Back to resultsEfficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis
Primary Purpose
Amyotrophic Lateral Sclerosis
Status
Completed
Phase
Phase 2
Locations
Spain
Study Type
Interventional
Intervention
Albumin
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring plasma exchange, albumin
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.)
Sites / Locations
- Hospital Universitari Bellvitge
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Albumin
Arm Description
Plasma exchange with Albumin
Outcomes
Primary Outcome Measures
Changes From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) includes 12 questions classified into 3 subdomains: bulbar function (3 questions), fine and gross motor skills (6 questions), and respiratory function (3 questions) to assess the level self sufficiency. Each task was graded according to a 5-point scale from 0 (incapable) to 4 (normal ability) with a total score from 0 (worst) to 48 (best).
Changes From Baseline in Percent Predicted Forced Vital Capacity (FVC)
Secondary Outcome Measures
Changes From Baseline in ALS Cognitive Function Determined by the ALS-Cognitive Behavioral Screen (ALS-CBS) Test
ALS-CBS test is composed of 2 sections. The first section of the ALS-CBS test includes a questionnaire completed by the caregiver regarding behavior and symptoms status. It is comprised of 15 questions inquiring about possible changes over time in participant's behavior that the caregiver has noticed since the onset of ALS symptoms. Each item is scored on a scale ranging from 0 (worst) to 3 (best) yielding a total 0= large changes to 45=no changes. There were 4 additional questions related to current behavioral symptoms (depression, anxiety, fatigue, and emotional liability). Each question was scored as 0= the presence of symptoms and 1= no current symptoms, giving a total score between 0 and 4. The second section of the ALB-CBS test is for cognitive screening and consists of four items: attention, concentration, follow-up and monitoring, and initiation and recovery. These items were scored 0-5 with a maximum score of 20 (0= cognitive impairment, 20= no apparent cognitive impairment).
Motor Evoked Potential in Thenar and Hypothenar Eminence, and Anterior Tibialis Muscle
Surface electromyography was performed to record motor evoked potential in the distal muscles of the upper limbs (thenar and hypothenar eminence) and dorsiflexor muscles of the lower limbs (anterior tibialis) after electrical stimulation.
Changes From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSA-Q40).
The Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSA-40) consists of 40 items grouped into 5 representative dimensions associated with quality of life. The first 4 scales (physical mobility, activities of daily living, food and drink, communication) refer to deficits and subsequent disabilities as a result of the disease. The fifth scale (emotional functioning) reflects how the subject is facing his/her physical deterioration emotionally. Each item is scored from 0 to 4 according to a gradation of symptom onset frequency (never, rarely, sometimes, often, and always). From raw scores, an index from 0 to 100 is obtained for each dimension, which allow comparisons to be made with the other dimensions as well as a straightforward interpretation of results (0 = better state of health as measured by the questionnaire; 100 = poorer state of health).
Percentage of Plasma Exchange Sessions Associated With One Adverse Event or Adverse Reaction, Including Clinically Significant Changes in Vital Signs or Lab Parameters
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02479802
Brief Title
Efficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis
Official Title
Pilot Study on the Effects of Plasma Exchange on Motor Dysfunction and Cognitive Function in Patients With Amyotrophic Lateral Sclerosis
Study Type
Interventional
2. Study Status
Record Verification Date
June 2020
Overall Recruitment Status
Completed
Study Start Date
November 2014 (undefined)
Primary Completion Date
June 2016 (Actual)
Study Completion Date
June 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Instituto Grifols, S.A.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief 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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
plasma exchange, albumin
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
13 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Albumin
Arm Type
Experimental
Arm Description
Plasma exchange with Albumin
Intervention Type
Biological
Intervention Name(s)
Albumin
Other Intervention Name(s)
Human Albumin 5%, Albutein 5%
Intervention Description
27 plasma exchange procedures using Albumin 5% (estimated 3000 mL per plasma exchange) as replacement solution:
three weeks of intensive treatment with two plasma exchanges per week
twenty-one weeks of maintenance treatment with one weekly plasma exchange
Primary Outcome Measure Information:
Title
Changes From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Description
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) includes 12 questions classified into 3 subdomains: bulbar function (3 questions), fine and gross motor skills (6 questions), and respiratory function (3 questions) to assess the level self sufficiency. Each task was graded according to a 5-point scale from 0 (incapable) to 4 (normal ability) with a total score from 0 (worst) to 48 (best).
Time Frame
Baseline, Weeks 4, 12, 25, 36, and 48
Title
Changes From Baseline in Percent Predicted Forced Vital Capacity (FVC)
Time Frame
Baseline, Weeks 4, 12, 25, 36, and 48
Secondary Outcome Measure Information:
Title
Changes From Baseline in ALS Cognitive Function Determined by the ALS-Cognitive Behavioral Screen (ALS-CBS) Test
Description
ALS-CBS test is composed of 2 sections. The first section of the ALS-CBS test includes a questionnaire completed by the caregiver regarding behavior and symptoms status. It is comprised of 15 questions inquiring about possible changes over time in participant's behavior that the caregiver has noticed since the onset of ALS symptoms. Each item is scored on a scale ranging from 0 (worst) to 3 (best) yielding a total 0= large changes to 45=no changes. There were 4 additional questions related to current behavioral symptoms (depression, anxiety, fatigue, and emotional liability). Each question was scored as 0= the presence of symptoms and 1= no current symptoms, giving a total score between 0 and 4. The second section of the ALB-CBS test is for cognitive screening and consists of four items: attention, concentration, follow-up and monitoring, and initiation and recovery. These items were scored 0-5 with a maximum score of 20 (0= cognitive impairment, 20= no apparent cognitive impairment).
Time Frame
Baseline, Weeks 25 and 48
Title
Motor Evoked Potential in Thenar and Hypothenar Eminence, and Anterior Tibialis Muscle
Description
Surface electromyography was performed to record motor evoked potential in the distal muscles of the upper limbs (thenar and hypothenar eminence) and dorsiflexor muscles of the lower limbs (anterior tibialis) after electrical stimulation.
Time Frame
Baseline, Weeks 4, 12, 25, 36, and 48
Title
Changes From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSA-Q40).
Description
The Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSA-40) consists of 40 items grouped into 5 representative dimensions associated with quality of life. The first 4 scales (physical mobility, activities of daily living, food and drink, communication) refer to deficits and subsequent disabilities as a result of the disease. The fifth scale (emotional functioning) reflects how the subject is facing his/her physical deterioration emotionally. Each item is scored from 0 to 4 according to a gradation of symptom onset frequency (never, rarely, sometimes, often, and always). From raw scores, an index from 0 to 100 is obtained for each dimension, which allow comparisons to be made with the other dimensions as well as a straightforward interpretation of results (0 = better state of health as measured by the questionnaire; 100 = poorer state of health).
Time Frame
Baseline, Weeks 25 and 48
Title
Percentage of Plasma Exchange Sessions Associated With One Adverse Event or Adverse Reaction, Including Clinically Significant Changes in Vital Signs or Lab Parameters
Time Frame
During the Treatment Phase (24 weeks)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
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.)
Facility Information:
Facility Name
Hospital Universitari Bellvitge
City
Hospitalet de Llobregat
State/Province
Barcelona
ZIP/Postal Code
08907
Country
Spain
12. IPD Sharing Statement
Citations:
PubMed Identifier
34791571
Citation
Povedano M, Paipa A, Barcelo M, Woodward MK, Ortega S, Dominguez R, Aragones ME, Horrillo R, Costa M, Paez A. Plasma exchange with albumin replacement and disease progression in amyotrophic lateral sclerosis: a pilot study. Neurol Sci. 2022 May;43(5):3211-3221. doi: 10.1007/s10072-021-05723-z. Epub 2021 Nov 18.
Results Reference
derived
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Efficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis
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