Type I Gaucher DiseasePhase 1/2NCT01463215
Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease
A Phase 1/2 study testing Ambroxol in people with Type I Gaucher Disease, run by Exsar Corporation at 1 site in United States.
Trial summary
Ambroxol is expected to improve the signs and symptoms of patients with Type I Gaucher Disease. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Type I Gaucher Disease
- What is being tested?
- Ambroxol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2012to Aug 2015
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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).
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
ExSAR Corporation
Monmouth Junction, New Jersey, 08852
About this study
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
Ambroxol is expected to improve the signs and symptoms of patients with Type I Gaucher Disease.
Read the 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.
- Conditions
- Type I Gaucher Disease
- Being tested
- Ambroxol
- Keywords
- Ambroxol, Type I Gaucher Disease
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AmbroxolDrug
Also called Mucosolvon®
Ambroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months.
| Study group | Role | What they receive |
|---|---|---|
| Ambroxol | Experimental | Ambroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months. Receives: Drug: Ambroxol |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
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.
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2011 (estimated)
- Study startDec 2012
- Primary completionDec 2014 (estimated)
- Study completionAug 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type I Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Exsar Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert Johnston, Study chair, Exsar Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01463215
- Sponsor's protocol ID
- CL-Ambroxol-002
- Status last verified
- Feb 2012
- First submitted
- 27 Oct 2011
- Last updated
- 15 Feb 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01463215
- Acronym / sponsor ID
- CL-Ambroxol-002
- Conditions
- Type I Gaucher Disease
- Keywords
- Ambroxol, Type I Gaucher Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Suspended
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Exsar CorporationIndustry (pharma, biotech, devices)
- Study start
- Dec 2012
- Primary completion
- Dec 2014 (estimated)
- Study completion
- Aug 2015 (estimated)
- First posted
- 1 Nov 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ambroxol | Experimental | Drug: AmbroxolAmbroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AmbroxolAlso: Mucosolvon®Ambroxol at a dose level of 187.5 or 225 mg/day will be given once daily by mouth for 2 months. | Drug | Ambroxol |
Primary outcome
Safety assessment based on potential changes in physical exam, vital signs, ECG, adverse event query, and clinical lab results, when compared to baseline values.
Time frame: Safety will be assessed at baseline and biweekly for 2 months.
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.
1 secondary outcome
efficacy based on biomarker (glucocerebrosidase activities), lab results, as well as hepatic and splenic volumes from imaging scans.
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Johnston study chair
Exsar Corporation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Ambroxol in people with Type I Gaucher Disease, run by Exsar Corporation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 15 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety assessment based on potential changes in physical exam, vital signs, ECG, adverse event query, and clinical lab results, when compared to baseline values. (time frame: Safety will be assessed at baseline and biweekly for 2 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Feb 2013.