A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM)

Conditions

Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN)

Brief summary

The following primary safety endpoints will be measured up to 5 years: • Incidence and severity of treatment-emergent adverse events and serious adverse events (see study protocol for details)., • For patients who received Sirolimus: treatment-emergent hypercholesterolemia or hyperlipidemia; treatment-emergent proteinuria; treatment-emergent interstitial lung disease; and sirolimus trough levels., • For patients who received Rituximab: treatment-emergent hypogammaglobulinemia; and lymphocyte immunophenotyping., • Incidence of procedure or treatment-emergent safety findings per brain and spine MRI., • Change from baseline in immunogenicity of AAV9, PGRN, and NfL in blood and in CSF over time., The following are the primary efficacy endpoints: • Change from baseline in PGRN levels in blood over time. • Change from baseline in PGRN levels in CSF over time.

Detailed description

• Change from baseline in CDR plus NACC FTLD over time. • Change from baseline in NfL levels in blood over time. • Change from baseline in NfL levels in CSF over time.

Related papers

No related papers found yet.

Interventions

DRUGRapamune 1 mg coated tablets

DRUGPREDNISONA CINFA 10 MG COMPRIMIDOS

DRUGRapamune 0.5 mg coated tablets

DRUGSIROLIMUS

DRUGPrednisone Mylan Pharma 5 mg compresse

DRUGSolu-Medrol 1000 mg por és oldószer oldatos injekcióhoz

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
The following primary safety endpoints will be measured up to 5 years: • Incidence and severity of treatment-emergent adverse events and serious adverse events (see study protocol for details)., • For patients who received Sirolimus: treatment-emergent hypercholesterolemia or hyperlipidemia; treatment-emergent proteinuria; treatment-emergent interstitial lung disease; and sirolimus trough levels., • For patients who received Rituximab: treatment-emergent hypogammaglobulinemia; and lymphocyte immunophenotyping., • Incidence of procedure or treatment-emergent safety findings per brain and spine MRI., • Change from baseline in immunogenicity of AAV9, PGRN, and NfL in blood and in CSF over time., The following are the primary efficacy endpoints: • Change from baseline in PGRN levels in blood over time. • Change from baseline in PGRN levels in CSF over time.	—

Measure

Time frame

The following primary safety endpoints will be measured up to 5 years: • Incidence and severity of treatment-emergent adverse events and serious adverse events (see study protocol for details)., • For patients who received Sirolimus: treatment-emergent hypercholesterolemia or hyperlipidemia; treatment-emergent proteinuria; treatment-emergent interstitial lung disease; and sirolimus trough levels., • For patients who received Rituximab: treatment-emergent hypogammaglobulinemia; and lymphocyte immunophenotyping., • Incidence of procedure or treatment-emergent safety findings per brain and spine MRI., • Change from baseline in immunogenicity of AAV9, PGRN, and NfL in blood and in CSF over time., The following are the primary efficacy endpoints: • Change from baseline in PGRN levels in blood over time. • Change from baseline in PGRN levels in CSF over time.

—

Secondary

Measure	Time frame
• Change from baseline in CDR plus NACC FTLD over time. • Change from baseline in NfL levels in blood over time. • Change from baseline in NfL levels in CSF over time.	—

Countries

Belgium, France

Outcome results

None listed