Efficacy and Safety of Plasma Exchange With Albutein® 5% in Participants With Amyotrophic Lateral Sclerosis
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Intervention: Albutein 5%; Plasma Exchange
Intervention description: Albutein is manufactured from human plasma. The dose of Albutein 5% for replacement following plasma removal will be calculated based on gender, weight, and the hematocrit of the participant.; Plasma Exchange will be performed using albutein 5% as the replacement solution.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 12.0
Sex: All
Minimum age: 19 Years
Maximum age: 69 Years
Healthy volunteers: No

Summary:

      This is a pilot, phase 2, prospective, open-label, single-arm study to evaluate disease
      progression, forced vital capacity, and the safety and tolerability of plasma exchange (PE)
      using Albutein® 5% in participants with amyotrophic lateral sclerosis (ALS).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Signed informed consent

          -  Subjects over 18 years of age and less than 70 years old

          -  Subjects with a possible, probable-lab supported, probable, or definite diagnosis of
             Amyotrophic Lateral Sclerosis (ALS), according to the revised El Escorial criteria

          -  Subjects having experienced their first ALS symptoms within 18 months prior to
             recruitment/consent

          -  Forced Vital Capacity > 70%

          -  Subjects must be medically suitable for study participation and willing to comply with
             all planned aspects of the protocol, including blood sampling, at the time of
             inclusion in the study.

        Exclusion Criteria:

          -  Subjects with pre-existing clinically significant lung disease not attributable to ALS

          -  Subjects diagnosed with other neurodegenerative diseases or diseases associated with
             other motor neuron dysfunction

          -  Participation in another investigational product study within one month prior to
             screening

          -  Females who are pregnant, breastfeeding, or, if of child-bearing potential, unwilling
             to practice a highly effective method of contraception (oral, injectable or implanted
             hormonal methods of contraception, placement of an intrauterine device or intrauterine
             system, condom or occlusive cap with spermicidal foam/ gel/ film/ cream/ suppository,
             male sterilization, or true abstinence) throughout the study

          -  Difficult or problematic peripheral vein access and inability to implant a central
             catheter which would make continuous Plasma exchange (PE) not feasible as per the
             visit protocol

          -  Contraindication to undergo PE or subject has abnormal coagulation parameters at the
             discretion of the Outpatient Apheresis Unit team, including but not limited to:

               1. Thrombocytopenia (platelets <100,000/ microliter [μL])

               2. Fibrinogen <1.5 gram per liter (g/L)

               3. International Normalized Ratio >1.5

               4. Beta-blocker treatment and bradycardia <50 beats/min

               5. Treatment with angiotensin-converting enzyme inhibitors which may increase the
                  risk of allergic reactions, unless a preventive change in hypotensive treatment
                  occurs prior to enrollment

          -  History of anaphylaxis or severe systemic response to any plasma-derived albumin
             preparation, component of Albutein® 5%, or other blood product(s)

          -  Subjects unable to interrupt treatment with acetylsalicylic acid, other oral
             antiplatelet, or anticoagulant

          -  Renal dysfunction by elevated creatinine concentration >2 milligram per deciliter
             (mg/dL)

          -  Presence of heart disease that contraindicates PE treatment, including ischemic
             cardiopathy and congestive heart failure

          -  Presence of prior behavioural disorders requiring pharmacological intervention with
             less than 3 months of stable treatment

          -  Mentally challenged subject who cannot give independent informed consent

          -  Any condition that would complicate compliance with the study protocol (i.e., illness
             with the expectation of less than one year survival, abuse of drugs or alcohol, etc.)