Ignite Creation Date:
2024-07-17 @ 12:02 PM
Last Modification Date:
2024-10-26 @ 3:34 PM
Study NCT ID:
NCT06487481
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-07-15
First Post:
2024-07-03
Brief Title:
National Ophthalmic Genotyping and Phenotyping Network eyeGENER Stage 3-Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases
Sponsor:
National Cancer Institute NCI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Phase I Dose-escalation Study of Preoperative Radiation Therapy in Participants With Resectable Recurrent Abdominal Adrenocortical Carcinoma
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background
The eyeGENER program is a research resource for inherited eye conditions which includes genotypic and phenotypic data imaging and a corresponding biobank of DNA samples from people with a variety of eye diseases Since 2007 this registry has been helping researchers learn more about the genetic sources for many inherited eye diseases These findings helped them create better treatments Now researchers want to expand eyeGENER to include more people for certain eye diseases
Objective To collect information and DNA samples for the study of eye diseases
Primary objective
-To expand the current eyeGENER data repository with targeted participant accrual
Secondary objectives
To enhance recruitment for clinical trials and investigations in inherited eye diseases
To establish genotype-phenotype correlations for rare eye diseases
Eligibility
People of any age with certain eye diseases These can include aniridia Best disease blue-cone monochromacy corneal dystrophy and disorders of pigmentation such as albinism Relatives unaffected by the eye disease of interest may also be needed
Design
Researchers will select participants based on their diagnosis The data may include images and test results from eye exams
Participants will provide a sample of saliva They will receive a kit with written instructions They will spit in a tube and mail it to the NIH
Participants may be asked to provide a blood sample The blood may be drawn at the NIH or at a local clinic
The eyeGENER repository will offer researchers data about the participants eye conditions The data may include pictures of their eyes results of genetic testing and history of other diseases Researchers will be able to see data such as age and gender but they will not see names dates of birth or contact information
The eyeGENER program is a research resource for inherited eye conditions which includes genotypic and phenotypic data imaging and a corresponding biobank of DNA samples from people with a variety of eye diseases Since 2007 this registry has been helping researchers learn more about the genetic sources for many inherited eye diseases These findings helped them create better treatments Now researchers want to expand eyeGENER to include more people for certain eye diseases
Objective To collect information and DNA samples for the study of eye diseases
Primary objective
-To expand the current eyeGENER data repository with targeted participant accrual
Secondary objectives
To enhance recruitment for clinical trials and investigations in inherited eye diseases
To establish genotype-phenotype correlations for rare eye diseases
Eligibility
People of any age with certain eye diseases These can include aniridia Best disease blue-cone monochromacy corneal dystrophy and disorders of pigmentation such as albinism Relatives unaffected by the eye disease of interest may also be needed
Design
Researchers will select participants based on their diagnosis The data may include images and test results from eye exams
Participants will provide a sample of saliva They will receive a kit with written instructions They will spit in a tube and mail it to the NIH
Participants may be asked to provide a blood sample The blood may be drawn at the NIH or at a local clinic
The eyeGENER repository will offer researchers data about the participants eye conditions The data may include pictures of their eyes results of genetic testing and history of other diseases Researchers will be able to see data such as age and gender but they will not see names dates of birth or contact information
Detailed Description:
Background
Although surgical resection is the treatment of choice in participants with localized or regionalized primary and recurrent abdominal adrenocortical carcinoma ACC loco-regional recurrence following complete resection of ACC occurs in 50-80 of the participants most commonly in the first five years postoperatively
Retrospective single institutional series report improvement in local control with postoperative adjuvant radiotherapy RT in selected participants with ACC
Preoperative RT is used in several abdominal and retroperitoneal malignancies to improve local control There is no prospective data available for the use of this treatment approach for participants with resectable ACC
We hypothesize that preoperative RT alone is safe and can result in a lower loco-regional recurrence in participants with resectable recurrent ACC with no peritoneal carcinomatosis Thus this phase I dose-escalation trial aims to evaluate the safety and feasibility of preoperative radiation in participants with resectable recurrent ACC
Health-related quality of life QOL is a well-accepted tool to measure the outcome of cancer treatments SF-36 v2 questionnaire has been frequently used to evaluate the QOL in participants with cancer There is no study evaluating the difference in QOL in participants with resectable recurrent ACC undergoing preoperative RT and surgery
Objectives
-To determine the maximum tolerated dose and the safety and toxicity profile of preoperative external beam radiation therapy EBRT with or without standard of care mitotane before surgical resection in participants with resectable ACC
Eligibility
Age 18 years
Pathological confirmation of ACC with clinical evidence of abdominal recurrence
ECOG 0-2
Surgically resectable disease at presentation with no or limited extra-abdominal disease and without ACC peritoneal carcinomatosis based on a diagnostic laparoscopy at screening
The last dose of chemotherapy treatment except for mitotane more than 4 weeks prior to starting treatment with this protocol and participants must have recovered from chemotherapy
No prior abdominal radiation
No contraindication to abdominal radiotherapy
Design
This study will enroll up to 24 evaluable participants as follows
Participants will be enrolled in Cohort 1 or 2 based on mitotane use or serum level Up to 6-18 evaluable participants per these cohorts ie resectable ACC with and without mitotane use will be enrolled to assess the safety of 3-level dose-escalating preoperative EBRT
Participants will be enrolled in Cohort 3 regardless of mitotane use or serum level and will only start enrollment when we observe no DLTs in 1 participant in Cohort 1 at Dose Level 1 Up to 12 evaluable participants will be enrolled in this cohort
Preoperative assessment of QOL using a standardized questionnaire SF-36 v2 will be obtained at baseline
Participants will be treated with preoperative external beam radiation therapy EBRT daily 5 daysweek M-F followed by a planned surgical resection 4-8 weeks after the completion of EBRT
A standard 3 3 design will be used to determine the MTD of dose-escalated EBRT with 3 dose levels DL1- 30 Gy delivered in 10 fractions DL2-36 Gy in 12 fractions DL3-42 Gy in 14 fractions
Postoperative surveillance imaging studies and laboratory tests will be performed every 3 months in the first 3 years then every 6 months thereafter in years 4-10 FDG-PET scan will be performed every 6 months postoperatively in the first 3 years then every year in years 4-10 Additional assessments may be performed if clinically indicated
Although surgical resection is the treatment of choice in participants with localized or regionalized primary and recurrent abdominal adrenocortical carcinoma ACC loco-regional recurrence following complete resection of ACC occurs in 50-80 of the participants most commonly in the first five years postoperatively
Retrospective single institutional series report improvement in local control with postoperative adjuvant radiotherapy RT in selected participants with ACC
Preoperative RT is used in several abdominal and retroperitoneal malignancies to improve local control There is no prospective data available for the use of this treatment approach for participants with resectable ACC
We hypothesize that preoperative RT alone is safe and can result in a lower loco-regional recurrence in participants with resectable recurrent ACC with no peritoneal carcinomatosis Thus this phase I dose-escalation trial aims to evaluate the safety and feasibility of preoperative radiation in participants with resectable recurrent ACC
Health-related quality of life QOL is a well-accepted tool to measure the outcome of cancer treatments SF-36 v2 questionnaire has been frequently used to evaluate the QOL in participants with cancer There is no study evaluating the difference in QOL in participants with resectable recurrent ACC undergoing preoperative RT and surgery
Objectives
-To determine the maximum tolerated dose and the safety and toxicity profile of preoperative external beam radiation therapy EBRT with or without standard of care mitotane before surgical resection in participants with resectable ACC
Eligibility
Age 18 years
Pathological confirmation of ACC with clinical evidence of abdominal recurrence
ECOG 0-2
Surgically resectable disease at presentation with no or limited extra-abdominal disease and without ACC peritoneal carcinomatosis based on a diagnostic laparoscopy at screening
The last dose of chemotherapy treatment except for mitotane more than 4 weeks prior to starting treatment with this protocol and participants must have recovered from chemotherapy
No prior abdominal radiation
No contraindication to abdominal radiotherapy
Design
This study will enroll up to 24 evaluable participants as follows
Participants will be enrolled in Cohort 1 or 2 based on mitotane use or serum level Up to 6-18 evaluable participants per these cohorts ie resectable ACC with and without mitotane use will be enrolled to assess the safety of 3-level dose-escalating preoperative EBRT
Participants will be enrolled in Cohort 3 regardless of mitotane use or serum level and will only start enrollment when we observe no DLTs in 1 participant in Cohort 1 at Dose Level 1 Up to 12 evaluable participants will be enrolled in this cohort
Preoperative assessment of QOL using a standardized questionnaire SF-36 v2 will be obtained at baseline
Participants will be treated with preoperative external beam radiation therapy EBRT daily 5 daysweek M-F followed by a planned surgical resection 4-8 weeks after the completion of EBRT
A standard 3 3 design will be used to determine the MTD of dose-escalated EBRT with 3 dose levels DL1- 30 Gy delivered in 10 fractions DL2-36 Gy in 12 fractions DL3-42 Gy in 14 fractions
Postoperative surveillance imaging studies and laboratory tests will be performed every 3 months in the first 3 years then every 6 months thereafter in years 4-10 FDG-PET scan will be performed every 6 months postoperatively in the first 3 years then every year in years 4-10 Additional assessments may be performed if clinically indicated
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 001587-C | None | None | None |