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Study of AMDX-2011P in Subjects With CAA

Primary Purpose

Cerebral Amyloid Angiopathy

Status
Not yet recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
AMDX-2011P
Sponsored by
Amydis Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional diagnostic trial for Cerebral Amyloid Angiopathy focused on measuring CAA

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: .Diagnosis of hereditary CAA or probable and definite symptomatic or asymptomatic sporadic CAAdiagnosed through genetic testingoraccording to the modified Boston neuroradiological criteria(Table 9), who had undergone at least one brain magnetic resonance imaging (MRI)prior to entry into study. Abnormality consistent with CAA on historical MRI. No other causes of cerebral hemorrhage (brain tumors, arteriovenous malformations, aneurysms, cavernous angiomas). Male and female subjects 18 years and older at the time of signing the informed consent. Ability to undergo retinal imagingfor both eyes Subject or legally authorized representative must provide signed informed consent (or signed assent form) prior to study entry and have the ability and willingness to attend and comply with the necessary study procedures and visits at the study site. For subjects unable to physically sign the informed consent, a guardian or trusted caregiver can sign on their behalfin presence of an independent witness. Contraception use by study subjects of childbearing potential (male and female) and female partners of childrearing potential male subjectsshould be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. For details,refer to Section10.3. Female subjects of childbearing potential and female partners of childbearing potential male subjects must refrain from oocyte donation for up to 30 days after study drug administration. Male subjects must refrain from sperm donation for90 days after study drug administration.1 Females of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day -1. Females not of childbearing potential must be surgically infertile or postmenopausal (defined as cessation of regular menstrual periods for at least 12 months) at Screening. Exclusion Criteria: Presence of any underlying physical or psychological medical condition that, in the opinion of the investigator, would make it unlikely that the subject will complete the study per protocol. Clinically significantlaboratory abnormalitiesassessed by the investigator. Active malignancy and/or history of malignancy in the past 5 years, with the exception of completely excised non-melanoma skin cancer or low-grade cervical intraepithelial neoplasia. Prolonged QTcF (>450 ms for males and >470 ms for females),cardiac arrhythmia, or any clinically significant abnormality in the resting ECG, as judged by the investigator. Presence of any ocular condition that, in the opinion of the investigator, would significantly hinder the ability to detect and quantify hyper-fluorescent puncta (e.g., eyes with significant hyper-autofluorescence that would mask the ability to detect, quantify, and discern post-injection hyper-fluorescent signal from pre-injection hyper-autofluorescence signal). These conditions may include, but are not limited to; age-related macular degeneration, central serous chorioretinopathy, diabetic retinopathy, macular dystrophies such as Stargardt disease, retinitis pigmentosa, choroideremia, white dot syndromes, and drug toxicities such as hydroxychloroquine toxicity. Use of any new prescription therapies or vaccines within 7days prior to study drug administration. Drugs with potential phototoxicity per Package Insert are prohibited within 48hoursor 5half-lives, whichever is longer, prior to first study drug until End-of-Study (EOS)visit, except for those required for treatment of underlying disease. Examples of such drugs include the following: Chloroquine (Aralen), hydroxychloroquine (Plaquenil), Thioridazine (Mellaril), Topiramate (Topamax), vemurafenib, voriconazole, doxycycline, hydrochlorothiazide, amiodarone, furosemide, allopurinol, phenothiazine, and chlorpromazine. Administration of investigational product in another study within 30 days prior to the first study drug administration, or five half-lives, whichever is longer. Females who are pregnant or breastfeeding.

Sites / Locations

  • Eye Research Foundation
  • California Eye Specialists

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

AMDX-2011P 50 mg

AMDX-2011P 100 mg

AMDX-2011P 150 mg or 200 mg

AMDX-2011P TBD

Arm Description

AMDX2011P 50mg (2ml) single bolus injection intravenous for diagnostic review

AMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review

AMDX2011P 200mg (6-8ml) single bolus injection intravenous for diagnostic review

AMDX2011P (dose TBD) single bolus injection intravenous for diagnostic review

Outcomes

Primary Outcome Measures

AMDX-2011P adverse events profile
Rate and nature of adverse events after a single intravenous (IV) dose of AMDX-2011P in subjects with CAA

Secondary Outcome Measures

Detection of Amyloid Deposits in the Retina After AMDX-2011P Administration
The presence of hyperfluorescent spots in retinal images
Concentration of AMDX-2011P
Peak plasma concentration (Cmax)
Pharmacokinetic Concentration
Area under the plasma concentration versus time curve (AUC)

Full Information

First Posted
January 24, 2023
Last Updated
July 27, 2023
Sponsor
Amydis Inc.
Collaborators
National Institutes of Health (NIH)
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1. Study Identification

Unique Protocol Identification Number
NCT05709314
Brief Title
Study of AMDX-2011P in Subjects With CAA
Official Title
Open Label, Blinded Endpoint Assessment Study of AMDX-2011P as a Retinal Tracer in Subjects With Cerebral Amyloid Angiopathy (CAA)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
September 2023 (Anticipated)
Primary Completion Date
April 2024 (Anticipated)
Study Completion Date
July 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amydis Inc.
Collaborators
National Institutes of Health (NIH)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this research study is to assess safety and tolerability of a single intravenous (given through a vein) dose of the investigational retinal tracer AMDX-2011P in patients with cerebral amyloid angiopathy (CAA).
Detailed Description
This open, blinded endpoint assessment study will evaluate the activity, safety, tolerability, and pharmacokinetics (PK) of escalating intravenous (IV) doses of AMDX-2011P in CAA subjects. Assessments of retinal images will be conducted by central masked assessors. To determine the safest dose, participants will receive different amounts of the investigational retinal tracer. The first group of participants taking part in the study will receive a low dose of AMDX-2011P. If no major side effects occur, the dose will be increased for the next group of participants. Participants will receive a 1 time intravenous injection. This study plans to enroll up to 20 adult (=/>18 years of age) subjects diagnosed with hereditary CAA or probable or definite CAA according to the modified Boston neuroradiological criteria.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Amyloid Angiopathy
Keywords
CAA

7. Study Design

Primary Purpose
Diagnostic
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Dose escalating via Cohorts (4 total cohorts)
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
AMDX-2011P 50 mg
Arm Type
Experimental
Arm Description
AMDX2011P 50mg (2ml) single bolus injection intravenous for diagnostic review
Arm Title
AMDX-2011P 100 mg
Arm Type
Experimental
Arm Description
AMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review
Arm Title
AMDX-2011P 150 mg or 200 mg
Arm Type
Experimental
Arm Description
AMDX2011P 200mg (6-8ml) single bolus injection intravenous for diagnostic review
Arm Title
AMDX-2011P TBD
Arm Type
Experimental
Arm Description
AMDX2011P (dose TBD) single bolus injection intravenous for diagnostic review
Intervention Type
Drug
Intervention Name(s)
AMDX-2011P
Intervention Description
AMDX-2011P single bolus injection intravenous for diagnostic review
Primary Outcome Measure Information:
Title
AMDX-2011P adverse events profile
Description
Rate and nature of adverse events after a single intravenous (IV) dose of AMDX-2011P in subjects with CAA
Time Frame
1 week
Secondary Outcome Measure Information:
Title
Detection of Amyloid Deposits in the Retina After AMDX-2011P Administration
Description
The presence of hyperfluorescent spots in retinal images
Time Frame
6 hours
Title
Concentration of AMDX-2011P
Description
Peak plasma concentration (Cmax)
Time Frame
6 hours
Title
Pharmacokinetic Concentration
Description
Area under the plasma concentration versus time curve (AUC)
Time Frame
6 hours

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: .Diagnosis of hereditary CAA or probable and definite symptomatic or asymptomatic sporadic CAAdiagnosed through genetic testingoraccording to the modified Boston neuroradiological criteria(Table 9), who had undergone at least one brain magnetic resonance imaging (MRI)prior to entry into study. Abnormality consistent with CAA on historical MRI. No other causes of cerebral hemorrhage (brain tumors, arteriovenous malformations, aneurysms, cavernous angiomas). Male and female subjects 18 years and older at the time of signing the informed consent. Ability to undergo retinal imagingfor both eyes Subject or legally authorized representative must provide signed informed consent (or signed assent form) prior to study entry and have the ability and willingness to attend and comply with the necessary study procedures and visits at the study site. For subjects unable to physically sign the informed consent, a guardian or trusted caregiver can sign on their behalfin presence of an independent witness. Contraception use by study subjects of childbearing potential (male and female) and female partners of childrearing potential male subjectsshould be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. For details,refer to Section10.3. Female subjects of childbearing potential and female partners of childbearing potential male subjects must refrain from oocyte donation for up to 30 days after study drug administration. Male subjects must refrain from sperm donation for90 days after study drug administration.1 Females of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day -1. Females not of childbearing potential must be surgically infertile or postmenopausal (defined as cessation of regular menstrual periods for at least 12 months) at Screening. Exclusion Criteria: Presence of any underlying physical or psychological medical condition that, in the opinion of the investigator, would make it unlikely that the subject will complete the study per protocol. Clinically significantlaboratory abnormalitiesassessed by the investigator. Active malignancy and/or history of malignancy in the past 5 years, with the exception of completely excised non-melanoma skin cancer or low-grade cervical intraepithelial neoplasia. Prolonged QTcF (>450 ms for males and >470 ms for females),cardiac arrhythmia, or any clinically significant abnormality in the resting ECG, as judged by the investigator. Presence of any ocular condition that, in the opinion of the investigator, would significantly hinder the ability to detect and quantify hyper-fluorescent puncta (e.g., eyes with significant hyper-autofluorescence that would mask the ability to detect, quantify, and discern post-injection hyper-fluorescent signal from pre-injection hyper-autofluorescence signal). These conditions may include, but are not limited to; age-related macular degeneration, central serous chorioretinopathy, diabetic retinopathy, macular dystrophies such as Stargardt disease, retinitis pigmentosa, choroideremia, white dot syndromes, and drug toxicities such as hydroxychloroquine toxicity. Use of any new prescription therapies or vaccines within 7days prior to study drug administration. Drugs with potential phototoxicity per Package Insert are prohibited within 48hoursor 5half-lives, whichever is longer, prior to first study drug until End-of-Study (EOS)visit, except for those required for treatment of underlying disease. Examples of such drugs include the following: Chloroquine (Aralen), hydroxychloroquine (Plaquenil), Thioridazine (Mellaril), Topiramate (Topamax), vemurafenib, voriconazole, doxycycline, hydrochlorothiazide, amiodarone, furosemide, allopurinol, phenothiazine, and chlorpromazine. Administration of investigational product in another study within 30 days prior to the first study drug administration, or five half-lives, whichever is longer. Females who are pregnant or breastfeeding.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Masoud Mokhtarani, MD
Phone
310-229-5710
Email
masoud@amydis.com
First Name & Middle Initial & Last Name or Official Title & Degree
Joyce Simpauco
Email
joyce@amydis.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Masoud Mokhtarani, MD
Organizational Affiliation
Amydis Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
Eye Research Foundation
City
Newport Beach
State/Province
California
ZIP/Postal Code
92663
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Linda Wirta
Phone
949-650-1863
Email
info@drwirta.com
Facility Name
California Eye Specialists
City
Pasadena
State/Province
California
ZIP/Postal Code
91107
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brittany Nicholl
Phone
626-305-9100
Email
bnicholl@azulvision.com

12. IPD Sharing Statement

Plan to Share IPD
No

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Study of AMDX-2011P in Subjects With CAA

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