(ASK-HD-01-CS-101) An Open-Label Phase I/II Dose Finding Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Striatal Administration of AB-1001 in Adult Subjects with Early Manifest Huntington’s Disease (HD)

Conditions

Huntington's disease

Brief summary

Incidence of Dose Limiting Toxicities (DLTs), Treatment Emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs) through Week 52

Detailed description

Magnitude and variability of change from baseline in anatomical and volumetric measures of brain regions impacted by HD (caudate, putamen, whole brain volume, grey matter volume, white matter volume, ventricular volume) as assessed by MRI at Week 52, Change from baseline in the Unified Huntington’s Disease Rating Scale composite score [cUHDRS: Total Motor Score (TMS), Total Functional Capacity (TFC), Symbol Digit Modalities Test (SDMT) and Stroop Word Reading (SWRT)] at Week 52, Change from baseline in blood and cerebrospinal fluid (CSF) mHTT, Neurofilament light chain (NfL), and 24-OH cholesterol at Week 52, Change from baseline in Magnetic Resonance Spectroscopy (MRS) metabolic profile (measuring lactate, N-acetyl aspartate, creatine, choline, glutamate, glutamine, glutathione, taurine, and myo-inositol) at Week 52, Change from baseline in Positron Emission Tomography (PET) Fluoro-deoxyglucose (FDG) striatal profile at Week 52

Related papers

No related papers found yet.

Interventions

DRUGAB-1001

DRUGFLUDEOXYGLUCOSE (18F)

DRUGMETHYLPREDNISOLONE HEMISUCCINATE

DRUGGADOTERIC ACID

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of Dose Limiting Toxicities (DLTs), Treatment Emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs) through Week 52	—

Secondary

Measure	Time frame
Magnitude and variability of change from baseline in anatomical and volumetric measures of brain regions impacted by HD (caudate, putamen, whole brain volume, grey matter volume, white matter volume, ventricular volume) as assessed by MRI at Week 52, Change from baseline in the Unified Huntington’s Disease Rating Scale composite score [cUHDRS: Total Motor Score (TMS), Total Functional Capacity (TFC), Symbol Digit Modalities Test (SDMT) and Stroop Word Reading (SWRT)] at Week 52, Change from baseline in blood and cerebrospinal fluid (CSF) mHTT, Neurofilament light chain (NfL), and 24-OH cholesterol at Week 52, Change from baseline in Magnetic Resonance Spectroscopy (MRS) metabolic profile (measuring lactate, N-acetyl aspartate, creatine, choline, glutamate, glutamine, glutathione, taurine, and myo-inositol) at Week 52, Change from baseline in Positron Emission Tomography (PET) Fluoro-deoxyglucose (FDG) striatal profile at Week 52	—

Measure

Time frame

Magnitude and variability of change from baseline in anatomical and volumetric measures of brain regions impacted by HD (caudate, putamen, whole brain volume, grey matter volume, white matter volume, ventricular volume) as assessed by MRI at Week 52, Change from baseline in the Unified Huntington’s Disease Rating Scale composite score [cUHDRS: Total Motor Score (TMS), Total Functional Capacity (TFC), Symbol Digit Modalities Test (SDMT) and Stroop Word Reading (SWRT)] at Week 52, Change from baseline in blood and cerebrospinal fluid (CSF) mHTT, Neurofilament light chain (NfL), and 24-OH cholesterol at Week 52, Change from baseline in Magnetic Resonance Spectroscopy (MRS) metabolic profile (measuring lactate, N-acetyl aspartate, creatine, choline, glutamate, glutamine, glutathione, taurine, and myo-inositol) at Week 52, Change from baseline in Positron Emission Tomography (PET) Fluoro-deoxyglucose (FDG) striatal profile at Week 52

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Countries

France

Outcome results

None listed