Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease

NCT01463215 | Suspended Phase 1

• 1 other identifier: CL-Ambroxol-002
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Ambroxol is expected to improve the signs and symptoms of patients with Type I Gaucher Disease.

Conditions

Type I Gaucher Disease

Keywords

Ambroxol; Type I Gaucher Disease

Outcome Measures

Primary Outcomes (1)

• Safety assessment based on potential changes in physical exam, vital signs, ECG, adverse event query, and clinical lab results, when compared to baseline values.

  Safety will be based on physical exam, vital signs, ECG, adverse event query, and clinical pathology (includes chemistry, hematology and coagulation), asessed at baseline and approximately biweekly during the study.

  Safety will be assessed at baseline and biweekly for 2 months.

Secondary Outcomes (1)

• efficacy based on biomarker (glucocerebrosidase activities), lab results, as well as hepatic and splenic volumes from imaging scans.

  Biomarker, lab results (phenotype), as well as hepatic and spenic volumes will be assessed at baseline and after 2 months of treatment, and lab results (phenotypes) will also be assessed biweekly during the 2-month treatment period.

Study Arms (1)

Ambroxol

EXPERIMENTAL

Ambroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months.

Drug: Ambroxol

Interventions

Ambroxol DRUG

Ambroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months.

Also known as: Mucosolvon®

Ambroxol

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Biochemically and genetically confirmed diagnosis of Gaucher disease caused by β-glucocerebrosidase deficiency resulting from mutations in the GBA genes, which have been shown to respond to Ambroxol according to in vitro screening assay.
• Must be 16 years of age or older at the time of study initiation.
• With an intact, enlarged spleen.
• A hemoglobin level of at least 10 g/L.
• Able to understand and cooperate with the requirements of the study protocol.
• Mentally competent, have ability to understand and willingness to sign the informed consent form.
• Able to travel to a participating study site.
• Women of child-bearing potential must use accepted contraceptive methods, and must have a negative serum or urine pregnancy test within one week prior to treatment initiation. An additional pregnancy test is to be performed, and results obtained, prior to administration of the first dose of Ambroxol.
• Fertile men must practice effective contraceptive methods during the study period, unless documentation of infertility exists.
• Body weight \>40 kg (88 lbs).

You may not qualify if:

• Receipt of any form of glucocerebrosidase \<\<4 weeks prior study initiation.
• Total splenectomy.
• Serious medical illness, significant cardiac disease, chronic bronchitis, emphysema, and cystic fibrosis, as well as disorders causing ventilation perfusion mismatch.
• Substance abuse.
• Any complex disease that may confound treatment assessment.
• Pregnant women, or women of child-bearing potential not using reliable means of contraception.
• Lactating females because of the potential for adverse reactions in nursing infants.
• Fertile men unwilling to practice contraceptive methods during the study period.
• Unwilling or unable to follow protocol requirements.
• Known hypersensitivity reactions, intolerance or adverse reactions to Ambroxol or to the inactive ingredients.
• Evidence of systemic infection, or serious infection within the past month.
• Known to have HIV infection.
• Known to have hepatitis B or hepatitis C.
• Patients with a history of convulsive disorders.
• Patients receiving any other investigational treatment for any indication within the past 4 weeks prior to initiation of Ambroxol treatment.

Sponsors & Collaborators

• Exsar Corporation: lead

Study Sites (1)

ExSAR Corporation

Monmouth Junction, New Jersey, 08852, United States

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

Ambroxol

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Bromhexine; Aniline Compounds; Amines; Organic Chemicals; Cyclohexylamines

Study Officials

• Robert Johnston

  Exsar Corporation

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 27, 2011
• First Posted: November 1, 2011
• Study Start: December 1, 2012
• Primary Completion: December 1, 2014
• Study Completion: August 1, 2015
• Last Updated: February 15, 2013

Record last verified: 2012-02

Locations

US (1)