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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT01463215 |
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Date of registration:
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27/10/2011 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease
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Scientific title:
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An Open-Label, Dose Escalation With 2 Dose Levels, Proof-of-Concept Clinical Trial of Ambroxol for the Treatment of Type I Gaucher Disease |
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Date of first enrolment:
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December 2012 |
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Target sample size:
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20 |
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Recruitment status: |
Suspended |
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URL:
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http://clinicaltrials.gov/show/NCT01463215 |
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Study type:
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Interventional |
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Study design:
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Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Robert Johnston |
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Address:
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Telephone:
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Email:
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Affiliation:
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Exsar Corporation |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Biochemically and genetically confirmed diagnosis of Gaucher disease caused by
ß-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.
Age minimum:
16 Years
Age maximum:
N/A
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Type I Gaucher Disease
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Intervention(s)
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Drug: Ambroxol
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Primary Outcome(s)
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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.]
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Secondary Outcome(s)
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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.]
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Secondary ID(s)
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CL-Ambroxol-002
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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