A Follow up Study to Protocol 101/2 - Continued Treatment by IPL344 IV

An Open-Label, Multi-Center, Follow up Study to Protocol 101/2 - Continued Treatment by Intravenously Administered IPL344 to Amyotrophic Lateral Sclerosis (ALS) Patients

This is a prospective, open-label, follow up study to protocol 101/2 - continued treatment by IPL344 IV administered once a day in up to 15 participants with ALS.

The study is designed to determine the safety, tolerability and initial efficacy of IPL344, administered once a day, by IV infusion for up to 36 months

Study Overview

• Status: Suspended
• Conditions: Amyotrophic Lateral Sclerosis (ALS)
• Intervention / Treatment: Drug: IPL344

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Marc Gotkine, M.D.; Phone Number: +972 2 6778899; Email: marc@gotkine.com

Study Locations

• Israel
  • Jerusalem, Israel
    • Hadassah Medical Center -Motor Neuron Disease Clinic
  • Jerusalem, Israel, 91120
    • Hadassah Medical Center/Neuromuscular / EMG service and ALS / Motor Neuron Disease Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female participants ages ≥ 18 to 75 years.
2. Participants that have completed study protocol #101/2
3. A written informed consent signed prior to any study procedure being performed
4. Medically capable to undergo study procedures at the time of study entry

Exclusion Criteria:

1. Participants that did not participate or did not complete 28 treatment days of study protocol #101/2.
2. Concurrent therapy, that in the PI's opinion, would interfere with the evaluation of the safety or efficacy of the study medication
3. Presence of any other condition or circumstance that, in the judgment of the Investigator, might contraindicate or increase the risk to the participant.
4. Females must not be lactating or pregnant at Screening or Baseline (as documented by a negative serum pregnancy test from protocol 101/2)
5. Women of child-bearing potential or males whose partners are women of child-bearing potential, unwilling or unable to use an effective method of contraception throughout the trial

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IPL344; IV IPL344 administered once a day	Drug: IPL344; IPL344 will be administered by intravenously (IV) infusion once a day (every 24±6 hours), using a Peripherally Inserted Central Catheter (PICC) line or a permanent port. IPL344 will be administered using an electronic pump at a flow rate that will be determined in protocol 101/2. The dose will be fixed as MTD as established in protocol 101/2 (up to 3.2 mg/kg).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events (AEs) and Serious Adverse Events (SAEs) Reporting	All AEs will be recorded, whether considered minor or serious, drug-related or not	upto 36 month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes from baseline in ALS disease progression	Evaluated by the Amyotrophic Lateral Sclerosis Functional Rating Scale(ALSFRS-R). The ALSFRS-R is a quickly administered (5 min) ordinal rating scale used to determine a subject's assessment of their capability and independence in 12 functional activities. There are 12 questions, graded by the subject 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing	upto 36 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes from baseline in Pulmonary Function	Measured by Vital Capacity (VC)	upto 36 months
Changes from baseline in Muscle strength	Assessed by using a quantitative strength testing tool, Hand Held Dynamometry (HHD)	upto 36 months
Changes from baseline in Muscle strength	Assessed by using a quantitative strength testing tool - hand grip	upto 36 months
Changes from baseline in Anti-Depression effect	Evaluated by ALS Depression Inventory (ADI-12). Scales: For each question, the following is selected: "I fully agree", "I agree", "I don't agree", "I do not agree at all"	upto 36 months
Changes from baseline in Anti-Depression effect	Evaluated by the Hospital Anxiety and Depression Scale (HADS). The HADS is a fourteen item scale. Seven of the items relate to anxiety and seven relate to depression. The anxiety and depression subscales each range from 0 to 21, with higher scores indicating higher anxiety/depression complains	upto 36 months

Collaborators and Investigators

• Sponsor: Immunity Pharma Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 9, 2018
• Primary Completion (Estimated): January 1, 2026
• Study Completion (Estimated): February 1, 2026

Study Registration Dates

• First Submitted: November 21, 2018
• First Submitted That Met QC Criteria: November 25, 2018
• First Posted (Actual): November 27, 2018

Study Record Updates

• Last Update Posted (Estimated): December 8, 2023
• Last Update Submitted That Met QC Criteria: December 3, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 101/3

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No