A Randomized, Placebo-controlled, Double-blind, Parallel group, 6 Month Study to Evaluate the Safety, Tolerability, and Potential Efficacy of Monthly Trappsol® Cyclo™ (hydroxypropyl beta cyclodextrin, HPβCD) Infusions in Patients With Early Alzheimer’s Disease

2022-500828-30-00 Protocol CTD-TCAD-501 Therapeutic exploratory (Phase II) Not authorised

Status Not authorised · 2 EU/EEA countries · 2 sites · Protocol CTD-TCAD-501

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)
Status Not authorised
Participants planned 90
Countries 2
Sites 2

Early Alzheimer´s Disease

• Assess the safety and tolerability of Trappsol Cyclo over 24 weeks in patients with EAD • Explore the potential efficacy of Trappsol Cyclo over 24 weeks in patients with EAD • Determine the pharmacokinetics (PK) of Trappsol Cyclo over 24 weeks in patients with EAD

Key facts

Sponsor
Cyclo Therapeutics Inc.
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male and Female
Therapeutic area
Diseases [C] - Nervous System Diseases [C10]
Decision date (initial)
2022-09-19
Transition trial
No
Low-intervention
No
Rare-disease indication
No
Vulnerable population
Yes
Funding sources
Cyclo Therapeutics, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Efficacy

• Assess the safety and tolerability of Trappsol Cyclo over 24 weeks in patients with EAD
• Explore the potential efficacy of Trappsol Cyclo over 24 weeks in patients with EAD
• Determine the pharmacokinetics (PK) of Trappsol Cyclo over 24 weeks in patients with EAD

Conditions and MedDRA coding

Early Alzheimer´s Disease

VersionLevelCodeTermSystem organ class
20.0 LLT 10001896 Alzheimer's disease 10029205

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

  1. For the complete list please refer to the protocol section 5.1. Patients will be eligible to participate in this study if they meet ALL of the following criteria: 1. Male or female 50 to 80 years of age, at the time of consent. a. Have at least an eighth-grade education. b. Primary language for United States-based patients must be English and patients should be able to understand and read English at a level that will ensure adequate completion of study procedures including all cognitive tests. 2. MCI due to AD (Stage 3) according to the FDA Guidance for Industry on Early Alzheimer’s Disease: Developing Drugs for Treatment:69

Exclusion criteria 1

  1. For the complete list please refer to the protocol section 5.2. Patients will not be eligible to participate in this study if they meet any of the following exclusion criteria: 1. Clinically significant renal disease that in the opinion of the Investigator precludes the patient from participating in the study. 2. Evidence of a neurodegenerative disease other than AD or any medical history or brain imaging abnormality that might cause the dementia in the opinion of the Investigator, including, but not limited to vascular dementia, chronic traumatic encephalopathy, or uncontrolled major depressive disorder (MDD). 3. Severe hypothyroidism as defined as hypothyroidism that is refractory to standard of care and with no or inadequate response to clinically relevant dosage of levothyroxine as determined by thyroid-stimulating hormone (TSH), T3 and T4, and presentation of clinical signs and symptoms.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Full details of primary endpoint being Safety and Pharmacokinetic Endpoints are outlined in section 3.1 and 3.4 of the Study Protocol (CTD-TCAD-501, Version 2.0, Final, 31 Mar 2022). • Incidence and severity of TEAEs • Adverse events of special interest (AESIs): o MRI assessments of ARIA-H or ARIA E o Hearing loss based audiological examination o Infusion reactions • Vital signs and/or physical examinations • Safety clinical laboratory values (hematology, clinical chemistry, and urinalysis) i

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Trappsol Cyclo

PRD9672704 · Product

Active substance
Hydroxypropylbetadex
Substance synonyms
HYDROXYPROPYL-BETADEX, 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN, HYDROXYPROPYL BETADEX
Pharmaceutical form
SOLUTION FOR INFUSION
Route of administration
INFUSION
Max daily dose
1000 mg/Kg milligram(s)/kilogram
Max total dose
7000 mg/Kg milligram(s)/kilogram
Max treatment duration
24 Week(s)
Authorisation status
Not Authorised
MA holder
CYCLO THERAPEUTICS INC
Paediatric formulation
No
Orphan designation
No

Placebo 1

Sodium chloride 0.45% solution for infusion

N/A · Product

Other product name
N/A
Pharmaceutical form
N/A
ATC code
N/A — N/A
Marketing authorisation
N/A
MA holder
N/A
MA country
Iceland
Paediatric formulation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Cyclo Therapeutics Inc.

2 Total trials 1 Ended
Academic / Non-commercial
Sponsor organisation
Cyclo Therapeutics Inc.
Address
6714 Northwest 16th Street
City
Gainesville
Postcode
32653-3975
Country
United States

Scientific contact point

Organisation
Cyclo Therapeutics Inc.
Contact name
Lise Kjems Chief Medical Officer

Public contact point

Organisation
Cyclo Therapeutics Inc.
Contact name
Lise Kjems Chief Medical Officer

Third parties 2

OrganisationCity, countryDuties
Specialized Medical Services-Oncology B.V.
ORG-100027147
Schiphol, Netherlands On site monitoring, Code 10, Code 11, Code 12, Code 13, Code 2, Code 5, Data management, E-data capture, Code 8
Acm Global Central Laboratory Limited
ORG-100042459
York, United Kingdom Laboratory analysis

Locations

2 EU/EEA countries · 2 investigational sites

By country

CountryMS statusPlanned subjectsSites
Austria Not authorised 30 1
Germany Not authorised 30 1
Rest of world
United States
30

Investigational sites

Austria

1 site · Not authorised
Medical University Of Graz
Universitätsklinik fur Neurologie, Auenbruggerplatz 2, 8036, Graz

Germany

1 site · Not authorised
University Hospital Cologne AöR
Klinik und Poliklinik for Neurologie, Kerpener Strasse 62, Lindenthal, Cologne

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2022-05-25 Germany Not acceptable
2022-09-12
2022-09-16