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

NCT06050967 | Completed Phase 2

• 1 other identifier: 0056-21-SZMC
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Gaucher Disease Type 1

Outcome Measures

Primary Outcomes (4)

• Platelet

  number of platelets per L. normal range 150,000 to 450,000. In Gaucher disease lower platelets means higher disease activity

  6 month

• 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

• 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

• SF-36

  score of 0-100. 0 means worse and 100 means best.

  6 month

Study Arms (1)

individualized treatment plan with random dosage and time of administration

EXPERIMENTAL

Baseline clinical and laboratory parameters acquired at the screening included: a physical examination, complete blood count (CBC), Lyso-GB1 , and a 36-item short-form survey (SF-36) for quality-of-life examination. Once providing informed consent, the Altus Care™ application was installed on the patient's cellular phone. In coordination with the patient's treating physician and the home treating nurse5 , 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 wasn't changed, but each dose and the timing of administration was changed randomly using the app.

Combination Product: Enzyme replacement therapy for Gaucher disease in combination with altus care application

Interventions

Enzyme replacement therapy for Gaucher disease in combination with altus care application COMBINATION_PRODUCT

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.

individualized treatment plan with random dosage and time of administration

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Hadassah Medical Organization: lead

Study Sites (1)

Hebrew university

Jerusalem, 96955, Israel

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

Enzyme Replacement Therapy

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Enzyme Therapy; Drug Therapy; Therapeutics

Study Officials

• Noa Hurvitz, MD

  Faculty of Medicine, Hebrew University, Jerusalem, Israel.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 1, 2023
• First Posted: September 22, 2023
• Study Start: April 1, 2022
• Primary Completion: March 1, 2023
• Study Completion: March 1, 2023
• Last Updated: September 22, 2023

Record last verified: 2023-08

Locations

IL (1)