Randomized, Double-Blind Clinical Trial for Parkinson's Disease (Early and Moderate)

Parkinson Disease Clinical Trial

— PD  

Official title:

"A Randomized, Double-Blind, Single Center, Phase 2, Efficacy and Safety Study of Autologous HB-adMSCs vs Placebo for the Treatment of Patients With Parkinson's Disease"

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04928287
• Other study ID #: HBPD03
• Status: Completed
• Phase: Phase 2
• Start date: June 28, 2021
• Est. completion date: February 6, 2023

Study information

• Verified date: May 2024
• Source: Hope Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a randomized, double-blind, single center, phase 2 study to assess efficacy and safety of multiple HB-adMSCs vs Placebo for the treatment of Parkinson's disease.

The trial includes a screening period of up to 4 weeks, a 32-week treatment period, and a safety Follow-up period of 20 weeks after the last investigational product administration.

This clinical trial will be open to enroll 24 eligible participants diagnosed with Parkinson's disease. Patients' recruitment will be conducted by the study team, if eligible participants are identified based on eligibility criteria, a screening visit will be scheduled. Informed consent form will be given to the study participants and signed before any study procedures. Informed consent form will include information about the clinical trial and some aspects should be considered during this process.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: February 6, 2023
• Est. primary completion date: February 6, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

• A study participant will be eligible for inclusion in this study only if all of the following criteria apply:

1. Male and female participants 18 - 75 years of age.

2. Study participant must have been diagnosed with early and/or moderate Parkinson's disease at least 6 months before study participation.

3. Study participants must have previously banked their mesenchymal stem cells with Hope Biosciences.

4. Study participants should be able to read, understand and to provide written consent.

5. Voluntarily signed informed consent obtained before any clinical-trial related procedures are performed.

6. Female study participants should not be pregnant or plan to become pregnant during study participation and for 6 months after last investigational product administration.

7. Male participants if their sexual partners can become pregnant should use a method of contraception during study participation and for 6 months after the last administration of the investigated product.

8. Study participant is able and willing to comply with the requirements of this clinical trial.

Exclusion Criteria:

• A study participant will not be eligible for inclusion in this clinical trial if any of the following criteria apply:

1. Pregnancy, lactation. Women of childbearing age who are not pregnant but do not take effective contraceptive measures.

2. Study participants with advanced Parkinson's disease described as, severe disability, wheelchair bound or bedridden.

3. Study participant has any active malignancy, including evidence of cutaneous basal, squamous cell carcinoma or melanoma.

4. Study participant has known alcoholic addiction or dependency or has current substance use or abuse.

5. Study participant has 1 or more significant concurrent medical conditions (verified by medical records), including the following:

• Poorly controlled diabetes mellitus (PCDM) defined as history of deficient standard of care treatment and/or pre-prandial glucose >130mg/dl during screening visit or post-prandial glucose >200mg/dl.

• Medical History of Chronic kidney disease (CKD) diagnosis and/or screening results of eGFR < 59mL/min/1.73m2.

• Presence of New York Heart Association (NYHA) Class III/IV heart failure during screening visit.

• Any medical history of myocardial infarction in any of the different types, such as ST-elevation myocardial infarction (STEMI) or non-ST-elevated myocardial infarction (NSTEMI), coronary spasm, or unstable angina.

• Medical history of uncontrolled high blood pressure defined as a deficient standard of care treatment and/or blood pressure > 180/120 mm/Hg during screening visit.

• Medical history of inherited thrombophilias, recent major general surgery, (within 12 months before the Screening), lower extremity paralysis due to spinal cord injury, fracture of the pelvis, hips or femur, cancer of the lung, brain, lymphatic, gynecologic system (ovary or uterus), or gastrointestinal tract (like pancreas or stomach).

• History of brain surgery for Parkinson's disease.

6. Study participant has received any stem cell treatment within 6 months before first dose of investigational product other than stem cells produced by Hope Biosciences.

7. Receiving any investigational therapy or any approved therapy for investigational use within 1 year prior first dose of the investigational product other than COVID-19 vaccines.

8. Study participant has a laboratory abnormality during screening, including the following:

• White blood cell count < 3000/mm3

• Platelet count < 80,000mm3

• Absolute neutrophil count < 1500/mm3

• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) 10 upper limit of normal (ULN) x 1.5

9. Study participant has any other laboratory abnormality or medical condition which, in the opinion of the investigator, poses a safety risk or will prevent the subject from completing the study.

10. Study participant is unlikely to complete the study or adhere to the study procedures.

11. Study participant with known concurrent acute or chronic viral hepatitis B or C or human immunodeficiency virus (HIV) infection.

12. Study participant has a previously diagnosed psychiatric condition which in the opinion of the investigator may affect self-assessments.

13. Study participant with any systemic infection requiring treatment with antibiotics, antivirals, or antifungals within 30 days prior to first dose of the investigational product.

14. Male study participants who plan to donate sperm during the study or within 6 months after the last dose. Female patients who plan to donate eggs or undergo in vitro fertilization treatment during the study or within 6 months after the last dose.

15. Study participants who are determined by the Investigator to be unsuitable for study enrollment for other reasons

Related Conditions & MeSH terms

• Parkinson Disease

Intervention

Biological:
• HB-adMSCs
HB-adMSCs will be administered intravenously to study participants who qualify.

Other:
• Placebo
Placebo will be administered intravenously to study participants who qualify.

Locations

Country	Name	City	State
United States	Hope Biosciences Stem Cell Research Foundation	Sugar Land	Texas

Sponsors (2)

Lead Sponsor	Collaborator
Hope Biosciences Stem Cell Research Foundation	Hope Biosciences

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in MDS-UPDRS Part II.	The MDS-UPDRS scale refers to Movement Disorder Society - Unified Parkinson Disease Rating Scale, and it is a rating tool used to gauge the course of Parkinson's disease in patients. The MDS-UPDRS scale consists of the following 4 segments. Part II tests "Motor Aspects of Experiences of Daily Living". Each answer to the scale is evaluated by the principal investigator during the study visit. Some sections of the MDS-UPDRS scale require multiple grades assigned to each extremity.; There are 13 items included in Part II. Part II score ranges from 0 - 52; 12 and below is mild, 30 and above is severe. Each item has 0-4 ratings: 0 (normal), 1 (slight), 2 (mild), 3 (moderate), and 4 (severe). The total score ranges from 0 to 260, with 0 indicating no disability and 260 indicating total disability. Higher values represent a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Primary	Laboratory Values. CBC (% of WBC)	Changes from baseline in CBC laboratory values with unit of % of white blood cell count.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CBC (10^9 Cells/L)	Changes from baseline in CBC laboratory values with units of 10^9 cells/L (Leukocytes, Platelets)	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CBC (10^12 Cells/L)	Changes from baseline in CBC laboratory values with units of 10^12 cells/L.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CBC (pg)	Changes from baseline in CBC laboratory values with unit of pg.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CBC (fL)	Changes from baseline in CBC laboratory values with unit of fL.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CBC (g/dL)	Changes from baseline in CBC laboratory values with unit of g/dL.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CBC (% Difference in Volume and Size of RBC)	Changes from baseline in CBC laboratory values with unit of % difference in volume and size of RBC	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CBC (% of Total Blood Cell Count)	Changes from baseline in CBC laboratory values with unit of % of total blood cell count.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Vital Signs. - Blood Pressure (mmHg)	Change from baseline in Blood Pressure.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Primary	Weight in kg.	Change from baseline in Weight in kg.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Primary	Laboratory Values. CMP (mg/dL)	Change from baseline in CMP values with units of mg/dL	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CMP (g/dL)	Changes from baseline in CMP laboratory values with units of g/dL	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CMP (IU/L)	Changes from baseline in CMP laboratory values with units of IU/L.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CMP (mL/Min/1.73m^2)	Changes from baseline in CMP laboratory values with units of mL/min/1.73m^2.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CMP (mmol/L)	Changes from baseline in CMP laboratory values with units of mmol/L.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CMP (Ratio: Albumin (g/dL) to Calc. Globulin (g/dL))	Changes from baseline in CMP laboratory values with units of Ratio: Albumin(g/dL) to Calc. Globulin(g/dL)	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. CMP (Ratio: Urea Nitrogen (mg/dL) to Creatinine (mg/dL))	Changes from baseline in CMP laboratory values with units of Ratio: Urea Nitrogen (mg/dL) to Creatinine (mg/dL)	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. Coagulation Panel (Seconds)	Changes from baseline in Coagulation Panel values with units of seconds.	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Laboratory Values. Coagulation Panel (Ratio: Prothrombin Time (Seconds) / Mean Normal Prothrombin Time (Seconds))	Changes from baseline in Coagulation laboratory values with units of Ratio: Prothrombin time (seconds) / Mean normal prothrombin time (seconds).	Baseline (Week 0), Week 24, and End of Study (Week 52)
Primary	Vital Signs. - Respiratory Rate (Breaths Per Minute)	Changes from Baseline in Respiratory Rate.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Primary	Vital Signs. - Heart Rate (Beats Per Minute)	Change from baseline in Heart Rate.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Primary	Vital Signs. - Body Temperature (Celsius )	Change from baseline in Body Temperature.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in MDS-UPDRS Part I.	The MDS-UPDRS scale refers to Movement Disorder Society - Unified Parkinson Disease Rating Scale, and it is a rating tool used to gauge the course of Parkinson's disease in patients. Part I tests "Nonmotor experiences of daily living". Non-Motor Aspects of Experiences of Daily Living (nM-EDL), including complex behaviors such as, cognitive impairment, hallucinations and psychosis, depressed mood, anxious mood, apathy, features of dopamine dysregulation syndrome, sleep problems, daytime sleepiness, pain and other sensations, urinary problems, constipation problems, light headedness on standing and fatigue.; There are 13 items included in Part I. Part I score ranges from 0 - 52; 10 and below is mild, 22 and above is severe. Each item has 0-4 ratings:0 (normal), 1 (slight), 2 (mild), 3 (moderate), and 4 (severe). The total score ranges from 0 to 260, with 0 indicating no disability and 260 indicating total disability. Higher values represent a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in MDS-UPDRS Part III.	The MDS-UPDRS scale refers to Movement Disorder Society - Unified Parkinson Disease Rating Scale, and it is a rating tool used to gauge the course of Parkinson's disease in patients. Part III tests "Motor examination". Motor Examination includes speech, facial expression, rigidity, finger and hand movement, pronation-supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement, postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, constancy of rest tremor, dyskinesias impact and Hoehn and Yahr stage.; There are 18 items included in Part III. Part III score ranges from 0 - 132; 32 and below is mild, 59 and above is severe. Each item has 0-4 ratings:0 (normal), 1 (slight), 2 (mild), 3 (moderate), and 4 (severe). The total score ranges from 0 to 260, with 0 indicating no disability and 260 indicating total disability. Higher values represent a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in MDS-UPDRS Part IV.	The MDS-UPDRS scale refers to Movement Disorder Society - Unified Parkinson Disease Rating Scale, and it is a rating tool used to gauge the course of Parkinson's disease in patients. Part IV tests "Motor Complications", including time spent with dyskinesias and others.; There are 6 items included in Part IV. Part IV score ranges from 0 - 24; 4 and below is mild, 13 and above is severe. Each item has 0-4 ratings:0 (normal), 1 (slight), 2 (mild), 3 (moderate), and 4 (severe). The total score ranges from 0 to 260, with 0 indicating no disability and 260 indicating total disability. Higher values represent a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Communication	Communication - Short Form; Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The communication short form has 5 questions regarding communicative abilities of the patient, and each question ranges from 0 points to 5 points (making the total 25 points). A higher score represents a better outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Social Roles and Activities	Ability to Participate in Social Roles and Activities - Short Form; Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Ability to Participate in Social Roles and Activities" short form has 8 questions regarding social abilities of the patient (familial and friend relationships), and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a better outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Changes From Baseline in Neuro-QOL. - Anxiety	Anxiety - Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Anxiety" short form has 8 questions regarding the anxiety level of the patient (inquiring about level of uneasiness and worry), and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Depression	Depression - Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Depression" short form has 8 questions regarding the depression level of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Dyscontrol	Emotional and Behavioral Dyscontrol - Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Dyscontrol" short form has 8 questions regarding the emotional and behavioral dyscontrol level of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Changes From Baseline in Neuro-QOL. - Fatigue	Fatigue - Short Form Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Fatigue" short form has 8 questions regarding the Fatigue level of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Mobility	Lower Extremity Function (Mobility) - Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Mobility" short form has 8 questions regarding the Lower Extremity Function (Mobility) level of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Well-Being	Positive Affect and Well-Being - Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Positive Affect and Well-Being" short form has 8 questions regarding the well-being level of the patient, and each question ranges from 0 points to 5 points (making the total 45 points). A higher score represents a better outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Sleep	Sleep Disturbance - Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Sleep disturbance" short form has 8 questions regarding the sleep level of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Fine Motor	Upper Extremity Function (Fine Motor, ADL) - Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Upper Extremity Function (Fine Motor, ADL)" short form has 8 questions regarding the fine motor levels of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a better outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Stigma	Stigma-Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Stigma" short form has 8 questions regarding the stigma levels of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Social Roles	Satisfaction with Social Roles and Activities - Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Satisfaction with Social Roles and Activities" short form has 8 questions regarding the satisfaction levels of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a better outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Neuro-QOL. - Cognition	Cognition Function- Short Form Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The "Cognition Function" short form has 8 questions regarding the cognition function levels of the patient, and each question ranges from 0 points to 5 points (making the total 40 points). A higher score represents a better outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Parkinson's Disease Fatigue Scale (PFS-16).	The Parkinson's disease fatigue scale (PFS-16) is an 16 question assessment that measures the level of fatigue in patients diagnosed with Parkinson's disease. Each question has 5 answer choices established as "strongly disagree", "disagree", "do not agree or disagree", "agree", and "strongly agree", which are scored as 1 to 5, respectively, making the total score from 16 to 80 points. Higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39).	The Parkinson's Disease Questionnaire (PDQ-39) assesses how often people with Parkinson's experience difficulties across 8 dimensions of daily living including relationships, social situations and communication. Items are grouped into eight scales that are scored by expressing summed item scores as a percentage score ranging between 0 and 100. The final reported score (summary index) is calculated by averaging the 8 scaled scores. Higher score represents a worse outcome.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Total Visual Analog Scale	Visual Analog Scale for Pain and Muscle Spasm. The pain and muscle spasm VAS is a unidimensional measure of pain/ muscle spasm intensity, used to record patients' pain progression and muscle spasm progression, or compare pain and muscle spasm severity between patients with similar conditions. Using a ruler, the score is determined by measuring the distance (mm) on the 10-cm line between the "no pain" anchor and the patient's mark summed with the distance (mm) on the 10-cm line between the "no muscle spasm" anchor and the patient's mark, providing a range of scores from 0-200. A higher score indicates greater pain/spasm intensity.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Dosage of Carbidopa/Levodopa	Dosage of medications.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary	Change From Baseline in Vital Signs. - Oxygen Saturation.	Changes in Oxygen Saturation.	Baseline through Week 32, Follow-up at week 42 and End of Study at week 52