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: March 2023
• 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	Changes in MDS-UPDRS Part II.	MDS-UPDRS Part II.	Baseline to Weeks 52.
Primary	Incidence of treatment-emergent Adverse Event (TEAEs).	treatment-emergent Adverse Event.	Baseline to Weeks 52.
Primary	Incidence of treatment-emergent Serious Adverse Events (SAEs).	SSAEs.	Baseline to Weeks 52.
Primary	AEs of special interest (serious or non-serious) - thromboembolic events.	Incidence of thromboembolic events.	Baseline to Weeks 52.
Primary	AEs of special interest (serious or non-serious) - thromboembolism of the extremities	Incidence and risk of AEs of special interest (serious or non-serious), including peripheral events defined as, thromboembolism of the extremities.	Baseline to Weeks 52.
Primary	AEs of special interest (serious or non-serious) - infections	Incidence and risk of AEs of special interest (serious or non-serious), including infections.	Baseline to Weeks 52.
Primary	AEs of special interest (serious or non-serious) - hypersensitivities.	Incidence and risk of AEs of special interest (serious or non-serious), including hypersensitivities.	Baseline to Weeks 52.
Primary	Laboratory values. CBC	Clinically significant changes in CBC values.	Baseline to Weeks 52.
Primary	Laboratory values. CMP	Clinically significant changes in CMP values.	Baseline to Weeks 52.
Primary	Laboratory values. Coagulation Panel	Clinically significant changes in Coagulation Panel values.	Baseline to Weeks 52.
Primary	Vital signs. - Respiratory Rate (breaths per minute)	Clinically significant changes in Respiratory Rate.	Baseline to Weeks 52.
Primary	Vital signs. - Heart Rate (beats per minute)	Clinically significant changes in Heart Rate.	Baseline to Weeks 52.
Primary	Vital signs. - Body Temperature (Fahrenheit )	Clinically significant changes in Heart Rate.	Baseline to Weeks 52.
Primary	Vital signs. - Blood Pressure (mmHg)	Clinically significant changes in Blood Pressure.	Baseline to Weeks 52.
Primary	Weight in lb.	Clinically significant changes in Weight in lb.	Baseline to Weeks 52.
Primary	Physical examination results. General	Clinically significant changes in general physical examination results.	Baseline to Weeks 52.
Primary	Physical examination results. Body Systems.	Clinically significant changes in body systems physical examination results.	Baseline to Weeks 52.
Secondary	Changes in MDS-UPDS total score.	MDS-UPDS Parts I, II, III and IV.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in MDS-UPDRS Part I.	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.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in MDS-UPDRS Part III.	Motor Examination, including speech, facial expression, rigidity, finger tapping, hand movements, pronation-supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement (body bradykinesia), postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, constancy of rest tremor, dyskinesias impact and Hoehn and Yahr stage.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in MDS-UPDRS Part IV.	Motor Complications, including time spent with dyskinesias and others.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Communication	Communication - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Social Roles and Activities	Ability to Participate in Social Roles and Activities - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Anxiety	Anxiety - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Depression	Depression - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Dyscontrol	Emotional and Behavioral Dyscontrol - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Fatigue	Fatigue - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Mobility	Lower Extremity Function (Mobility) - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Well-Being	Positive Affect and Well-Being - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Sleep	Sleep Disturbance - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Fine Motor	Upper Extremity Function (Fine Motor, ADL) - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Stigma	Stigma-Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Social Roles	Satisfaction with Social Roles and Activities - Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Neuro-QOL. - Cognition	Cognition Function- Short Form	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Parkinson's disease fatigue scale (PFS-16).	Score of =8 indicates the presence of significant fatigue.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Parkinson's disease Questionnaire (PDQ-39).	Difficulties across the 8 quality of life dimensions of functioning of wellbeing.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Visual Analog Scale for Pain.	Visual Analog Scale for Pain.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Dosage of medications taken to treat Parkinson's disease.	Dosage of medications.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Incidence of treatment-emergent Adverse Event (TEAEs) and serious Adverse Events (SAEs).	Adverse Event (TEAEs)	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Incidence and risk of AEs of special interest (serious or non-serious),	Including thromboembolic events, peripheral events defined as, thromboembolism of the extremities, also infections and hypersensitivities.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in laboratory values. - CBC	CBC	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Changes in Visual Analog Scale for muscle spasms.	Visual Analog Scale for muscle spasms.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in vital signs. - Respiratory Rate	Changes in Respiratory Rate.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in vital signs. - Heart Rate	Changes in Heart Rate.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in vital signs. - Body Temperature.	Changes in Body Temperature.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in vital signs. - Blood Pressure.	Changes in Blood Pressure.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in vital signs. - Oxygen Saturation.	Changes in Oxygen Saturation.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in physical examination results.	Physical examination results.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in weight in lb.	Weight in lb.	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in laboratory values. - CMP	CMP	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.
Secondary	Clinically significant changes in laboratory values. - Coagulation Panel	Coagulation Panel	Baseline to Weeks 4, 8, 16, 24, 32, 42 and 52.