Clinical Trial: A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Brief Summary: This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH). Treatment duration will range from a minimum of approximately 2 months to 6 months per subject. A subject may receive a minimum of one dose level or up to a maximum of 5 dose levels, in sequentially increasing dose strengths. Each dose level will last 28 days.
Detailed Summary:
Sponsor: Millendo Therapeutics, Inc.
Current Primary Outcome: Reduction of 17-OHP to </= 2X ULN [ Time Frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels. ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Number of participants with abnormal laboratory values and/or adverse events that are related to treatment [ Time Frame: Baseline, Day 1 and Day 15 of each dose level. Each subject will have up to 5 dose levels. ]Safety evaluations will include adverse events, vital signs, physical examinations, laboratory measures and ECGs.
- Pharmacokinetics: Area under the curve [ Time Frame: Evaluated at Day 1 (pre-dose, 1, 2, 4 hours post-dose) and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]
- Pharmacokinetics: Maximum plasma concentration [ Time Frame: Evaluated at Day 1 (predose, 1, 2, 4, hours post-dose and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]
- Pharmacokinetics: Time to maximum concentration [ Time Frame: Evaluated at Day 1 (pre-dose, 1, 2, 4, hours post-dose) and Day 15 (pre-dose) of each dose level. Each subject will have up to 5 dose levels. Dose levels begin with 125 mg BID and increase up to 1000 mg BID. ]
Original Secondary Outcome: Same as current
Information By: Millendo Therapeutics, Inc.
Dates:
Date Received: June 1, 2016
Date Started: May 2016
Date Completion: September 2017
Last Updated: December 20, 2016
Last Verified: December 2016