Status:

RECRUITING

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN)

Lead Sponsor:

Denali Therapeutics Inc.

Collaborating Sponsors:

Takeda

Conditions:

Frontotemporal Dementia

Eligibility:

All Genders

18-80 years

Phase:

PHASE1

PHASE2

Brief Summary

This is a Phase 1/2, multicenter, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses...

Eligibility Criteria

Inclusion

  • Key
  • Part A:
  • Women of non-childbearing potential (surgically sterilized or post menopausal) or men, aged ≥18 to ≤ 55 years
  • BMI of ≥ 18 to ≤ 32 kg/m²
  • When engaging in sex with a woman of child bearing potential, two forms of birth control are required
  • Part B:
  • Women of non-childbearing potential (surgically sterilized or post menopausal) or men, aged ≥18 to ≤ 80 years. Women who are of childbearing potential but on highly effective, low user dependent contraceptive methods will be allowed.
  • BMI of ≥ 18 to ≤ 32 kg/m²
  • Have a Clinical Dementia Rating® plus National Alzheimer's Coordinating Center frontotemporal lobar degeneration global score ≥ 0.5
  • Have confirmed granulin (GRN) mutation via genetic testing or historical records available for review by investigator
  • When engaging in sex with a woman of child bearing potential, both the male participant and his female partner must use highly effective contraception
  • Part C:
  • All participants who completed Part B of this trial are eligible for an 18-month OLE if the participant has no unresolved clinically significant TEAEs, where continued dosing may represent a risk to participant safety.
  • Key

Exclusion

  • Have any history of clinically significant neurologic, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematologic, immunologic, or allergic disease, or other major disorders
  • Have a history of malignancy, except fully resected basal cell carcinoma or other malignancies at low risk of recurrence
  • Have a clinically significant history of stroke, cognitive impairment due to causes other than FTD, seizure within 5 years of screening, or head trauma with loss of consciousness within 2 years of screening
  • Have a positive serum pregnancy test or are currently lactating or breastfeeding

Key Trial Info

Start Date :

February 1 2022

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

November 1 2028

Estimated Enrollment :

106 Patients enrolled

Trial Details

Trial ID

NCT05262023

Start Date

February 1 2022

End Date

November 1 2028

Last Update

September 15 2025

Active Locations (32)

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Page 1 of 8 (32 locations)

1

University of California San Francisco

San Francisco, California, United States, 94143

2

John Hopkins University

Baltimore, Maryland, United States, 21218

3

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

4

University of Antwerp

Antwerp, Belgium