Pilot Study to Evaluate the Efficacy and Safety of Plasma Exchange With Albutein® 5% in Patients With Amyotrophic Lateral Sclerosis

Study 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).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Other

Study Status:

Active, currently recruiting

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT02872142

Neals Affiliated?

No

Coordinating Center Contact Information

Dartmouth-Hitchcock Medical Center
Miquel Barcelo / .(JavaScript must be enabled to view this email address)
.(JavaScript must be enabled to view this email address) Lebanon, New Hampshire 03756 United States

Full Study Summary:

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.

Study Sponsor:

Grifols Therapeutics Inc.

Participant Duration:

Estimated Enrollment:

10

Estimated Study Start Date:

07/01/2016

Estimated Study Completion Date:

01/01/2018

Posting Last Modified Date:

11/29/2016

Date Study Added to alsconsortium.org:

11/29/2016
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    19

    Maximum Age:

    69

    Min Vital Capacity (% predicted normal):

    70

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    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:
    Thrombocytopenia (platelets <100,000/μL)
    Fibrinogen <1.5 g/L
    International Normalized Ratio >1.5
    Beta-blocker treatment and bradycardia <50 beats/min
    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.)

  • Site Contact Information

    Dartmouth-Hitchcock Medical Center
    Miquel Barcelo / .(JavaScript must be enabled to view this email address) / Lebanon, New Hampshire 03756
    United States