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).
This is a phase 2, prospective, open-label, single-arm pilot study to evaluate the efficacy and safety of PE with Albutein® 5% in participants with ALS. The planned enrollment is 10 participants who have a definite, possible, or probable diagnosis of ALS, according to the revised El Escorial criteria. Enrolled participants will be treated with PE using Albutein 5% as a replacement solution during an Intensive Treatment Phase (2 PEs per week over 3 weeks) followed by a Maintenance Treatment Phase (weekly PE for 21 weeks) for a total treatment duration of 6 months. A 6-month follow up will begin after the last PE.
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.)