124I-p5+14 Injection Safety in Subjects With Systemic Amyloidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 4 April 2022
• Main ID: NCT03678259
• Date of registration: 30/07/2018
• Prospective Registration: Yes
• Primary sponsor: University of Tennessee Graduate School of Medicine
• Public title: 124I-p5+14 Injection Safety in Subjects With Systemic Amyloidosis
• Scientific title: Evaluation of 124I-p5+14 Injection as an Imaging Agent for the Detection of Systemic Amyloidosis
• Date of first enrolment: October 1, 2018
• Target sample size: 57
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03678259
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Diagnostic. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Jonathan Wall, Ph.D.
• Affiliation: UTHSC Graduate School of Medicine

Key inclusion & exclusion criteria

Subjects included in this trial are volunteers with a confirmed diagnosis of systemic
amyloidosis, a genetically well-defined mutation of the TTR gene but without clinical
evidence of active disease, and at an age typical of development of symptomology = 50, or
healthy control subjects (=30 years).

Prior to participation in Parts 1 or 2 of the trial (n = 73), the following inclusion
criteria must be met

Subjects included in this trial are volunteers with a confirmed diagnosis of systemic
amyloidosis, a genetically well-defined mutation of the TTR gene but without clinical
evidence of active disease, and at an age typical of development of symptomology = 50, or
healthy control subjects (=30 years).

Prior to participation in Parts 1 or 2 of the trial (n = 73), the following inclusion
criteria must be met:

1. Patients must have a confirmed diagnosis of systemic amyloidosis, based on either a
histologic confirmation that a biopsy contains deposits of apple-green birefringent,
Congophilic material or genetic screening and presence of amyloid-related pathology,
or amyloid-specific imaging study. Additionally, the type of amyloidosis (AL, ATTR,
ALect2, or other) should be characterized.

2. Patients enrolled in Part 1 (n = 3) must have widespread AL amyloidosis, defined as
biopsy proven or clinically detectable involvement, of at least two organs (excluding
the peripheral nervous system).

3. All patients in Parts 1 and 2 will be 18 years of age or older. There are no gender or
racial restrictions.

4. Women of child bearing potential (those who have not been surgically sterilized, are
not postmenopausal [typically understood to mean last menstrual period >2 y ago
without pharmaceutical intervention], and women who are fertile) must test negative
for pregnancy in a laboratory test administered by the site physician.

5. Patients who have had or are currently receiving therapy or other drug based
anti-amyloid regimens can be included on study (Parts 1 and 2).

6. Patients must provide signed, written, informed consent and be willing to comply with
eligibility requirements, scheduled visits, and follow-up studies.

7. Due to annual dosimetry limitations, patients who have participated in another nuclear
medicine amyloid imaging clinical trial protocol can be included in this study no
earlier than 12 months after the previous radiotracer injection.

8. In Part 2, inclusion of patients with amyloid subsets AL, ATTR, and ALect2 will
continue until the trial has achieved recruitment goals for each subset: 30 AL; 20
ATTR; 5 ALect2; and 10 15 "Other".

Prior to participation in Part 3 (n = 10 subjects) of the trial, the following inclusion
criteria must be met:

1. Patients must have a well-defined germline mutation of the transthyretin (TTR) gene
rendering them at risk for amyloidosis, and be free of clinical evidence of SA.

2. All patients in Part 3 will be >50 years of age. There are no gender or racial
restrictions.

3. Women of child bearing potential (those who have not been surgically sterilized, are
not postmenopausal [typically understood to mean last menstrual period >2 y ago
without pharmaceutical intervention], and women who are fertile) must test negative
for pregnancy in a laboratory test administered by the site physician.

4. Subjects must provide signed, written, informed consent and be willing to comply with
eligibility requirements, scheduled visits, and follow-up studies.

5. Due to annual dosimetry limitations, subjects who have participated in another nuclear
medicine amyloid imaging clinical trial protocol can be included in this study no
earlier than 12 months after the previous radiotracer injection.

Prior to participation Part 4 of the trial (n = 5), the following inclusion criteria must
be met:

1. Healthy Control Subjects (HC) will be generally healthy adults, either male or female,
and will not have a diagnosis of amyloidosis, will not have a first- or second-degree
relative (parent, sibling, child, aunt, uncle, niece, nephew) with confirmed or
suspected familial amyloidosis, and will not have diabetes mellitus (type 2).

2. All Part 4 subjects will be > 30 years of age or older.

3. Women of child bearing potential (those who have not been surgically sterilized, are
not postmenopausal [typically understood to mean last menstrual period >2 y ago
without pharmaceutical intervention], and women who are fertile) must test negative
for pregnancy in a laboratory test administered by the site physician.

4. Patients must provide signed, written, informed consent and be willing to comply with
eligibility requirements, scheduled visits, and follow-up studies.

5. Due to annual dosimetry limitations, patients who have participated in another nuclear
medicine imaging clinical trial protocol can be included in this study no earlier than
12 months after the previous radiotracer injection.

Prior to participation in Part 5 of the trial, the following inclusion criteria must be
met:

1. Patients must have successfully completed participation in Part 2 or Part 3 of this
trial with CT/PET images confirming the presence of abnormal amyloid deposits in
thoracoabdominal organs.

2. Patients must have received a 2 mCi dose of 124I-p5+14 Injection during Part 2 or 3 of
this trial with visual evidence of uptake of radiotracer in abdominothoracic organs
associated with amyloid.

3. The repeat exposure to the study agent must occur at least 6 months after the first
exposure.

4. Patients must submit a serum specimen for exploratory evaluation of the presence of
anti-p5+14 peptide antibodies, with results reviewed by the Principal Investigator
prior to a second exposure to the study agent.

5. The patient must meet all of the Inclusion criteria for participation in Part 2 or
Part 3 outlined in Sections 5.2 or Section 5.3 (as applicable), including the signing
of an informed consent for Part 5.

In addition, the following criteria will exclude candidates from participation in the
trial, Parts 1 and 2:

1. Those with significant co-morbidity (e.g., Eastern Cooperative Oncology Group [ECOG]
score of 3 or greater), uncontrolled infection, or other serious illness.

2. Patients with a sustained SpO2 of = 92% as noted in the medical record.

3. Patients that require renal dialysis.

4. Women who are of child bearing potential (those who have not been surgically
sterilized, are not postmenopausal [typically understood to mean last menstrual period
>2 y ago without pharm


Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Systemic Amyloidosis

Intervention(s)

Drug: 124I-p5+14 Injection

Primary Outcome(s)

Organ-specific radioactivity dosimetry (Part 1). [Time Frame: Through 48 hours]

Secondary Outcome(s)

Organ and tissue-specific sensitivity of the 124I-p5+14 Injection radiotracer [Time Frame: 6 hours and 24 hours]

Clinically defined amyloidosis organ involvement. [Time Frame: Baseline]

Concentration of radiotracer in specific anatomic sites, for each subject and anatomic site [Time Frame: 6 hours and 24 hours]

Correlation between concentration of the radiotracer (Bq/cc) in the heart with organ-associated clinical biomarkers. [Time Frame: 6 hours and 24 hours]

Correlation between uptake of peptide and clinical status of kidney, heart and other organs [Time Frame: Baseline and 24 hours]

Measure of background radioactivity uptake. [Time Frame: 6 hours and 24 hours]

Peptide uptake in the kidney [Time Frame: 6 hours and 24 hours]

Measure of radioactivity uptake by each organ [Time Frame: 6 hours and 24 hours]

Peptide uptake in organ(s) other than kidney or heart if clinically relevant [Time Frame: 6 hours and 24 hours]

Absolute values and changes from baseline in clinical laboratory values. [Time Frame: Baseline and 24 hours]

Correlation between concentration of the radiotracer (Bq/cc) in the kidney with organ-associated clinical biomarkers. [Time Frame: 6 hours and 24 hours]

Peptide uptake in the heart. [Time Frame: 6 hours and 24 hours]

Secondary ID(s)

AMY1001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors