Phase 2
N=6
Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia
Classic Galactosemia
Bottom Line
View on ClinicalTrials.gov: NCT04117711 ↗Enrolled (actual)
6
Serious AEs
0.9%
Results posted
May 2024
Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events — 1; 0; 0; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AT-007 (Drug); Placebo (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Applied Therapeutics, Inc.
- Primary completion
- Dec 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-emergent Adverse Events |
7; 1; 2 | — |
| PRIMARY Number of Participants With Treatment-emergent Adverse Events |
7; 1; 2 | — |
| PRIMARY Number of Participants With Treatment-emergent Adverse Events |
7; 1; 2 | — |
| PRIMARY Number of Participants With Treatment-emergent Adverse Events |
7; 1; 2 | — |
| SECONDARY Cmax of AT-007 |
1.53; 8.92; 15.2; 27; 57.4; 15.2 | — |
| SECONDARY Tmax of AT-007 |
4.00; 4.00; 4.00; 4.00; 4.00; 4.00 | — |
| SECONDARY t1/2 of AT-007 |
13.7; 22.0; 10.3; 8.42; 8.2; 13.1 | — |
| SECONDARY AUClast of AT-007 |
12.6; 89.0; 158; 352; 624; 181 | — |
| SECONDARY AUCinf of AT-007 |
14.3; 81.8; 169; 163; 655; 109 | — |
| SECONDARY Maximal Galactitol Reduction in Plasma |
-475; -1175; -1259; -393; -974.4; -344.0 | — |
| SECONDARY Galactose Concentration in Plasma |
8.94; -27.8; 25.4; 39.8; -153.78; 52.29 | — |
| SECONDARY Galactose-1-Phosphate (Gal-1p) Concentration in Plasma |
5.71; 31.5; 7.86; 25.2; 33.1; 16.04 | — |
Summary
This study is a first-in-human, randomized, placebo-controlled, 4-Part, single ascending dose (SAD) and multiple ascending dose (MAD) study in healthy adult subjects and adult subjects with Classic Galactosemia.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of Classic Galactosemia confirmed by evidence of absent or significantly decreased ( 100 mmol/mol creatinine
- Galactose-restricted diet
Exclusion Criteria
- Complications of CG resulting in disability that, in the opinion of the Investigator, may prevent the subject from completing all study requirements (e.g., severe neurological deficits, severe cognitive impairment, or severe language difficulty).
- Renal disease (eGFR < 90 mL/min/1.73 m2 or albuminuria).
Data sourced from ClinicalTrials.gov (NCT04117711). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.