Study of NBMI Treatment in Patients With Atypical Parkinsons (PSP or MSA)

• Register: ClinicalTrials.gov
• Last refreshed on: 27 September 2021
• Main ID: NCT04184063
• Date of registration: 29/07/2019
• Prospective Registration: Yes
• Primary sponsor: EmeraMed
• Public title: Study of NBMI Treatment in Patients With Atypical Parkinsons (PSP or MSA) EMERA006
• Scientific title: A Pilot Exploratory, Randomised, Placebo-controlled, Double Blinded, Cross-over , Phase 2a Study to Explore Efficacy and Safety of NBMI Treatment in Patients With Progressive Supranuclear Palsy (PSP) or Multiple System Atrophy (MSA)
• Date of first enrolment: September 16, 2019
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04184063
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 2

Countries of recruitment

Slovenia

Contacts

• Name: Tanja Turk, M. Pharm
• Affiliation: CRS d.o.o.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Patient has clinically confirmed documented diagnosis of PSP or MSA, according to the
current clinical criteria.

2. Patient has a brain MRI finding consistent with the diagnosis of PSP or MSA at
Screening.

3. Patient is aged 40 years to 85 years inclusive at screening age.

4. Patient is fluent in the local language and possesses sufficient auditory and visual
capacities to allow neuropsychological testing.

5. Patient and caregiver are able to read and understand informed consent.

6. Patient is on a stable therapy for PSP, MSA for at least 1 month prior to screening
visit.

7. If the patient received i.v. amantadine treatment, the last infusion must have been
administered at least 6 months prior to the screening (V01).

8. Availability of a caregiver who sufficiently knows the patient and will be able to
accompany the patient on the study visits and to participate in study assessments of
the patient where required.

9. Female patients are only eligible for the study if they are either surgically sterile
or at least 2 years postmenopausal or have a negative result of serum hCG test at
screening and apply to criteria no. 10.

10. Female of childbearing potential can only participate in the study if willing to use
acceptable, effective methods of contraception during the trial and for three month
after the end of trial participation as defined in point 6.7. of this protocol.

11. Male patients must either be surgically sterile or he and his female spouse/partner
who is of childbearing potential must be willing to use highly effective methods of
contraception consisting of 2 forms of birth control (1 of which must be a barrier
method) starting at screening and continuing throughout the study.

12. Patient provides written informed consent.

Exclusion Criteria:

1. Known history or presence of clinically significant other neurologic, hematologic,
endocrine, oncologic, pulmonary, immunologic, genitourinary, psychiatric, or
cardiovascular disease or any other condition which, in the opinion of the
Investigator, would jeopardize the safety of the subject or impact the validity of the
study results.

2. Known or suspected allergy hypersensitivity or idiosyncratic reaction to NBMI or any
other drug substances with similar activity.

3. Patient has known contraindication for MRI imaging such as MRI-incompatible metallic
endoprosthesis or MRI-incompatible stent implantation or other as judged by the
Investigator.

4. Patient has claustrophobia that could prevent MRI imaging

5. History of drug or alcohol addiction requiring treatment.

6. Patient who had previous chronic exposure (within one year before recruitment) to iron
from taking preparations/medications for rising iron

7. History of malabsorption within the last year or presence of clinically significant
gastrointestinal disease or surgery that may affect drug bioavailability, including
but not limited to cholecystectomy.

8. Patient is ridden to bed.

9. Presence of hepatic or renal dysfunction. (SGOT and SGPT and bilirubin > X2 UNL.
creatinine > 1.5mg/dl)

10. Female patient who is pregnant (serum hCG level consistent with pregnancy diagnosis);
or breastfeeding.

11. Participation in a clinical trial that involved administration of an investigational
medicinal product within 90 days prior to drug administration, or recent participation
in a clinical investigation that, in the opinion of the Investigator, would jeopardize
subject safety or the integrity of the study results.

12. Patient has a history with evidence of cerebrovascular disease (ischemic or
haemorrhagic), or diagnosis of possible, probable or definite vascular Parkinsonism or
dementia.

13. Have clinically significant abnormal laboratory values (e.g. liver enzymes)

14. Have clinically significant findings from a physical examination (e.g. fever)

15. Patient has claustrophobia that could prevent him from attending MRI imaging



Age minimum: 40 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Multiple System Atrophy

Progressive Supranuclear Palsy

Intervention(s)

Drug: NBMI

Other: Placebo

Primary Outcome(s)

Changes in Progressive Supranuclear Palsy rating Scale (PSPRS) individual scales scores from baseline in PSP patients compared to placebo treatment [Time Frame: through study completion, an average of 85 days]

Changes in QOL individual scores from baseline by EQ-5D score in PSP patients compared to placebo treatment [Time Frame: through study completion, an average of 85 days]

Changes in QOL individual scores from baseline by MSA questionnaire in MSA patients using Visual Analog score [Time Frame: through study completion, an average of 85 days]

Changes in QOL individual scores from baseline by MSA questionnaire in MSA patients [Time Frame: through study completion, an average of 85 days]

Changes in Non-Motor Symptoms assessment scale (NMSS) individual scale from baseline in MSA patients compared to placebo treatment [Time Frame: through study completion, an average of 85 days]

Changes in Unified Multiple System Atrophy Rating Scale (UMSARS) individual scale from baseline in MSA patients compared to placebo treatment [Time Frame: through study completion, an average of 85 days]

Changes in FAB individual scales scores from baseline in PSP patients compared to placebo treatment [Time Frame: through study completion, an average of 85 days]

Secondary Outcome(s)

Changes in Parkinson's Disease Fatigue Score (PFS) score from Baseline - V1 to V2 (D29) and V1 to V5 (D57) in PSP and MSA patients compared to placebo treatment [Time Frame: through study completion, an average of 85 days]

Frequency, type and severity of adverse events compared to placebo treatment . [Time Frame: through study completion, an average of 85 days]

Percentage of NBMI-treated patients who develop a response to NBMI. [Time Frame: through study completion, an average of 85 days]

Changes in Geriatric depression scale (GDS) (PSP patients) score from Baseline - V1 to V2 (D29) and to V4 (D57) compared to placebo treatment [Time Frame: through study completion, an average of 85 days]

Changes in Beck's Depression Inventory (BDI) scale (MSA patients) score from Baseline V1 to V2 (D29) and to V4 (D57) compared to placebo treatment [Time Frame: through study completion, an average of 85 days]

Secondary ID(s)

EMERA006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors