CompletedEarly Phase 1

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

United States, Canada46 participantsStarted 2023-10-19

Plain-language summary

Study ABX1100-1001 is a first-in-human (FIH), phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a single ascending dose (SAD) and multiple doses (MD) of ABX1100 administered intravenously to healthy participants and patients with LOPD. * Part A features a SAD study with a double-blind, placebo-controlled, randomized design in NHVs involving 3 cohorts (A1-A3). This Part also includes a single dose, open-labeled cohort (A4) in NHVs which will commence after cohorts A1-3. * Part B is a MD, double-blind, placebo-controlled, randomized design in NHVs. The MD Part B will commence after completion of Cohorts A1, A2 and A3 in the SAD Part A and SRC review of these 3 cohorts. * Part C opened for enrollment after the Safety Review Committee (SRC) review of safety, PK and PD data from both Part A and Part B. Part C is a MD, open-label design in patients with Late-Onset Pompe Disease.

Who can participate

Age range18 Years

SexALL

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Parts A-B Inclusion Criteria: * Body mass index (BMI) between 18.0 and 32.0 kg/m2, inclusive and weight between 50 and 90 kg, inclusive. * Agree not to have a tattoo or body piercing until the end of the study. * Agree not to receive COVID-19 vaccination from 7 days prior to first study drug administration until at least 7 days after the last study drug administration. * Agree not to receive a vaccination (live attenuated vaccine) during the study and until 60 days after the study has ended (last study procedure). * Willing to undergo needle muscle biopsies. * Willing to avoid strenuous activities 48 hours before needle muscle biopsy and throughout the study. * Female participants who are sexually active with a non-sterilized partner must be non-pregnant and non-lactating and agree to use a highly effective method of contraception. * Males of childbearing potential must agree to use a highly effective method of contraception with female sexual partners of childbearing potential and not donate sperm during study participation and for 90 days after last administration of study drug ABX1100 or placebo. Parts A-B Exclusion Criteria: * Known history or presence of any clinically significant hepatic, renal/genitourinary, gastrointestinal, cardiovascular, cerebrovascular, pulmonary, endocrine, immunological, musculoskeletal, neurological, psychiatric, dermatological or hematological disease or condition. * History of any inherited or acquired skeletal muscle diseases (for example…

What they're measuring

Number of subjects with treatment-emergent adverse events (TEAEs)

Timeframe: up to 20 weeks

Trial details

NCT IDNCT06109948

SponsorAro Biotherapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-02-13