A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT05517603|
Recruitment Status : Recruiting
First Posted : August 26, 2022
Last Update Posted : August 7, 2023
|Condition or disease||Intervention/treatment||Phase|
|Spinal and Bulbar Muscular Atrophy Kennedy's Disease||Drug: AJ201 Drug: Placebo||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Phase 1/2a, Randomized, Double-Blind, Placebo-Controlled, First-In-Patient Study Of AJ201 To Evaluate Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics In Adults With Spinal And Bulbar Muscular Atrophy (SBMA)|
|Actual Study Start Date :||February 28, 2023|
|Estimated Primary Completion Date :||September 30, 2023|
|Estimated Study Completion Date :||December 31, 2023|
Active Comparator: Experimental: AJ201
Subjects taking active drug AJ201 600mg/day for 12 weeks.
Other Name: JM17
Placebo Comparator: Placebo Comparator
Subjects taking placebo for 12 weeks.
- Incidence and proportion of subjects with AEs including SAEs and TEAEs. [ Time Frame: 12 weeks ]Safety will be monitored throughout the study.
- Pharmacokinetics: Maximum Plasma Concentration (Cmax) will be assessed [ Time Frame: Visit 3/Week 2 and Visit 4/Week 6 at the following time points: predose and 0.5, 1, 2, 4, 8, and 12 hours postdose. ]Blood sampling will be collected at Visit 3 and Visit 4.
- Pharmacokinetics: Area Under the Curve (AUC) will be assessed [ Time Frame: Visit 3/Week 2 and Visit 4/Week 6 at the following time points: predose and 0.5, 1, 2, 4, 8, and 12 hours postdose. ]Blood sampling will be collected at Visit 3 and Visit 4.
- Pharmacodynamics: Change from baseline in mutant androgen receptor protein levels in skeletal muscle in treatment vs placebo group. [ Time Frame: Visit 2/Week 1 and Visit 5/Week 12 ]Samples will be collected at Visit 2 and Visit 5.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05517603
|Contact: Andy Chen, PhDfirstname.lastname@example.org|
|United States, California|
|University of California, Irvine||Recruiting|
|Orange, California, United States, 92868|
|Contact: Pola Gaid 714-456-6191 email@example.com|
|Principal Investigator: Tahseen Mozaffar, MD|
|Palo Alto, California, United States, 94304|
|Contact: Emily Lien 650-407-7912 firstname.lastname@example.org|
|Principal Investigator: John Day, MD|
|United States, Florida|
|Jacksonville, Florida, United States, 32224|
|Contact: Megan Donahue 904-953-3647 email@example.com|
|Principal Investigator: Bjorn Oskarsson, MD|
|United States, Maryland|
|National Institutes of Health||Recruiting|
|Bethesda, Maryland, United States, 20814|
|Contact: Angela Kokkinis 301-451-8146 firstname.lastname@example.org|
|Principal Investigator: Christopher Grunseich, MD|
|United States, Minnesota|
|Mayo Clinic||Not yet recruiting|
|Rochester, Minnesota, United States, 55905|
|Contact: Jane L. Sultze 507-538-5523 email@example.com|
|Principal Investigator: Eric Sorenson, MD|
|United States, Missouri|
|Washington University in St. Louis||Recruiting|
|Saint Louis, Missouri, United States, 63110|
|Contact: Oliver Doerr 314-362-1626 firstname.lastname@example.org|
|Contact: Jennifer Koniak (314) 362-1626 email@example.com|
|Principal Investigator: Alan Pestronk, MD|