AT-007 for Abetalipoproteinemia

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Hassman Research Institute, Berlin, NJ
Abetalipoproteinemia
AT-007 - Drug
Eligibility
18 - 65
All Sexes
What conditions do you have?
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Study Summary

This study is designed to assess the efficacy and safety of AT-007 treatment in patients with SORD Deficiency. This randomized, double-blind study will assess the effect of AT-007 compared to Placebo in SORD Deficiency patients for 24 months.

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

4 Primary · 0 Secondary · Reporting Duration: Baseline and up to month 24

Month 24
CMT-Fom (Charcot Marie Tooth Functional Outcome Measure)
blood sorbitol levels
Month 24
Muscle MRI (Magnetic Resonance Imaging)
Month 24
10-meter walk-run test (10MWRT).

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

2 Treatment Groups

AT-007
1 of 2
Placebo
1 of 2
Active Control
Non-Treatment Group

72 Total Participants · 2 Treatment Groups

Primary Treatment: AT-007 · Has Placebo Group · Phase 2 & 3

AT-007
Drug
ActiveComparator Group · 1 Intervention: AT-007 · Intervention Types: Drug
Placebo
Drug
ShamComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline and up to month 24
Closest Location: Hassman Research Institute · Berlin, NJ
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2017First Recorded Clinical Trial
0 TrialsResearching Abetalipoproteinemia
97 CompletedClinical Trials

Who is running the clinical trial?

Applied Therapeutics, Inc.Lead Sponsor
4 Previous Clinical Trials
829 Total Patients Enrolled
Michael E Shy, MDStudy ChairUniversity of Iowa
4 Previous Clinical Trials
7,113 Total Patients Enrolled

Eligibility Criteria

Age 18 - 65 · All Participants · 5 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are male and are unable to procreate or agree to use an acceptable form of birth control.
You are willing and able to provide signed and dated informed consent prior to any study-related procedures and willing and able to comply with all study procedures.
You are between the ages of 18 and 55 years, inclusive.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.