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Efficacy and Safety of Plasma Exchange With Albutein® 5% in Patients With Amyotrophic Lateral Sclerosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2016 by Grifols Therapeutics Inc.
Sponsor:
Collaborator:
Grifols Biologicals Inc.
Information provided by (Responsible Party):
Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT02872142
First received: August 11, 2016
Last updated: August 15, 2016
Last verified: August 2016
  Purpose
This is a pilot, phase IIa, prospective, open-label, single-arm study to evaluate disease progression, forced vital capacity, and the safety and tolerability of plasma exchange (PE) using Albutein® 5% in subjects with amyotrophic lateral sclerosis (ALS).

Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Biological: Albutein 5%
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Pilot Study to Evaluate the Efficacy and Safety of Plasma Exchange With Albutein® 5% in Patients With Amyotrophic Lateral Sclerosis

Resource links provided by NLM:


Further study details as provided by Grifols Therapeutics Inc.:

Primary Outcome Measures:
  • Changes from baseline in the ALS Functional Rating Scale - Revised [ Time Frame: Weeks 0 to 48 ]
  • Changes from baseline in forced vital capacity [ Time Frame: Weeks 0 to 48 ]

Secondary Outcome Measures:
  • Changes from baseline in cognitive function determined by the ALS - Cognitive Behavioral Screen test [ Time Frame: Weeks 0 to 48 ]
  • Changes from baseline in the motor evoked potential in thenar and hypothenar eminence and anterior tibialis muscle determined by electromyography [ Time Frame: Weeks 0 to 48 ]
  • Evaluation of quality of life using the ALS Assessment Questionnaire 40 [ Time Frame: Weeks 0 to 48 ]
  • Changes from baseline in plasma human apolipoproteins [ Time Frame: Weeks 0 to 48 ]
  • Changes from baseline in cerebrospinal fluid human apolipoproteins [ Time Frame: Weeks 0 to 25 ]
  • Changes from baseline in plasma beta-methylamino-L-alanine levels [ Time Frame: Weeks 0 to 48 ]
  • Changes from baseline in cerebrospinal fluid beta-methylamino-L-alanine levels [ Time Frame: Weeks 0 to 25 ]
  • Changes from baseline in absolute leukocyte count [ Time Frame: Weeks 0 to 48 ]
  • Changes from baseline in plasma neurofilament analysis [ Time Frame: Weeks 0 to 48 ]
  • Changes from baseline in cerebrospinal fluid neurofilament analysis [ Time Frame: Weeks 0 to 25 ]

Other Outcome Measures:
  • Percentage of PE sessions associated with at least one adverse reaction (AR) [ Time Frame: During or within 72 hours after the completion of the product infusion ]
  • Percentage of PE sessions associated with at least one AE, irrespective of causality [ Time Frame: During or within 72 hours after the completion of the product infusion ]
  • Incidence of all AEs [ Time Frame: Weeks -2 to 48 ]

Estimated Enrollment: 10
Study Start Date: July 2016
Estimated Study Completion Date: January 2018
Estimated Primary Completion Date: January 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Albutein 5%
Plasma exchanges with Albutein 5% as a replacement solution
Biological: Albutein 5%
Plasma exchange will be performed using Albutein 5% as the replacement solution. Albutein is manufactured from human plasma. The dose of Albutein 5% for replacement following plasma removal will be calculated based on gender, weight, and the hematocrit of the subject.
Other Name: Albutein 5% solution for perfusion

Detailed Description:
This is a phase IIa, prospective, open-label, single-arm pilot study to evaluate the efficacy and safety of PE with Albutein® 5% in subjects with ALS. The planned enrollment is 10 subjects who have a definite, possible, or probable diagnosis of ALS, according to the revised El Escorial criteria. Enrolled subjects will be treated with PE using Albutein 5% as a replacement solution during an Intensive Treatment Phase (2 PEs per week over 3 weeks) followed by a Maintenance Treatment Phase (weekly PE for 21 weeks) for a total treatment duration of 6 months. A 6-month follow up will begin after the last PE.
  Eligibility

Ages Eligible for Study:   19 Years to 69 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Signed informed consent
  • Subjects over 18 years of age and less than 70 years old
  • Subjects with a possible, probable-lab supported, probable, or definite diagnosis of ALS, according to the revised El Escorial criteria
  • Subjects having experienced their first ALS symptoms within 18 months prior to recruitment/consent
  • Forced Vital Capacity > 70%
  • Subjects must be medically suitable for study participation and willing to comply with all planned aspects of the protocol, including blood sampling, at the time of inclusion in the study.

Exclusion Criteria:

  • Subjects with pre-existing clinically significant lung disease not attributable to ALS
  • Subjects diagnosed with other neurodegenerative diseases or diseases associated with other motor neuron dysfunction
  • Participation in another investigational product study within one month prior to screening
  • Females who are pregnant, breastfeeding, or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device or intrauterine system, condom or occlusive cap with spermicidal foam/ gel/ film/ cream/ suppository, male sterilization, or true abstinence) throughout the study
  • Difficult or problematic peripheral vein access and inability to implant a central catheter which would make continuous PE not feasible as per the visit protocol
  • Contraindication to undergo PE or subject has abnormal coagulation parameters at the discretion of the Outpatient Apheresis Unit team, including but not limited to:

    1. Thrombocytopenia (platelets <100,000/μL)
    2. Fibrinogen <1.5 g/L
    3. International Normalized Ratio >1.5
    4. Beta-blocker treatment and bradycardia <50 beats/min
    5. Treatment with angiotensin-converting enzyme inhibitors which may increase the risk of allergic reactions, unless a preventive change in hypotensive treatment occurs prior to enrollment
  • History of anaphylaxis or severe systemic response to any plasma-derived albumin preparation, component of Albutein® 5%, or other blood product(s)
  • Subjects unable to interrupt treatment with acetylsalicylic acid, other oral antiplatelet, or anticoagulant
  • Renal dysfunction by elevated creatinine concentration >2 mg/dL
  • Presence of heart disease that contraindicates PE treatment, including ischemic cardiopathy and congestive heart failure
  • Presence of prior behavioral disorders requiring pharmacological intervention with less than 3 months of stable treatment
  • Mentally challenged subject who cannot give independent informed consent
  • Any condition that would complicate compliance with the study protocol (i.e., illness with the expectation of less than one year survival, abuse of drugs or alcohol, etc.)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02872142

Contacts
Contact: Miquel Barcelo Miquel.Barcelo@Grifols.com

Locations
United States, New Hampshire
Dartmouth-Hitchcock Medical Center Recruiting
Lebanon, New Hampshire, United States, 03756
Sponsors and Collaborators
Grifols Therapeutics Inc.
Grifols Biologicals Inc.
  More Information

Responsible Party: Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT02872142     History of Changes
Other Study ID Numbers: GBI1501
Study First Received: August 11, 2016
Last Updated: August 15, 2016
Individual Participant Data  
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on May 25, 2017