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Study was suspended due to IPL344 shortage and may resume once drug supply is available. Survival follow-up is still ongoing following participants' consent
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| IPL344 | Experimental | IV IPL344 administered once a day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IPL344 | Drug | 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). |
| 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 |
| 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 |
| 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) |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hadassah Medical Center/Neuromuscular / EMG service and ALS / Motor Neuron Disease Clinic | Jerusalem | 91120 | Israel | |||
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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|
| upto 36 months |
| 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 |
| Hadassah Medical Center -Motor Neuron Disease Clinic |
| Jerusalem |
| Israel |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |