Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy

PHASE2CompletedINTERVENTIONAL
Enrollment

40

Participants

Timeline

Start Date

March 1, 2019

Primary Completion Date

November 30, 2022

Study Completion Date

December 30, 2022

Conditions
Gaucher Disease, Type 1
Interventions
DRUG

Ambroxol

"Ambroxol Hydrochloride therapy will be dosed up to 600 mg/day divided to twice a day starting 150 mg for the first month, 300 mg for the following month and 600 mg for the following month.~The study was conducted in accordance with the provisions of the Declaration of Helsinki, Good Clinical Practice guidelines, and local laws and regulations."

Trial Locations (1)

Unknown

Shaare Zedek Medical Center, Jerusalem

All Listed Sponsors
lead

Shaare Zedek Medical Center

OTHER

NCT03950050 - Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy | Biotech Hunter | Biotech Hunter