Efficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis

NCT02479802 | Completed Phase 2 Results

• 1 other identifier: IG1309
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

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).

Conditions

Amyotrophic Lateral Sclerosis

Keywords

plasma exchange; albumin

Outcome Measures

Primary Outcomes (2)

• 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).

  Baseline, Weeks 4, 12, 25, 36, and 48

• Changes From Baseline in Percent Predicted Forced Vital Capacity (FVC)

  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

  Baseline, Weeks 25 and 48

• Motor Evoked Potential in Thenar and Hypothenar Eminence, and Anterior Tibialis Muscle

  Baseline, Weeks 4, 12, 25, 36, and 48

• Changes From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 (ALSA-Q40).

  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

  During the Treatment Phase (24 weeks)

Study Arms (1)

Albumin

EXPERIMENTAL

Plasma exchange with Albumin

Biological: Albumin

Interventions

Albumin BIOLOGICAL

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

Also known as: Human Albumin 5%, Albutein 5%

Albumin

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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%

You may not qualify if:

• 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.)

Sponsors & Collaborators

• Instituto Grifols, S.A.: lead

Study Sites (1)

Hospital Universitari Bellvitge

L'Hospitalet de Llobregat, Barcelona, 08907, Spain

Location

Related Publications (1)

• 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.

  PMID: 34791571 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Albumins

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Proteins; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Miquel Barcelo, PhD
• Organization: Grifols Bioscience Industrial Group

Miquel.Barcelo@Grifols.com

+34 935.712.368

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 10, 2015
• First Posted: June 24, 2015
• Study Start: November 1, 2014
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2016
• Last Updated: June 16, 2020
• Results First Posted: June 16, 2020

Record last verified: 2020-06

Locations

ES (1)