Phase 1 Study of AT-02 in Healthy Volunteers and Subjects with Systemic Amyloidosis

• Research type

  Research Study

• Full title

  A Three-part, Phase 1, Single-ascending and Multiple-ascending Dose Escalation Study in Healthy Volunteers and Subjects with Systemic Amyloidosis to Assess the Safety, Tolerability, and Pharmacokinetics of "AT 02"

• IRAS ID

  1007521

• Contact name

  Scott Stephens

• Contact email

  Sstephens@attralus.com

• Sponsor organisation

  Attralus, Inc.

• Clinicaltrials.gov Identifier

  NCT05521022

• Research summary

  This is a multicenter, international, three-part, Phase 1 study designed to evaluate the safety, tolerability, and PK of rising single doses of AT-02 in healthy volunteers and in subjects with systemic amyloidosis and to assess the safety, tolerability, PK, and clinical activity of multiple doses of AT-02 in subjects with systemic amyloidosis. Study AT02-001 includes the 3 parts. Only Part 3 will be conducted in the UK: an open label, multiple-ascending dose, dose escalation study in subjects with systemic amyloidosis to assess the safety, tolerability, PK, PD, and clinical activity of AT-02. The participants who will take part have to be male or female ≥18 and ≤85 years of age. The total participant duration is approximately 7 months. All Part 3 dose escalation will proceed with a 3+3 design. Part 3 dose levels will be determined by the SRC. There will be Groups 1 and 2 dosed over a 4-week treatment period with different dosing schedules:
  Group 1: Weekly Dosing for 4 Weeks; Group 2: Dosing on Day 1 and Week 4 Visits; Group 2: Optional Dose Titration Cohort
  Expansion of Cohorts
  Up to a total of 5 cohorts may be included in Part 3. Total enrollment will be up to 150 systemic amyloidosis subjects in this study (and the AT02-003 OLE). AL subjects may only be enrolled in Group 1 Cohort 1 and the cohort is limited to 5 AL subjects. All subsequent cohorts will be restricted to ATTR subjects with up to 5 ATTRv subjects in each cohort.
  Part 3 subjects may enter OLE (AT02-003) study by initiating dosing in the OLE at the Part 3 Week 8 visit.
  Once dosing is initiated in the OLE, participation in the AT02-001 study is concluded and the Week 8 visit will be the EOS visit for AT02-001 Part 3.
  Subjects who do not enter the OLE will complete all visits in this study as outlined in the Schedule of Activities.

• REC name

  HSC REC B

• REC reference

  23/NI/0111

• Date of REC Opinion

  2 Nov 2023

• REC opinion

  Further Information Favourable Opinion