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Trial record 62 of 414 for:    shaare zedek

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

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ClinicalTrials.gov Identifier: NCT03950050
Recruitment Status : Recruiting
First Posted : May 15, 2019
Last Update Posted : May 15, 2019
Sponsor:
Information provided by (Responsible Party):
Shaare Zedek Medical Center

Brief Summary:

Ambroxol hydrochloride, an over-the-counter antitussive available in many markets , was identified as an interesting pharmacological chaperone. In addition to a mucolytic action, ambroxol has antioxidant and anti-inflammatory properties. Importantly, ambroxol therapy was found safe when given to pregnant women for prevention of neonatal respiratory distress syndrome .

Thus, ambroxol, an oral available drug on the market, may be a safe option for GD patients with potential disease-specific efficacy and should be expanded into a clinical trial using higher doses and placebo-controlled design. The investigators propose to start with a phase II study for patients with type 1 GD and suboptimal response to ERT. In addition the investigators plan to open an international registry of patients with GD currently receiving ambroxol (off study).


Condition or disease Intervention/treatment Phase
Gaucher Disease, Type 1 Drug: Ambroxol Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Intervention Model: Single Group Assignment
Intervention Model Description:

The aim of this single arm, phase II study is to evaluate the efficacy and safety of adding ambroxol to patients with type 1 GD and suboptimal response to ERT.

HYPOTHESIS The addition of ambroxol will improve the disease related symptoms and disease impact of patients with GD with suboptimal response to ERT.

Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
Actual Study Start Date : March 1, 2019
Estimated Primary Completion Date : July 1, 2020
Estimated Study Completion Date : March 1, 2021


Arm Intervention/treatment
Experimental: Ambroxol

Ambroxol 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.

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.

Other Name: Ambroxol Hydrochloride




Primary Outcome Measures :
  1. Platelets count [ Time Frame: 12 months. ]
    Increase in platelet count

  2. bone mineral density evaluated by Dual Energy X-ray Absorptiometry (DEXA) [ Time Frame: 12 months. ]
    Bone Mineral Densitometry (BMD)

  3. Lyso-GB1 biomarker for Gaucher disease [ Time Frame: 12 months ]
    decrease in Lyso-GB1.


Secondary Outcome Measures :
  1. Patient-reported outcomes (PRO) [ Time Frame: 12 months ]
    Improve in PRO from baseline

  2. Fatigue Severity Scale (FSS) [ Time Frame: 12 months ]
    Improve in FSS from baseline



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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

*Adult patients ≥ 18 years with type 1 GD and suboptimal response to ERT defined as one or more than one of the following: platelet count < 100 x 103/mm3 bone mineral density < -2 T score Lyso-GB1 > 200 ng/ml.

*No change in dose or preparation of ERT in the last 12 months (Except for Naive patients)

Exclusion Criteria:

  • Patients with comorbidity that may impact on the primary and/or secondary endpoint.
  • Pregnant women will be excluded from the study.
  • Inability to cooperate with the study procedure
  • Hypersensitivity or any other contraindication listed in the local labeling of ambroxol
  • Refusal of patients to participate in the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03950050


Contacts
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Contact: Joleen Istaiti, B.Sc. +97226555143 joleenist@szmc.org.il
Contact: Ari Zimran, MD +97226555143 azimran@gmail.com

Locations
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Israel
Shaare Zedek Medical Center Recruiting
Jerusalem, Israel
Contact: Joleen Istaiti, BS.c    +97226555143    joleenist@szmc.org.il   
Contact: Ari Zimran, MD    +97226555143    azimran@gmail.com   
Sponsors and Collaborators
Shaare Zedek Medical Center
Investigators
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Principal Investigator: Ari Zimran Ari Zimran - Shaare Zedek

Publications of Results:
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Responsible Party: Shaare Zedek Medical Center
ClinicalTrials.gov Identifier: NCT03950050     History of Changes
Other Study ID Numbers: 0005-18-SZMC
First Posted: May 15, 2019    Key Record Dates
Last Update Posted: May 15, 2019
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Gaucher Disease
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
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Ambroxol
Expectorants
Respiratory System Agents