A Study of IPL344 in the Treatment of ALS Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 11 December 2023
• Main ID: NCT03652805
• Date of registration: 13/08/2018
• Prospective Registration: No
• Primary sponsor: Immunity Pharma Ltd.
• Public title: A Study of IPL344 in the Treatment of ALS Patients ALS
• Scientific title: Phase 1/2a, Multi-center, Open-Label, Dose-escalating Study to Assess Safety, Tolerability, and Pharmacokinetics of Intravenously Administered IPL344 for The Treatment of Amyotrophic Lateral Sclerosis (ALS)
• Date of first enrolment: August 1, 2018
• Target sample size: 15
• Recruitment status: Suspended
• URL: https://clinicaltrials.gov/ct2/show/NCT03652805
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Israel

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female participants ages =18 to 80 years

2. Consenting participants fulfilling the El Escorial criteria for probable and definite
ALS (sporadic and familial)

3. Participant has ALSFRS-R score >20, the latest ALSFRS-R test should be no more than 6
weeks before screening visit, AND:

1. a disease progression rate greater than 0.55 ALSFRS-R point per month on average,
over at least 4 months, prior to the latest ALSFRS-R test OR

2. a decline of at least 3 points in ALSFRS-R score within the last 4 months prior
to the latest ALSFRS-R test

4. Previous data of Force Vital Capacity (FVC) of =60% at least 3 months before screening
and not more than 12 months.

5. Written informed consent consistent with ICH-GCP and local laws, signed prior to any
study procedures being performed.

6. BMI 18.5 to 30 kg/m2 inclusive and weigh at least 50 kg and no more than 100 kg.

7. If taking riluzole or edaravone, the participant must be on a stable dose for =30 days
prior to Day 1 and expected to remain at that dose until the final study visit.

8. Medically able to undergo the study procedures, and to adhere to the visit schedule at
the time of study entry.

9. Medically is able and willing to undergo placement and maintain a central venous
catheter as determined by the investigator.

10. Participant has a competent caregiver or qualified individual who can and will be
responsible for the administration of study drug and reporting home activities.

11. Geographic accessibility to the study site

12. Females must not be lactating or pregnant at Screening, as documented by a negative
beta-human chorionic gonadotropin [ß-hCG] (or human chorionic gonadotropin [hCG].

13. Women of child-bearing potential or males whose partners are women of child-bearing
potential use an effective method of contraception throughout the trial.

Exclusion Criteria:

1. Concurrent therapy that, in the PI's opinion, would interfere with the evaluation of
the safety or efficacy of the study medication.

2. Co-existing psychiatric disorder excluding a depression disorder occurred after ALS
diagnosis.

3. Participant is a respiratory dependent.

4. Subjects with a significant pulmonary disorder not attributed to ALS.

5. Slow Vital Capacity (SVC) <60.

6. Presence of any other condition or circumstance that, in the judgment of the
Investigator, might contraindicate or increase the risk to the participant or decrease
the chance of obtaining satisfactory data to achieve the objectives of the study.

7. History of HIV, positive HBV or HCV serology.

8. Participants suffering from significant cardiac, or any other disease that may
endanger the participant or interfere with the ability to interpret the results.

9. A participant with active infections.

10. Documented active cancer.

11. Treatment with another investigational drug, biological agent, or device within 2
months of the first dose, or investigational cell therapy within 6 months of the first
dose.



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: IPL344

Primary Outcome(s)

Adverse Events (AEs) and serious adverse events (SAEs) Reporting [Time Frame: (up-to Day 56)]

Maximum Tolerated Dose (MTD) [Time Frame: Study treatment duration (Day 1 -28 days)]

Secondary Outcome(s)

Pharmacokinetic (PK) profile - Maximum Plasma Concentration (Cmax) [Time Frame: Pre-Dose and 5, 10, 20, 30, 45, 60 and 120 minutes after dosing]

Pharmacokinetic (PK) profile - apparent terminal exponential half-life (T1/2) [Time Frame: Pre-Dose and 5, 10, 20, 30, 45, 60 and 120 minutes after dosing]

Pharmacokinetic (PK) profile - time to reach maximum plasma concentration (Tmax) [Time Frame: Pre-Dose and 5, 10, 20, 30, 45, 60 and 120 minutes after dosing]

Pharmacokinetic (PK) profile - Area Under the Curve (AUC) [Time Frame: Pre-Dose and 5, 10, 20, 30, 45, 60 and 120 minutes after dosing]

Secondary ID(s)

101/ 2

Source(s) of Monetary Support

Please refer to primary and secondary sponsors