Clinical trial NCT02479802: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: plasma exchange
albumin
Lead sponsor: Grifols Biologicals, LLC
Sponsor class: INDUSTRY
First posted: 2015-06-24
Enrollment: 13
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


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.)

Primary outcomes:
measure: Changes From Baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
timeFrame: Baseline, Weeks 4, 12, 25, 36, and 48
measure: Changes From Baseline in Percent Predicted Forced Vital Capacity (FVC)
timeFrame: Baseline, Weeks 4, 12, 25, 36, and 48
Secondary outcomes:
measure: Changes From Baseline in ALS Cognitive Function Determined by the ALS-Cognitive Behavioral Screen (ALS-CBS) Test
timeFrame: Baseline, Weeks 25 and 48
measure: Motor Evoked Potential in Thenar and Hypothenar Eminence, and Anterior Tibialis Muscle
timeFrame: Baseline, Weeks 4, 12, 25, 36, and 48
measure: Changes From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSA-Q40).
timeFrame: Baseline, Weeks 25 and 48
measure: Percentage of Plasma Exchange Sessions Associated With One Adverse Event or Adverse Reaction, Including Clinically Significant Changes in Vital Signs or Lab Parameters
timeFrame: During the Treatment Phase (24 weeks)
Other outcomes:
Locations:
city: Hospitalet de Llobregat
country: Spain
facility: Hospital Universitari Bellvitge
state: Barcelona
Study officials:
References:
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.