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ATR-101

Phase 2

Congenital Adrenal Hyperplasia | Small molecule | Endocrine |Tempest Therapeutics, Inc.|Last Updated: Mar 10, 2021

Development status

Highest phase Phase 2
Registered trials 3 across 2 sponsors since Jul 2013

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment10

FDA Designations

No designations recorded

Clinical trial landscape

ATR-101 · 2 trials · 4 indications

Phase 2 1Phase 1 1
NCT02804178A Study of ATR-101 for the Treatment of Congenital Adrenal HyperplasiaCongenital Adrenal Hyperplasia
COMPLETED10 Analytics
PHASE2COMPLETED
A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Congenital Adrenal HyperplasiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.

17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.

Frequency of dose-limiting toxicity and determination of maximum tolerated dose
Occurrence of DLT at 28 days

Adverse events will be recorded and tabulated by grade and system organ class according to CTCAE v 4.03. Laboratory measures and ECGs will be assessed.

Secondary Endpoints

Area under the plasma concentration versus time curve (AUC) of ATR-101
Day 1 and Day 22
Change in plasma cortisol levels
Baseline and day 22
Change in objective measurement of tumor size
Baseline and 8 weeks
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ATR-101EXPERIMENTALAscending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.

Interventions

NameTypeDescription
ATR-101DRUG125-1000 mg twice per week
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Eligibility Criteria

Age Range18 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: * Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone * Biochemical marker of diseas...

Countries:United StatesGermany
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Frequently asked questions about ATR-101

What is ATR-101 used for?

ATR-101 is an investigational small molecule being studied for the treatment of adrenocortical carcinoma and congenital adrenal hyperplasia. It has been evaluated in clinical trials for these conditions, though it is not yet approved and remains in clinical development.

Who makes ATR-101?

ATR-101 is being developed by Tempest Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol TPST. The company has conducted clinical trials of ATR-101 in the United States and Germany.

What phase is ATR-101 in?

ATR-101 has completed a Phase 1 trial for adrenocortical carcinoma and a Phase 2 trial for congenital adrenal hyperplasia. Both trials are completed, and the drug is investigational, meaning it is not yet approved by regulatory authorities.

What clinical trials is ATR-101 in?

ATR-101 has been studied in two completed trials: NCT01898715, a Phase 1 study in advanced adrenocortical carcinoma with 63 participants, and NCT02804178, a Phase 2 study in congenital adrenal hyperplasia with 10 participants. Both trials were conducted in the United States, with the first also including Germany.

Is ATR-101 the same as any other drug?

ATR-101 is the primary name used for this investigational drug in clinical trials. No alternative names have been reported for this compound in the available clinical trial information.