Safety, Efficacy of FFP From Healthy Donors to Ameliorate Frailty and Enhance Immune Function in Older Individuals

NCT03458429 | Enrolling By Invitation Phase 1 DMC FDA Drug

• 1 other identifier: 001.16-BMSCTI
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

1. 1.Primary Objective
2. 2.Secondary Objective

Conditions

Frailty

Keywords

Young Donor Plasma; Frailty; Aging; Immune Function; G-CSF

Outcome Measures

Primary Outcomes (1)

• Number of participants with treatment-related adverse events as assessed by clinical symptoms, laboratory abnormalities, serious adverse events and treatment-limiting adverse events

  To determine the safety and tolerability of 12 once monthly transfusions of GMFFP (Granulocyte - Colony Stimulating Factor (G-CSF) Mobilized Fresh Frozen Plasma harvested from young, healthy donors) given to older, frail individuals who are at risk due to unhealthy aging and who will be assessed throughout the duration of treatment. After the one-year treatment period, participants will complete follow-up assessments every 3 months for 1 year.

  24 months

Secondary Outcomes (4)

• Efficacy outcomes as measured by changes from baseline at 12 and 24 months assessing Immune Risk Profile (IRP)

  12 and 24 months

• Efficacy outcomes as measured by changes from baseline at 12 and 24 months assessing cognitive function (MME)

  12 and 24 months

• Efficacy outcomes as measured by changes from baseline at 12 and 24 months assessing quality of life (OPQOL-35)

  12 and 24 months

• Efficacy outcomes as measured by changes from baseline at 12 and 24 months assessing Frailty Index (FI)

  12 and 24 months

Study Arms (1)

Frail, older subjects

EXPERIMENTAL

Treated subjects are the frail, older subjects who will be treated with Granulocyte-Colony Stimulating Factor (G-CSF) Mobilized Fresh Frozen Plasma (GMFFP) in this protocol.

Drug: GMFFP

Interventions

GMFFP DRUG

Granulocyte-Colony Stimulating Factor (G-CSF) Mobilized Fresh Frozen Plasma

Also known as: Granulocyte-Colony Stimulating Factor (G-CSF) Young Plasma

Frail, older subjects

Eligibility Criteria

• Age: 55 Years - 95 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Provide written informed consent.
• Be aged between 55-95 years.
• Show signs of frailty apart from a concomitant condition as assessed by the Investigator with a score of 4-7 on the Clinical Frailty Scale (Appendix 1) and/or Have an abnormal Immune Risk Profile (IRP)
• Not be pregnant or nursing while participating in this trial. Both men and women of reproductive potential must agree to use an effective means of birth control while participating in the trial. Women of childbearing potential should have a negative serum pregnancy test before treatment, if not surgically sterile.
• Have a negative neutrophil antibody test.
• Have a negative Human Leukocyte Antigen (HLA) Class I and II antibody test.
• Have Cytomegalovirus (CMV) negative or positive sero-testing completed.
• Have a life expectancy of at least 24 months as judged by the PI at the time of consent.
• Have less than 4 weeks since prior medical therapy, radiation therapy, and/or surgery
• Have adequate organ function including:
• Hemoglobin greater than 10.0 g/dl Absolute neutrophils must be greater than 1,500/Microliter(uL) Platelet count must be greater than100,000/uL Serum bilirubin must be less than 2mg/dL Aspartate Aminotransferase (AST) must be less than 90 units/L Alanine Transaminase (ALT) must be less than 105 units/L Serum creatinine must be less than 2mg/dL

You may not qualify if:

• Have a score of less than 4 or greater than 7 on the Clinical Frailty Scale or Have a score of less than 4 and a No Immune Risk Profile (IRP)
• Have used anti-inflammatory medications within 7 days of study treatment,treated subjects may be re-screened after 14 days
• Abnormal clinical values including but not limited to:
• Platelet count less than 100,000/mm3 Absolute neutrophils less than 1500/uL Hemoglobin less than 10 g/dL Aspartate transaminase, alanine transaminase, or alkaline phosphatase Greater than 3 times upper limit of normal Serum bilirubin greater than 2 mg/dL
• Be an organ transplant recipient or have an active listing (or expected future listing) for transplant of any organ.
• Have a documented intolerance to plasma or its components, or prior intolerance to intravenous fluids.
• Have known serum antibodies to plasma proteins, such as haptoglobin, Complement Component (C3/C4), or alpha-1-antitrypsin.
• Previous plasma transfusion within 30 days of signing informed consent.
• Serious comorbid illness including, but not limited to:
• HIV or hepatitis Congenital deficiency of immunoglobulin A (IgA) Pulmonary edema Advanced liver or renal failure Uncontrolled diabetes mellitus, Significant and/or symptomatic cardiovascular disease (e.g. any history of myocardial infarction, congestive heart failure, unstable angina, uncontrolled arrhythmia) Active serious infection. Hemorrhagic cystitis Cardiac revascularization within last six months Severe obstructive ventilatory defect Renal insufficiency (serum creatinine greater than 177 mol/L or creatinine clearance less than 20 mL/min) Sickle cell disease or hemoglobinopathy Cancer Any other condition that the investigator believes may compromise the safety or compliance of the patient or the study evaluation.
• No vascular access
• Pregnant or nursing female
• Recent ( less than 24 months) history of drug or alcohol abuse
• Current participation in an investigational therapeutic or medical device trial
• Treatment with any human blood product, including intravenous immunoglobulin, during the 6 months prior to screening or during the trial.

Sponsors & Collaborators

• Dipnarine Maharaj: lead

Study Sites (1)

South Florida Bone Marrow/Stem Cell Transplant Institute DBA Maharaj Institute of Immune Regenerative Medicine

Boynton Beach, Florida, 33437, United States

Location

MeSH Terms

Conditions

Frailty

Interventions

Granulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Dipnarine Maharaj

  South Florida Bone Marrow/Stem Cell Transplant Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Model Details: To determine the safety and tolerability of 12 once monthly transfusions of GMFFP (Granulocyte - Colony Stimulating Factor (G-CSF) Mobilized Fresh Frozen Plasma harvested from young, healthy donors), given to older, frail individuals who are at risk due to unhealthy aging and who will then have a subsequent 12-month follow-up period.

Study Record Dates

• First Submitted: February 7, 2018
• First Posted: March 8, 2018
• Study Start: February 5, 2018
• Primary Completion (Estimated): December 30, 2029
• Study Completion (Estimated): December 30, 2030
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)