treatment-emergent suicidal ideation or behavior as measured on the C-SSRS;, 1.6. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in MRI results including edema, inflammation, asymptomatic/symptomatic hemorrhage, and other structural changes.	—

Measure

Time frame

1.1. Over a 26-week initial and 5-year total follow-up period: - Number and incidence of AEs, SAEs, and clinically meaningful laboratory test abnormalities;, 1.2. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in vital signs, ECG parameters, and physical and neurological examinations, 1.3. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in the MMSE;, 1.4. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in biochemistry and hematology safety laboratory tests, 1.5. Over a 26-week initial and 5-year total follow-up period: - Incidence of treatment-emergent suicidal ideation or behavior as measured on the C-SSRS;, 1.6. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in MRI results including edema, inflammation, asymptomatic/symptomatic hemorrhage, and other structural changes.

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Secondary

Measure	Time frame
1.1. Over a 26-week initial and 5-year total follow-up period: - Change from baseline in PGRN protein levels in CSF and blood., 1.2. Over a 5-year follow-up period: - Change from baseline in CDR + NACC FTLD-SB score; and - Change in CGI-C, PGI-C, and CaGI-C	—

Countries

Belgium, Italy, Netherlands, Poland, Spain, Sweden

Outcome results

None listed