Vamorolone

Generic Name: Vamorolone

Brand Names: Agamree

Drug Type: Small Molecule

Chemical Formula: C_₂₂H_₂₈O_₄

CAS Number: 13209-41-1

Unique Ingredient Identifier: 8XP29XMB43

Background: Vamorolone is under investigation in clinical trial NCT03439670 (A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)).

Indication: Vamorolone is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients ≥2 years of age.

Associated Conditions: Duchenne Muscular Dystrophy (DMD)

Clinical Trials

A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy

Phase 4

Recruiting

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: vamorolone 40 mg/mL oral suspension

First Posted Date: 2024-12-03

Last Posted Date: 2024-12-03

Lead Sponsor: Santhera Pharmaceuticals

Target Recruit Count: 80

Registration Number: NCT06713135

Locations: 🇧🇪
UZ Gent (Universitair Ziekenhuis Gent), Gent, Belgium
🇧🇪
UZ Leuven (Universitair Ziekenhuis Leuven), Leuven, Belgium
🇨🇿
University Hospital Brno, Brno, Czechia

and more 9 locations

Evaluation of Vamorolone CYP3A4 Induction on Midazolam (a Sensitive CYP 3A4 Substrate) Pharmacokinetics

Phase 1

Completed

Conditions: Drug Interaction Potentiation

Interventions: Drug: Vamorolone
Drug: Midazolam

First Posted Date: 2024-11-14

Last Posted Date: 2024-11-14

Lead Sponsor: Santhera Pharmaceuticals

Target Recruit Count: 18

Registration Number: NCT06689527

Locations: 🇩🇪
Nuvisan GmbH, Neu-Ulm, Germany

Evaluation of Vamorolone Mineralocorticoid Receptor Antagonism in Healthy Subjects

Phase 1

Completed

Conditions: Pharmacodynamic

Interventions: Drug: Fludrocortisone
Drug: Vamorolone
Drug: Eplerenone

First Posted Date: 2024-10-18

Last Posted Date: 2024-10-18

Lead Sponsor: Santhera Pharmaceuticals

Target Recruit Count: 30

Registration Number: NCT06649409

Locations: 🇩🇪
Nuvisan GmbH, Neu-Ulm, Germany

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.

Recruiting

Conditions: Duchenne Muscular Dystrophy

First Posted Date: 2024-08-21

Last Posted Date: 2025-05-20

Lead Sponsor: Catalyst Pharmaceuticals, Inc.

Target Recruit Count: 250

Registration Number: NCT06564974

Locations: 🇺🇸
Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸
Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸
Stanford University, Palo Alto, California, United States

and more 21 locations

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Phase 2

Completed

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: Vamorolone

First Posted Date: 2022-01-11

Last Posted Date: 2024-10-16

Lead Sponsor: Santhera Pharmaceuticals

Target Recruit Count: 54

Registration Number: NCT05185622

Locations: 🇨🇦
Alberta's Children Hospital, Calgary, Alberta, Canada
🇨🇦
Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada
🇨🇦
The Hospital for Sick Children, Toronto, Ontario, Canada

and more 2 locations

A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)

Phase 2

Active, not recruiting

Conditions: Becker Muscular Dystrophy

Interventions: Drug: Vamorolone
Drug: Placebo

First Posted Date: 2021-12-21

Last Posted Date: 2024-12-27

Lead Sponsor: ReveraGen BioPharma, Inc.

Target Recruit Count: 39

Registration Number: NCT05166109

Locations: 🇺🇸
University of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇮🇹
Azienda Ospedale Universita Padova, Padova, Italy

Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis

Phase 1

Withdrawn

Conditions: Pediatric Ulcerative Colitis

Interventions: Drug: Vamorolone 4% suspension for oral dosing

First Posted Date: 2020-04-16

Last Posted Date: 2020-09-29

Lead Sponsor: ReveraGen BioPharma, Inc.

Registration Number: NCT04348890

Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Conditions: Duchenne Muscular Dystrophy

First Posted Date: 2019-03-05

Last Posted Date: 2025-02-10

Lead Sponsor: Santhera Pharmaceuticals

Registration Number: NCT03863119

Locations: 🇺🇸
University of California Davis, Davis, California, United States
🇺🇸
Duke University, Durham, North Carolina, United States
🇺🇸
urie Children's Hospital of Chicago, Chicago, Illinois, United States

and more 3 locations

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Phase 2

Completed

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: Vamorolone
Other: Placebo
Drug: Prednisone

First Posted Date: 2018-02-20

Last Posted Date: 2023-03-09

Lead Sponsor: ReveraGen BioPharma, Inc.

Target Recruit Count: 121

Registration Number: NCT03439670

Locations: 🇨🇦
Montreal Children's Hospital, Montréal, Quebec, Canada
🇧🇪
Ghent University Hospital, Ghent, Belgium
🇺🇸
University of Texas Southwestern Medical Center, Dallas, Texas, United States

and more 30 locations

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Phase 2

Completed

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: Vamorolone 0.25 mg/day/day
Drug: Vamorolone 0.75 mg/day/day
Drug: Vamorolone 2.0 mg/day/day
Drug: Vamorolone 6.0 mg/day/day

First Posted Date: 2017-01-31

Last Posted Date: 2021-05-20

Lead Sponsor: ReveraGen BioPharma, Inc.

Target Recruit Count: 46

Registration Number: NCT03038399

Locations: 🇨🇦
Alberta Children's Hospital, Calgary, Alberta, Canada
🇺🇸
Duke University, Durham, North Carolina, United States
🇺🇸
Nemours Children's Hospital, Orlando, Florida, United States

and more 9 locations

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