Phase 2
N=13
Efficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis
Bottom Line
View on ClinicalTrials.gov: NCT02479802 ↗Enrolled (actual)
13
Serious AEs
30.8%
Results posted
Jun 2020
Primary outcome: Primary: Changes From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) — -1.0; -1.5; -4.0; -5.5 units on a scale — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Albumin (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Instituto Grifols, S.A.
- Primary completion
- Jun 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Changes From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) |
-1.0; -1.5; -4.0; -5.5; -10.0 | <0.0001 sig |
| PRIMARY Changes From Baseline in Percent Predicted Forced Vital Capacity (FVC) |
-6.0; -3.5; -9.0; -12.0; -23.0 | 0.0006 sig |
| SECONDARY Changes From Baseline in ALS Cognitive Function Determined by the ALS-Cognitive Behavioral Screen (ALS-CBS) Test |
0.0; -1.0; 0.0; 0.0; 0.0; -1.0 | — |
| SECONDARY Motor Evoked Potential in Thenar and Hypothenar Eminence, and Anterior Tibialis Muscle |
2.6; 4.9; 3.1; 1.9; 2.1; 1.2 | — |
| SECONDARY Changes From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSA-Q40). |
10.0; 32.5; 12.5; 25.0; 0.0; 8.3 | — |
| SECONDARY Percentage of Plasma Exchange Sessions Associated With One Adverse Event or Adverse Reaction, Including Clinically Significant Changes in Vital Signs or Lab Parameters |
0.9 | — |
Summary
Pilot, phase II, prospective, open-label, uncontrolled study of plasma exchange with 5% albumin in 10 subjects having a definite, possible, or probable diagnosis of Amyotrophic Lateral Sclerosis (ALS).
Eligibility Criteria
Inclusion Criteria
- Signed written-informed consent.
- Subjects over 18 years of age, and less than 70 years old.
- Subjects with a definite, possible, or probable diagnosis of ALS, according to the revised El Escorial criteria.
- Subjects having experienced their first ALS symptoms within 18 months before recruitment/consent.
- FVC > 70%
- Subjects must be medically suitable for study participation and of complying with all planned aspects of the protocol including blood sampling at the time of inclusion in the study.
Exclusion Criteria
- Subjects with a clinically significant preexisting lung disease not attributable to ALS.
- Subjects with a diagnosis of other neurodegenerative diseases or diseases associated with dysfunction of the motor neurons that can confuse the diagnosis of ALS.
- Participation in other clinical trials, or the reception of any other investigational drug in the six months prior to the start of the study.
- Female subjects who are pregnant, currently breastfeeding, or attempting to conceive during the study.
- Difficult peripheral venous access precluding plasma exchange and inability to implement a viable alternative catheter to make continued performing plasma exchange visits according to protocol
- Any contraindication for plasma exchange or abnormal coagulation parameters according clinical criteria from apheresis team
- A history of frequent adverse reactions (serious or otherwise) to blood products.
- Hypersensitivity to albumin or allergies to any of the components of Albutein.
- Subjects that can not interrupt treatment with acetylsalicylic acid or oral anticoagulants
- Plasma creatinine > 2mg/dl.
- Present a history of heart disease including ischemic heart disease or congestive heart failure.
- Presence of prior conduct disorders requiring pharmacologic intervention, with less than 3 months of stable treatment
- Any condition that complicates adherence to study protocol (illness with less than one year of expected survival, drug or alcohol abuse, etc.)
Data sourced from ClinicalTrials.gov (NCT02479802). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.