Clinical trial NCT04928287: Randomized, Double-Blind Clinical Trial for Parkinson's Disease (Early and Moderate)
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"
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Parkinson Disease
Lead sponsor: Hope Biosciences
Sponsor class: INDUSTRY
Collaborators: name: Hope Biosciences
First posted: 2021-06-16
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: A parallel study is a type of clinical study where two groups of treatments, A (HB-adMSCs) and B (Placebo), are given so that one group receives only A while another group receives only B.
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Change From Baseline in MDS-UPDRS Part II.
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Laboratory Values. CBC (% of WBC)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CBC (10^9 Cells/L)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CBC (10^12 Cells/L)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CBC (pg)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CBC (fL)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CBC (g/dL)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CBC (% Difference in Volume and Size of RBC)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CBC (% of Total Blood Cell Count)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Vital Signs. - Blood Pressure (mmHg)
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Weight in kg.
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Laboratory Values. CMP (mg/dL)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CMP (g/dL)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CMP (IU/L)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CMP (mL/Min/1.73m^2)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CMP (mmol/L)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CMP (Ratio: Albumin (g/dL) to Calc. Globulin (g/dL))
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. CMP (Ratio: Urea Nitrogen (mg/dL) to Creatinine (mg/dL))
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. Coagulation Panel (Seconds)
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Laboratory Values. Coagulation Panel (Ratio: Prothrombin Time (Seconds) / Mean Normal Prothrombin Time (Seconds))
timeFrame: Baseline (Week 0), Week 24, and End of Study (Week 52)
measure: Vital Signs. - Respiratory Rate (Breaths Per Minute)
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Vital Signs. - Heart Rate (Beats Per Minute)
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Vital Signs. - Body Temperature (Celsius )
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Secondary outcomes:
measure: Change From Baseline in MDS-UPDRS Part I.
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in MDS-UPDRS Part III.
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in MDS-UPDRS Part IV.
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Communication
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Social Roles and Activities
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Changes From Baseline in Neuro-QOL. - Anxiety
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Depression
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Dyscontrol
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Changes From Baseline in Neuro-QOL. - Fatigue
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Mobility
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Well-Being
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Sleep
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Fine Motor
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Stigma
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Social Roles
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Neuro-QOL. - Cognition
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Parkinson's Disease Fatigue Scale (PFS-16).
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Parkinson's Disease Questionnaire (PDQ-39).
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Total Visual Analog Scale
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Dosage of Carbidopa/Levodopa
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
measure: Change From Baseline in Vital Signs. - Oxygen Saturation.
timeFrame: Baseline through Week 32, Follow-up at week 42 and End of Study at week 52
Other outcomes:
Locations:
city: Sugar Land
country: United States
facility: Hope Biosciences Stem Cell Research Foundation
state: Texas
Study officials:
affiliation: Hope Biosciences Stem Cell Research Foundation
name: Djamchid Lotfi, MD
role: PRINCIPAL_INVESTIGATOR
References: