Back to resultsClinical Trial of Ambroxol in Patients With Type I Gaucher Disease
Primary Purpose
Type I Gaucher Disease
Status
Suspended
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Ambroxol
Sponsored by
About this trial
This is an interventional treatment trial for Type I Gaucher Disease focused on measuring Ambroxol, Type I Gaucher Disease
Eligibility Criteria
Inclusion Criteria:
- 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).
Exclusion Criteria:
- 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.
- A history of cancer of any type.
- Patients who have received immunotherapy of any type within the past 4 weeks prior to study initiation.
- Any condition or abnormality which may, in the opinion of the investigator, compromise the safety of patients.
Sites / Locations
- ExSAR Corporation
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ambroxol
Arm Description
Ambroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
efficacy based on biomarker (glucocerebrosidase activities), lab results, as well as hepatic and splenic volumes from imaging scans.
Efficacy is based on biomarker (glucocerebrosidase activities), phenotypes according to specific lab results (acid phosphatase, angiotensin-converting enzyme, serum bilirubin, hemoglobin, platelet counts, peripheral blood leukocyte counts, serum iron, clotting time, etc.), as well as hepatic and splenic volumes.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01463215
Brief Title
Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease
Official Title
An Open-Label, Dose Escalation With 2 Dose Levels, Proof-of-Concept Clinical Trial of Ambroxol for the Treatment of Type I Gaucher Disease
Study Type
Interventional
2. Study Status
Record Verification Date
February 2012
Overall Recruitment Status
Suspended
Study Start Date
December 2012 (undefined)
Primary Completion Date
December 2014 (Anticipated)
Study Completion Date
August 2015 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Exsar Corporation
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
Ambroxol is expected to improve the signs and symptoms of patients with Type I Gaucher Disease.
Detailed Description
This is an Open-Label, Dose Escalation with 2 Dose Levels, Proof-of-Concept Clinical Trial of Ambroxol for the Treatment of Patients with Type I Gaucher Disease.
This study is a randomized clinical trial involving 20 evaluable patients affected with Type 1 Gaucher disease who are responsive to Ambroxol in vitro. There are 2 treatment groups, involving 2 dose levels of Ambroxol (187.5 and 225 mg/day), given once daily PO for 2 months in both groups. The 187.5-mg/day dose level will be tested first. If there are no significant adverse events, defined as >Grade 3 toxicity according to the latest version of the National Cancer Institute (NCI) Common Toxicity Criteria (CTC), the 225-mg/day dose level will be tested.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Type I Gaucher Disease
Keywords
Ambroxol, Type I Gaucher Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Ambroxol
Arm Type
Experimental
Arm Description
Ambroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months.
Intervention Type
Drug
Intervention Name(s)
Ambroxol
Other Intervention Name(s)
Mucosolvon®
Intervention Description
Ambroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months.
Primary Outcome Measure Information:
Title
Safety assessment based on potential changes in physical exam, vital signs, ECG, adverse event query, and clinical lab results, when compared to baseline values.
Description
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.
Time Frame
Safety will be assessed at baseline and biweekly for 2 months.
Secondary Outcome Measure Information:
Title
efficacy based on biomarker (glucocerebrosidase activities), lab results, as well as hepatic and splenic volumes from imaging scans.
Description
Efficacy is based on biomarker (glucocerebrosidase activities), phenotypes according to specific lab results (acid phosphatase, angiotensin-converting enzyme, serum bilirubin, hemoglobin, platelet counts, peripheral blood leukocyte counts, serum iron, clotting time, etc.), as well as hepatic and splenic volumes.
Time Frame
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.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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).
Exclusion Criteria:
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.
A history of cancer of any type.
Patients who have received immunotherapy of any type within the past 4 weeks prior to study initiation.
Any condition or abnormality which may, in the opinion of the investigator, compromise the safety of patients.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert Johnston
Organizational Affiliation
Exsar Corporation
Official's Role
Study Chair
Facility Information:
Facility Name
ExSAR Corporation
City
Monmouth Junction
State/Province
New Jersey
ZIP/Postal Code
08852
Country
United States
12. IPD Sharing Statement
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Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease
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