Clinical trial NCT06050967: A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy.
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Gaucher Disease Type 1
Lead sponsor: Hadassah Medical Organization
Sponsor class: OTHER
First posted: 2023-09-22
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 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.

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 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Platelet
timeFrame: 6 month
measure: Hemoglobin
timeFrame: 6 month
measure: Lyso GB1
timeFrame: 6 month
measure: SF-36
timeFrame: 6 month
Secondary outcomes:
Other outcomes:
Locations:
city: Jerusalem
country: Israel
facility: Hebrew university
Study officials:
affiliation: Faculty of Medicine, Hebrew University, Jerusalem, Israel.
name: Noa Hurvitz, MD
role: PRINCIPAL_INVESTIGATOR
References: