Status:
COMPLETED
A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy.
Lead Sponsor:
Hadassah Medical Organization
Conditions:
Gaucher Disease Type 1
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
An open-labeled, prospective, single-center proof-of-concept study. Patients with Gaucher Disease aged 18-75 who received intravenous Enzyme Replacement Therapy once every two weeks were enrolled. The...
Detailed Description
Patients diagnosed with GD and intravenously treated at home with a regular dose of Enzyme Replacement Therapy once every two weeks for 6 months (30- 60 U/kg per mouth) were included in the study. We...
Eligibility Criteria
Inclusion
- Adult \>18 years old
- Non-pregnant
- Diagnosed with Gaucher disease
- Treated with ERT for a minimum of 3 years
- An unchanged dose in the past 6/12 months
Exclusion
- Pregnant
- Patients with evidence of severe infectious, malignant, autoimmune, or other disabling systemic diseases
- Patients unable to provide written informed consent
- Patients that do not possess a smartphone
- Patients who cant adhere to the visit schedule and protocol.
Key Trial Info
Start Date :
April 1 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2023
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT06050967
Start Date
April 1 2022
End Date
March 1 2023
Last Update
September 22 2023
Active Locations (1)
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1
Hebrew university
Jerusalem, Israel, 96955