Amyotrophic Lateral SclerosisPhase 2NCT02479802
Efficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis
A Phase 2 study testing Albumin in people with Amyotrophic Lateral Sclerosis, run by Instituto Grifols, S.A. at 1 site in Spain.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Albumin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteSpain
- Study dates
- Nov 2014to Jun 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 13Show fewer
- 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.)
Trial locations and contacts
1 site in 1 country.
Spain1 site
Hospital Universitari Bellvitge
L'Hospitalet de Llobregat, Barcelona, 08907
About this study
Official title: Pilot Study on the Effects of Plasma Exchange on Motor Dysfunction and Cognitive Function in Patients With Amyotrophic Lateral Sclerosis
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).
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Albumin
- Keywords
- plasma exchange, albumin
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlbuminBiological
Also called Human Albumin 5%, Albutein 5%
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
| Study group | Role | What they receive |
|---|---|---|
| Albumin | Experimental | Plasma exchange with Albumin Receives: Biological: Albumin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Baseline, Weeks 4, 12, 25, 36, and 48
Changes From Baseline in Percent Predicted Forced Vital Capacity (FVC)
Measured: Baseline, Weeks 4, 12, 25, 36, and 48
Secondary outcomes (4)
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).
Measured: Baseline, Weeks 25 and 48
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.
Measured: Baseline, Weeks 4, 12, 25, 36, and 48
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).
Measured: Baseline, Weeks 25 and 48
Percentage of Plasma Exchange Sessions Associated With One Adverse Event or Adverse Reaction, Including Clinically Significant Changes in Vital Signs or Lab Parameters
Measured: During the Treatment Phase (24 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2015 (estimated)
- Study startNov 2014
- Primary completionJun 2016
- Study completionJun 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Instituto Grifols, S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02479802
- Sponsor's protocol ID
- IG1309
- Status last verified
- Jun 2020
- First submitted
- 10 Jun 2015
- Last updated
- 16 Jun 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 34791571
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study on the Effects of Plasma Exchange on Motor Dysfunction and Cognitive Function in Patients With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT02479802
- Acronym / sponsor ID
- IG1309
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- plasma exchange, albumin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Instituto Grifols, S.A.Industry (pharma, biotech, devices)
- Study start
- Nov 2014
- Primary completion
- Jun 2016 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 24 Jun 2015 (estimated)
- Results
- Posted 16 Jun 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Albumin | Experimental | Biological: AlbuminPlasma exchange with Albumin |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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%
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AlbuminAlso: Human Albumin 5%, Albutein 5%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 | Biological | Albumin |
Primary outcome
Changes From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Time frame: Baseline, Weeks 4, 12, 25, 36, and 48
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)
Time frame: Baseline, Weeks 4, 12, 25, 36, and 48
4 secondary outcomes
Changes From Baseline in ALS Cognitive Function Determined by the ALS-Cognitive Behavioral Screen (ALS-CBS) Test
Time frame: Baseline, Weeks 25 and 48
Motor Evoked Potential in Thenar and Hypothenar Eminence, and Anterior Tibialis Muscle
Time frame: Baseline, Weeks 4, 12, 25, 36, and 48
Changes From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSA-Q40).
Time frame: Baseline, Weeks 25 and 48
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)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jun 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34791571Povedano 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Albumin in people with Amyotrophic Lateral Sclerosis, run by Instituto Grifols, S.A. at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Jun 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) (time frame: Baseline, Weeks 4, 12, 25, 36, and 48). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02479802, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jun 2020.