A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD


About this study

The primary objective of this study is to assess the safety and tolerability of TPN-101 in patients with C9ORF72 amyotrophic lateral sclerosis (ALS)/frontotemporal dementia (FTD).



Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- Have documentation of a clinical genetic test demonstrating the presence of a
confirmed repeat expansion in the C9orf72 gene from a CLIA certified laboratory

- Score ≥ 18 on the Mini-Mental State Exam (MMSE) at Screening

- Have a reliable caregiver to accompany the patient to all study visits.

- For patients with ALS (with or without FTD):

- Diagnosis of ALS (probable, possible, laboratory-supported probable or definite)
according to the World Federation of Neurology revised E1 Escorial criteria

- Onset of weakness within 3 years prior to Screening

- Slow vital capacity (SVC) ≥ 60% of predicted normal adjusted for sex, age, and
height (from the sitting position)

- ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 at Screening

- For patients with FTD:

- A gradual, progressive decline in behavior, language, or motor function
consistent with C9ORF72 hexanucleotide expansion-related syndrome such as
behavioral variant FTD, primary progressive vaphasia, or amnestic syndrome

- CDR Dementia Staging Instrument plus National Alzheimer's Coordinating Center
Behavior and Language Domains (CDR plus NACC FTLD) global score of 0.5-2.0 at

Exclusion Criteria:

- Presence of other significant neurological or psychiatric disorders

- History of significant brain abnormality, including, but not limited to, prior
hemorrhage or infarct, cerebral contusion, encephalomalacia, aneurysm, vascular
malformation, subdural hematoma, hydrocephalus, space-occupying lesion (e.g., abscess
or brain tumor such as meningioma); symptoms or signs of elevated intracranial
pressure, e.g., symptoms or history of head injury or abnormal funduscopic exam. If
there is history or evidence on neurologic exam suggesting possible subdural hematoma
(SDH), patients should be fully evaluated, including magnetic resonance imaging (MRI)
if indicated, to exclude significant, new SDH

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Bradley Boeve, M.D.

Closed for enrollment

Contact information:

Kevin Nelson

(507) 284-9295


More information


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