A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy.

Gaucher Disease Type 1 Clinical Trial

Official title:

A Feasibility Open-labeled Clinical Trial Using a Second-generation Artificial Intelligence-based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT06050967
• Other study ID #: 0056-21-SZMC
• Status: Completed
• Phase: Phase 2
• Start date: April 1, 2022
• Est. completion date: March 1, 2023

Study information

• Verified date: August 2023
• Source: Hadassah Medical Organization
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 study utilized the Altus Care™ cellular phone-based application, which integrated an algorithm-based approach to provide random dosing regimens within a pre-defined range determined by the physician. The app allowed personalized therapeutic regimens with variations in dosages and administration times.

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 installed Altus Care™- this is a cellular phone-based application that allows easy digitization of treatment plans or research protocols and remote implementation. In coordination with the patient's treating physician and the home treating nurse, an individualized treatment plan was prepared for each patient within a pre-defined range of minimal and maximal once in two weeks ERT dosages and timing frames for its administration. Per protocol, the patient's monthly dose was not changed, but each dose and the timing of administration was changed randomly using the app.

During the follow-up period, the research coordinator made a regular weekly checkup by phone, questioning the patient's clinical well-being and adherence to the treatment plan. A physical examination, CBC, and Lyso-GB1 assessed response to therapy were done approximately every two months (twice during the study and once again at the end of the follow-up). The patients filled out SF-36 questionnaires at the beginning and the end of the follow-up.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: March 1, 2023
• Est. primary completion date: March 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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 Criteria:

• 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.

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease Type 1

Intervention

Combination Product:
• Enzyme replacement therapy for Gaucher disease in combination with altus care application
In coordination with the patient's treating physician and the home treating nurse, an individualized treatment plan was prepared for each patient within a pre-defined range of minimal and maximal once in two weeks enzyme replacement therapy dosages and timing frames for its administration. Per protocol, the patient's monthly dose was not changed, but each dose and the timing of administration was changed randomly using the app.

Locations

Country	Name	City	State
Israel	Hebrew university	Jerusalem

Sponsors (1)

Lead Sponsor	Collaborator
Hadassah Medical Organization

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Platelet	number of platelets per L. normal range 150,000 to 450,000. In Gaucher disease lower platelets means higher disease activity	6 month
Primary	Hemoglobin	Hemoglobin concentration in gram/dl. normal range 13.8 to 17.2 for males and 12.1 to 15.1 for females. The lower the hemoglobin the higher Gaucher disease activity	6 month
Primary	Lyso GB1	Lyso GB1 level in ng/ml. For healthy individuals the levels should be trace less than 4.9ng/ml. In patients with Gaucher disease the higher the level the higher the disease activity.	6 month
Primary	SF-36	score of 0-100. 0 means worse and 100 means best.	6 month