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

NCT ID: NCT02872142

Last Updated: 2021-05-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-08-29
• Study Completion Date: 2019-08-15

Brief Summary

This is a pilot, phase 2, 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 participants with amyotrophic lateral sclerosis (ALS).

Detailed Description

This is a phase 2, prospective, open-label, single-arm pilot study to evaluate the efficacy and safety of PE with Albutein® 5% in participants with ALS. The planned enrollment is 10 participants who have a definite, possible, or probable diagnosis of ALS, according to the revised El Escorial criteria. Enrolled participants 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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Albutein 5%

Plasma exchanges (PEs) with albutein 5% as a replacement solution during an intensive treatment phase of two PEs per week over 3 weeks followed by maintenance treatment phase of weekly PE for 21 weeks. The dose of albutein 5% for replacement following plasma removal was calculated based on gender, weight, and the hematocrit of the participant.

Group Type: EXPERIMENTAL

Albutein 5%

Intervention Type: BIOLOGICAL

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 participant.

Plasma Exchange

Intervention Type: PROCEDURE

Plasma Exchange will be performed using albutein 5% as the replacement solution.

Interventions

Albutein 5%

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 participant.

Intervention Type: BIOLOGICAL

Plasma Exchange

Plasma Exchange will be performed using albutein 5% as the replacement solution.

Intervention Type: PROCEDURE

Other Intervention Names

Albutein 5% solution for perfusion

Eligibility 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 Amyotrophic Lateral Sclerosis (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 Plasma exchange (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/ microliter [μL])

2. Fibrinogen <1.5 gram per liter (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 milligram per deciliter (mg/dL)
• Presence of heart disease that contraindicates PE treatment, including ischemic cardiopathy and congestive heart failure
• Presence of prior behavioural 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.)

• Minimum Eligible Age: 19 Years
• Maximum Eligible Age: 69 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Grifols Biologicals, LLC

INDUSTRY

Sponsor Role: collaborator

Grifols Therapeutics LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

GBI1501

Identifier Type: -

Identifier Source: org_study_id