Safety and Efficacy of POL6326 for Mobilization of Hematopoietic Stem Cells in Healthy Volunteers

POL6326 - A Phase I, Open, Single Intravenous Infusion Dose, Pharmacokinetic and Pharmacodynamic Study in Healthy Volunteers Who Are Volunteering as Haematopoietic Stem Cell (HSC) Donors

Phase I study to determine and compare the safety/tolerability of single ascending doses of POL6326 by intravenous infusion.

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: POL6326

Detailed Description

Phase I Study

• to determine and compare the safety of single ascending doses of POL6326 by intravenous infusion
• to determine the relationship between 2-hour single intravenous infusion doses of POL6326 and the concentration of HSC and mature WBC in peripheral blood using immunophenotypic assays.

• Study Type: Interventional
• Enrollment (Actual): 27
• Phase: Phase 1

Contacts and Locations

Study Locations

• Germany
  • Frankfurt, Germany, 60590
    • German Red Cross Blood Service and Institute for Transfusion Medicine and Immunohematology of the Goethe University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

1. Mobilization and collection of hematopoietic stem cells (HSCs) using G-CSF at least 6 weeks but less than 6 months prior to protocol enrollment.
2. Ages greater than or equal to 18 years and less than or equal to 55 years.
3. Normal liver and renal function, normal CBC, normal ECG, normal blood pressure including adequately medically controlled idiopathic arterial hypertension, no other contraindications to mobilized peripheral blood stem cell donation according to WMDA criteria and relevant SOPs at the study site defining additional exclusion criteria for stem cell donation.
4. Subject must be eligible for normal blood donation according to requirements for IDMs as laid out by national law for blood donors.
5. Ability to comprehend the investigational nature of the study and provide informed consent.

Exclusion Criteria:

1. Active infection or history of recurrent infection- hepatitis B and C (HBsAg, Anti-HBc, Anti-HCV), HIV and HTLV-1.
2. History of autoimmune disease such as rheumatoid arthritis, systemic lupus erythematous.
3. History of cancer within the past 5 years excluding basal cell or squamous cell carcinoma of the skin.
4. History of any hematologic disorders including thromboembolic disease.
5. History of cardiac disease such as uncontrolled hypertension, peripheral vascular disease, myocardial infarction, cardiac arrhythmias OR related symptoms such as tachycardia, chest pain, shortness of breath which have required medical intervention OR treatment or a Framingham coronary disease risk prediction score of greater than 10% 10 year coronary heart disease (CHD) risk.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: POL6326; 2-hour single intravenous infusion doses of POL6326	Drug: POL6326

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of single ascending doses of POL6326 by intravenous infusion according to standard criteria	Safety as measured by the incidence, type and severity of adverse events.	2 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetic outcome	Determine the pharmacokinetic profile of POL6326 following single intravenous administration, e.g. Cmax, AUC, terminal half life and clearance	24h
Mobilisation of CD34+ cells	Time dependent measurement of CD34+ cells during and after infusion of POL6326 in all subjects	2 days

Collaborators and Investigators

• Sponsor: Polyphor Ltd.
• Investigators: Principal Investigator: Halvard Boenig, M.D. Ph.D, German Red Cross Blood Service and Institute for Transfusion Medicine and Immunohematology of the Goethe University, Frankfurt, Germany

Publications and helpful links

General Publications

• Karpova D, Brauninger S, Wiercinska E, Kramer A, Stock B, Graff J, Martin H, Wach A, Escot C, Douglas G, Romagnoli B, Chevalier E, Dembowski K, Hooftman L, Bonig H. Mobilization of hematopoietic stem cells with the novel CXCR4 antagonist POL6326 (balixafortide) in healthy volunteers-results of a dose escalation trial. J Transl Med. 2017 Jan 3;15(1):2. doi: 10.1186/s12967-016-1107-2.

Study record dates

Study Major Dates

• Study Start: February 1, 2012
• Primary Completion (ACTUAL): August 1, 2015
• Study Completion (ACTUAL): September 1, 2015

Study Registration Dates

• First Submitted: April 2, 2013
• First Submitted That Met QC Criteria: April 23, 2013
• First Posted (ESTIMATE): April 26, 2013

Study Record Updates

• Last Update Posted (ESTIMATE): November 25, 2015
• Last Update Submitted That Met QC Criteria: November 24, 2015
• Last Verified: November 1, 2015

More Information

Terms related to this study

• Keywords: volunteers; Mobilization; CXCR4; Peripheral blood; HSC
• Other Study ID Numbers: POL-3