Safety and Tolerability of Autologous Non-hematopoietic Peripheral Blood Stem Cells Therapy in Healthy Adult Volunteers.

Healthy Clinical Trial

— SANTANA  

Official title:

A Phase 1 Study Evaluating the Safety and Tolerability of Autologous Non-hematopoietic Peripheral Blood Stem Cells Therapy in Healthy Adult Volunteers in Abu Dhabi, 2020 (SANTANA Study).

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04645485
• Other study ID #: CT.002.1.0.SANTANA
• Status: Completed
• Phase: Phase 1
• Start date: May 12, 2020
• Est. completion date: July 30, 2020

Study information

• Verified date: November 2020
• Source: Abu Dhabi Stem Cells Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

SANTANA Study is a phase 1, prospective, monocentric, open-label clinical trial involving healthy adult volunteers, with the objective to evaluate the safety and tolerability of the compressor nebulization of autologous non-hematopoietic peripheral blood stem cells (NHPBSC).

Description:

The study was conducted in Abu Dhabi Stem Cells Center (ADSCC), and the cell processing and investigational product formulation were performed at the same center according to Good Laboratory Practices (GLPs) and Good Manufacturing Practices (GMPs). Participants received the investigational therapy through compressor (jet) nebulization (two doses divided on Days 0 and 1). The primary endpoint was the safety assessment, to be measured as Adverse Reactions (ARs) incidence [by the World Health Organization - Uppsala Monitoring Centre (WHO-UMC) causality assessment system], and the nebulization tolerability rate (Days 0 and 1). The Laboratory testing profile was evaluated before treatment (Day 0 or -1) and on Days 14 and 28. Two thorax radiography tests (chest X-rays - CXR) were assessed: before the therapy (Day 0 or -1) and on Day 7. An electrocardiogram (ECG) assessment was performed on Days 0 (or -1), 14, and 28. The application of the validated St. George's Respiratory Questionnaire (SGRQ) on Days 0 and 28 was a surrogate safety endpoint. The whole clinical assessment in SANTANA Study was daily recorded until Day 28, taking into account the ARs incidence. The secondary endpoint was the tolerability assessment, measured by nebulization tolerability rate (Days 0 and 1), and Adverse Events (AEs) tolerability within 28-days. The trial was approved by the ADSCC Research Ethics Committee, and written informed consent was obtained from all participants. SANTANA Study was conducted following the Declaration of Helsinki principles and Good Clinical Practices (GCPs), and the authors are responsible for designing the trial, compiling, and analyzing the data.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: July 30, 2020
• Est. primary completion date: June 30, 2020
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

Inclusion Criteria:

• Healthy adult male or female volunteers (18-50 years inclusive). Healthy subjects:

volunteers not having any mental or physical diagnosed disorder or requiring regular or frequent medication, who is not known to suffer any significant illness, who should be within the ordinary range of body measurements)

• Non-smoking volunteers, based on self-reporting who have not used tobacco or nicotine-containing products (or less than 100 cigarettes or equivalent in his / her lifetime), including commercially available cigarettes, hand-rolled cigarettes, bidis, cigars, chewing tobacco, pipes, hookahs, shishas, snuff, snus, electronic cigarettes and similar devices, and nicotine replacement therapy within the last 12 months.

• Body mass index (BMI) = 19 kg/m².

• For females: premenopausal, non-lactating, and non-pregnant.

• Ability to comply with test requirements and peripheral blood stem cell collection.

• The subject agrees to participate in the study and signs the SANTANA Study informed consent form.

Exclusion Criteria:

• Pediatric subjects (aged < 18 years) or older than 50 years' volunteers.

• Any clinically relevant gastrointestinal, renal, hepatic, neurological, hematological, endocrine, oncological, urological, immunological, and cardiovascular diseases or any other condition including clinically significant abnormal Laboratory parameters that, in the opinion of the Investigator, could jeopardize the safety of the subject.

• Current smoking subjects (or former smokers who have quit smoking three months before eligibility assessment).

• Body mass index (BMI) < 19 kg/m².

• For females: Postmenopausal, lactating or pregnant women.

• Donation or receipt of whole blood or blood products within three months before the screening visit.

• Inability to comply with test requirements or peripheral blood stem cell collection.

• Organ or cell transplants in the past three months.

• History of malignancies in the past five years.

• Have participated in other clinical trials in the past three months.

• Employee of Abu Dhabi Stem Cells Center (ADSCC) or their first-degree relatives (parent, sibling, or child).

• Inability to provide informed consent.

Related Conditions & MeSH terms

• Healthy

Intervention

Biological:
• Nebulization of autologous non-hematopoietic peripheral blood stem cells (NHPBSC).
Participants received the investigational therapy through compressor (jet) nebulization, in two doses divided on Days 0 and 1. Each dose contains 10 mL of peripheral blood-derived stem cells CD45-/CD90+/CD133+.

Locations

Country	Name	City	State
United Arab Emirates	Abu Dhabi Stem Cells Center	Abu Dhabi

Sponsors (1)

Lead Sponsor	Collaborator
Abu Dhabi Stem Cells Center

Country where clinical trial is conducted

United Arab Emirates, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse reactions (AR) incidence.	Proportion of participants with treatment-related adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v5.0 and the WHO-UMC system for the causality assessment.	Days 0-28.
Primary	St. George's Respiratory Questionnaire (SGRQ) scoring.	Assessment of scoring changes.	Days 0, 28.
Primary	Nebulization tolerability rate.	Proportion of participants that tolerate the NHPBSC nebulization successfully.	Days 0, 1.
Primary	Adverse Events (AEs) tolerability rate.	Proportion of participants that tolerate AEs -if any, and finish the study.	Days 0-28.