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Trial registered on ANZCTR


Registration number
ACTRN12615000687594
Ethics application status
Not yet submitted
Date submitted
15/06/2015
Date registered
2/07/2015
Date last updated
22/01/2016
Type of registration
Prospectively registered

Titles & IDs
Public title
The safety and efficacy of combination treatment using adipose derived adult stem cells, platelet-rich-plasma and peptides on patients with Multiple Sclerosis.
Scientific title
A single group prospective study to evaluate the efficacy and safety of treatments using autologous, non-expanded adipose derived stem cells, platelet-rich-plasma and peptides on patients with Multiple Sclerosis.
Secondary ID [1] 286912 0
Nil
Universal Trial Number (UTN)
Trial acronym
StemMS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Multiple Sclerosis 295331 0
Condition category
Condition code
Neurological 295599 295599 0 0
Multiple sclerosis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This study aims to evaluate the efficacy and safety of treatments using Autologous, Non-Expanded, Adipose Derived Stem Cells (stromal vascular fraction, SVF), platelet-rich-plasma (PRP) and peptides on patients with Multiple Sclerosis (MS). Patients are assessed using inclusion and exclusion criteria, in order to determine whether they are eligible for an Autologous Stem cell transplant. If eligible, patients are prescribed, three proprietary peptide injections (growth factors) (dosage- 0.1 - 0.2 ml each, daily self-injection, 5 days/week for 4 weeks prior to liposuction procedure. The peptides are derived from those that are present naturally throughout the body and are critical to cellular processes particularly growth and regeneration. The ongoing maintenance dose of the peptides via injection is necessary due to their short half-life and most effective circulation and absorption. One injection continues for 5 weeks post-procedure while the other 2 injections continue for the trial duration) and specific Vitamin D3 dietary supplement (dosage- 4 capsules (1000 IU per capsule) per day starting 3 weeks prior and up to stem cell collection procedure).
The procedure commences with extraction of whole blood (60 ml) followed by preparation of PRP (approximately 7 ml) which is later combined with the SVF preparation for injection. Within the SVF, there will be an approximate minimum of 10^6 viable, nucleated cells deployed per kg body weight. Liposuction using local anaesthesia and syringe collection will be performed to collect the adipose tissue specimen (50 ml) for subsequent processing to isolate the SVF. SVF and PRP are filtered and irradiated. This preparation is administered with saline solution intravenously. Following the procedure, standard supportive measures including antimicrobial prophylaxis are followed as per clinic protocols. The duration of the day procedure including extraction, processing and injection is ~ 7 hours. This procedure and treatment is administered only once. Any adverse events are monitored at procedure and at 1 week follow-up visit. Monitoring is continued remotely for the trial duration. Condition specific validated questionnaires and physiological tests are applied at subsequent interviews at 3, 6, and 12 months via telephone and internet utilities. Data will be compared with previous medical history and baseline measurements taken before procedure.
Intervention code [1] 292097 0
Treatment: Other
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 295309 0
Disability score compared to baseline assessed by Multiple Sclerosis Functional Composite (MSFC revised) score
Timepoint [1] 295309 0
Post-procedure: 3 months, 6 months, 12 months
Primary outcome [2] 295310 0
Change from baseline of disability and health status: Scale (EDSS-revised).

Timepoint [2] 295310 0
Post-procedure: 3 months, 6 months, 12 months
Primary outcome [3] 295403 0
Patient Reported Outcomes (PROs): MS Impact Scale (MSIS-29).
Timepoint [3] 295403 0
Post-procedure: 3 months, 6 months, 12 months
Secondary outcome [1] 315324 0
The safety of protocol will be evaluated by assessment of the number, time-frame of occurrence and severity of Adverse Events (Adverse Events will be graded by the Common Terminology Criteria for Adverse Events v 4.0 (CTCAE) and coded according to the Medical Dictionary for Regulatory Activities (MedDRA) terminology. Adverse events monitoring will consist of measurement of vital signs (temperature, heart rate, blood pressure, respiration) and relevant blood tests if indicated.
Timepoint [1] 315324 0
day of procedure, and 1 week follow-up visit and participants reports throughout trial.

Eligibility
Key inclusion criteria
Patients who give written consent to participate in the study.
Patients diagnosed with Multiple Sclerosis clinically defined and supported by laboratory tests (eg: MRI) (revised McDonald criteria (2010)). Active MS, defined by: At least one outbreak in the last year or at least one Gadolinium enhancing lesion in the last 6 months.
EDSS (1.5) to (6.5) at screening evaluation.
Minimum age
18 Years
Maximum age
70 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Inability to give informed consent
Known allergies/ hypersensitivities to local anaesthetics or antibiotics
Any illness which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromise patient safety, or interfere with the interpretation of the study results
Females who are pregnant (detected by urinary hCG in fertile women) or lactation or females of childbearing potential who are unwilling to maintain contraceptive therapy for the duration of the study.
Anti-coagulant use (as below) that cannot be stopped or bleeding disorders (no use of Warfarin, Aspirin, Vitamin E, fish oil or anti-inflammatory medications 10 days before treatment)
Active or chronic infectious disease including infection with HIV1-2 or chronic Hepatitis B or Hepatitis C. For patients who have tested positive, an expert will be consulted as to patient eligibility based on the patient's infectious status.
Known drug or alcohol dependence.
Immunosuppressed.
Congestive heart failure (NYHA III / IV), cardiomyopathy, heart rhythm disorder requiring treatment, unstable or severe heart disease (CCS III or IV), severe hypertension (systolic greater than 180, diastolic greater than 110). Systolic blood pressure (supine) =90 mmHg; Resting heart rate > 100 bpm; Cerebrovascular accident within 6 months prior to study entry.
History of cancer (other than non-melanoma skin cancer or in-situ cervical cancer) in the last five years.
Previously demonstrated hematologic disease. Abnormal blood counts, a history of myelodysplasia or other cytopenia. Patients with WBC count less than 3000 cells per microliter, platelets less than 100,000 cells per microliter and un-transfused hemoglobin less than 10 grams per deciliter.
Previously demonstrated renal insufficiency (e.g. serum creatinine clearance less than 30ml/min) or An estimated glomerular filtration rate (eGFR)< 60 mL/min/1.73m2 history
Exposure to any investigational drug or procedure within 6 weeks prior to study entry or enrolled in a concurrent study that may confound results of this study.
Participation in clinical trials of any experimental drugs in the 6 months before study entry.
Interferon beta or Glatiramer acetate 6 weeks prior to screening
Treatment with Alemtuzumab (campath-1H) within the last 2 years.
Experimental treatment within 3 months prior to screening
Patients with EDSS less than 1.5 or greater than 6.5.
Patients on chronic immunosuppressive transplant therapy.
Immunosuppressive agents ( eg., Glucocorticosteroid therapy within the last 6 weeks), Natalizumab or Fingolimod in the 3 months prior to screening.


Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Funding & Sponsors
Funding source category [1] 291468 0
Commercial sector/Industry
Name [1] 291468 0
MasterCell Stem Cell Centre
Address [1] 291468 0
Niecon Plaza, 47 / 19 Victoria ave.,
Broadbeach
QLD 4218
Country [1] 291468 0
Australia
Funding source category [2] 291469 0
Commercial sector/Industry
Name [2] 291469 0
Australian Custom Pharmaceuticals Pty Ltd
Address [2] 291469 0
Unit 1, 4 Endeavour Road,
Taren Point,
NSW 2229
Country [2] 291469 0
Australia
Funding source category [3] 291470 0
Other
Name [3] 291470 0
Adult Stem Cell Foundation
Address [3] 291470 0
PO BOX 8468 GCMC
Bundall,
QLD 4217
Country [3] 291470 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
MasterCell Stem Cell Centre
Address
Niecon Plaza, 47 / 19 Victoria ave.,
Broadbeach
QLD 4218
Country
Australia
Secondary sponsor category [1] 290148 0
Other
Name [1] 290148 0
Adult Stem Cell Foundation
Address [1] 290148 0
PO BOX 8468 GCMC
Bundall,
QLD 4217
Country [1] 290148 0
Australia

Ethics approval
Ethics application status
Not yet submitted
Ethics committee name [1] 293018 0
Ethics committee address [1] 293018 0
Ethics committee country [1] 293018 0
Australia
Date submitted for ethics approval [1] 293018 0
29/02/2016
Approval date [1] 293018 0
Ethics approval number [1] 293018 0

Summary
Brief summary
Recent research and clinical evidence indicates that Human adipose-derived stem cells are safe for use in Humans. Researchers suggest that the treatment may alter the operation of the immune system to ameliorate degenerative activity. In a conducive environment supported by a combination of growth factors and certain peptides, stem cells can also be stimulated to differentiate into a range of different cell types when transported to the site of degradation. This trial is investigating to what extent an injection of the Stromal Vascular Fraction obtained from the participants own fat tissue and Platelet-Rich-Plasma, and in combination with proprietary peptides reduces the progression of disease and improves quality of life. The peptides are derived from those that are naturally present throughout the body and are critical to cellular processes particularly growth and regeneration. Age and disease conditions can reduce the levels and effectiveness of these peptides which normally promote healing. This study involves patients with neuro-degenerative disease specifically, multiple sclerosis of all categories.
Trial website
Trial related presentations / publications
Public notes
Participants are required to contribute to trial costs and no remuneration for personal expenses is available. Two visits to the study center on the Gold Coast are required over an eight day period.

Contacts
Principal investigator
Name 58106 0
Dr Soraya Felix
Address 58106 0
Mastercell Stem Cell Centre
Niecon Plaza, 47 / 19 Victoria ave.,
Broadbeach QLD 4218
Country 58106 0
Australia
Phone 58106 0
+61 7 5526 7001
Fax 58106 0
Email 58106 0
info@drfelix.com.au
Contact person for public queries
Name 58107 0
Dr Mark Edwards
Address 58107 0
Mastercell Stem Cell Centre
Niecon Plaza, 47 / 19 Victoria ave.,
Broadbeach QLD 4218
Country 58107 0
Australia
Phone 58107 0
+61 7 5526 7001
Fax 58107 0
Email 58107 0
info@drfelix.com.au
Contact person for scientific queries
Name 58108 0
Dr Mark Edwards
Address 58108 0
Mastercell Stem Cell Centre
Niecon Plaza, 47 / 19 Victoria ave.,
Broadbeach QLD 4218
Country 58108 0
Australia
Phone 58108 0
+61 7 5526 7001
Fax 58108 0
Email 58108 0
info@drfelix.com.au

No information has been provided regarding IPD availability
Summary results
No Results