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ALN-AT3SC

Phase 1

Hemophilia A | Small molecule | Hematology |Sanofi|Last Updated: Dec 19, 2020

Success Probability

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Market & Valuation

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Trial Design

RandomizedPLACEBO_CONTROLLED
Total Trials1
Total Enrollment51

FDA Designations

No designations recorded

Clinical trial landscape

ALN-AT3SC · 1 trial · 2 indications

Phase 1 1
NCT02035605A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B PatientsHemophilia A
COMPLETED51 Analytics
PHASE1COMPLETED
A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients
Hemophilia AUnlock trial analytics

Study Endpoints

Primary Endpoints

The safety of ALN-AT3SC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicities (DLTs), and AEs leading to study drug discontinuation.
Part A (SAD phase): through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112

Secondary Endpoints

The pharmacokinetics (PK) of ALN-AT3SC as characterized by plasma PK profiles and urine samples.
Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma AT levels.
Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma TG.
Part A (SAD) phase: through day 56; Part B (MAD) phase: through Day 70; Part C (MD Phase) through Day 112; Part D (MD Phase in patients with inhibitors) through Day 112
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Study Design & Arms

AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALN-AT3SCACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -

Interventions

NameTypeDescription
ALN-AT3SCDRUGAscending doses of ALN-AT3SC by subcutaneous (sc) injection
Sterile Normal Saline (0.9% NaCl)DRUGCalculated volume to match active comparator
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Eligibility Criteria

Age Range18 Years to 65 Years
SexMALE
Healthy VolunteersYes
Study Sites14

Inclusion Criteria: Part A (SAD phase) inclusion: * Healthy adult males aged 18 to 40 years inclusive at Screening. * Subjects with adequate complete blood counts and liver function tests. * Willing to provide written informed consent and willing to comply with study requirements. Part B \& C (MA...

Countries:United StatesBulgariaRussiaSwitzerlandUnited Kingdom
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Frequently asked questions about ALN-AT3SC

What is ALN-AT3SC used for in hemophilia A?

ALN-AT3SC is an investigational small molecule being studied for the treatment of hemophilia A. It is in Phase 1 clinical development, and a completed trial evaluated the drug in healthy volunteers and in patients with hemophilia A or B. The study was placebo-controlled and enrolled 51 male participants aged 18 years and older.

Who is developing ALN-AT3SC?

Sanofi (ticker: SNY) is developing ALN-AT3SC. The drug is currently in Phase 1 clinical development for hemophilia A. A Phase 1 trial of ALN-AT3SC has been completed, with the study conducted at sites in the United States, Bulgaria, Russia, Switzerland, and the United Kingdom.

What phase is ALN-AT3SC in?

ALN-AT3SC is in Phase 1 clinical development. It is an investigational drug and has not been approved. One Phase 1 trial, NCT02035605, has been completed. This trial enrolled 51 participants and was a randomized, placebo-controlled study in healthy volunteers and patients with hemophilia A or B.

What clinical trials is ALN-AT3SC in?

ALN-AT3SC has one completed clinical trial, NCT02035605, titled "A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients." The trial enrolled 51 male participants aged 18 years and older, was placebo-controlled, and took place in the United States, Bulgaria, Russia, Switzerland, and the United Kingdom.

Is ALN-AT3SC the same as any other drug?

ALN-AT3SC is the drug name used in clinical development. No alternative names have been reported for this investigational therapy. It is being studied for hemophilia A, and the completed Phase 1 trial also included patients with hemophilia B.