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SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome (RESCUE)

Primary Purpose

Cushing's Syndrome I, Cushing Disease Due to Increased ACTH Secretion, Cortisol Excess

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
SPI-62
Placebo
Sponsored by
Sparrow Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cushing's Syndrome I focused on measuring Cushing's Syndrome, ACTH Secretion, Cushing's Disease, Excessive Cortisol secretion, Cortisol, ectopic CRH secretion

Eligibility Criteria

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

Inclusion Criteria:

  • Male or non-menstruating female
  • 18 years or older
  • Active and consistent cortisol excess
  • Documented diagnosis of ACTH-dependent Cushing's syndrome including Cushing's disease, ectopic ACTH secretion, and ectopic CRH secretion.

Exclusion Criteria:

  • Recent (within 6 weeks) surgery for Cushing's or surgery planned within 24 weeks of randomization.
  • History of any fractionated radiation therapy for Cushing's within the past 2 years or conventional radiation therapy within 4 years.
  • History of bilateral adrenalectomy or exogenous, pseudo, cyclic, or non-ACTH-dependent Cushing's syndrome (including certain inherited conditions).
  • High risk of acute morbidity from corticotroph adenoma growth (similar to that which occurs with Nelson's syndrome) defined as current evidence of macroadenoma at risk of impingement of vital structures.
  • Any current or prior medical condition, medical or surgical therapies, or clinical trial participation expected to interfere with the conduct of the study or the evaluation of its results, including but not limited to poor venous access or recent receipt or donation of blood products.
  • Women who are currently pregnant, lactating or planning fertility and unwilling to adhere to approved contraceptives or abstinence.

Sites / Locations

  • St. Joseph's Hospital and Medical Center - Barrow Neurological Institute (BNI) - Pituitary CenterRecruiting
  • University of California Los Angeles - Gonda (Goldschmied) Diabetes Center
  • Southwest General Healthcare Center
  • Indiana University of Clinical MedicineRecruiting
  • Mayo Clinic Cancer Center (MCCC) - RochesterRecruiting
  • Washington University School of Medicine - Center for Advanced Medicine (CAM)Recruiting
  • Comprehensive and Interventional Pain Management LlpRecruiting
  • Memorial Sloan-Kettering Cancer CenterRecruiting
  • Diabetes and Endocrinology Associates of Stark County, Inc
  • Oregon Health & Science University (OHSU) - Northwest Pituitary CenterRecruiting
  • University of Pittsburgh Medical Center
  • Medical University of PlovdivRecruiting
  • Clinical Center of Endocrinology and Gerontology, University Hospital of Endocrinology, Medical University Sofia (USHATE)Recruiting
  • Medical University of SofiaRecruiting
  • University Hospital Stara Zagora
  • Carol Davila University of Medicine and PharmacyRecruiting
  • Iuliu Hațieganu University of Medicine and Pharmacy
  • Victor Babes University of Medicine and Pharmacy TimisoaraRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

SPI-62

Placebo

Arm Description

Active drug by mouth each morning for up to 12 weeks

Placebo by mouth each morning for up to 12 weeks

Outcomes

Primary Outcome Measures

Change from baseline in urinary HSD-1 ratio
The urinary HSD-1 ratio (tetrahydrocortisol + allotetrahydrocortisol ) / tetrahydrocortisone will be used as a biomarker of HSD-1 activity in liver. The primary analysis will include only subjects with Cushing's disease.

Secondary Outcome Measures

Treatment emergent adverse events
Adverse events including clinically significant abnormal values on clinical laboratory evaluations, continuous glucose monitoring (CGM), 12-lead ECGs, vital signs measurements (including orthostatic vital sign measurements), physical examinations, and HPA and HPG axis biomarkers

Full Information

First Posted
March 23, 2022
Last Updated
October 20, 2023
Sponsor
Sparrow Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT05307328
Brief Title
SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome
Acronym
RESCUE
Official Title
SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 1, 2022 (Actual)
Primary Completion Date
December 1, 2023 (Anticipated)
Study Completion Date
December 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sparrow Pharmaceuticals

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
This is a randomized, placebo-controlled, study of SPI-62 in subjects with ACTH-dependent Cushing's syndrome caused by a non-adrenal tumor. Subjects will receive each of the following 2 treatments for 24 weeks: SPI-62 and matching placebo with the option of long-term extension.
Detailed Description
This is a multicenter, randomized, placebo-controlled, Phase 2 study to evaluate the pharmacologic effect, efficacy, and safety of SPI-62 in subjects with ACTH-dependent Cushing's syndrome. Each subject who provides consent and meets all inclusion and exclusion criteria will participate in a screening period (Days -35 to -8), a baseline period (Days -7 to -1), and a treatment period (Day 1 of Week 1 to Day 168 ± 3 days of Week 24) and, the option of long-term extension. Subjects have the option to continue with the study on active study drug and return to the site every 3 months for blood tests and study drug dispensing. The visits may be conducted remotely if testing can be arranged.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cushing's Syndrome I, Cushing Disease Due to Increased ACTH Secretion, Cortisol Excess, Cortisol; Hypersecretion, Cortisol Overproduction, Ectopic ACTH Secretion
Keywords
Cushing's Syndrome, ACTH Secretion, Cushing's Disease, Excessive Cortisol secretion, Cortisol, ectopic CRH secretion

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Model Description
Staggered parallel crossover
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
26 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
SPI-62
Arm Type
Experimental
Arm Description
Active drug by mouth each morning for up to 12 weeks
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo by mouth each morning for up to 12 weeks
Intervention Type
Drug
Intervention Name(s)
SPI-62
Intervention Description
11β hydroxysteroid dehydrogenase type 1 (HSD-1) inhibitor
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Inactive tablets identical to SPI-62 tablets
Primary Outcome Measure Information:
Title
Change from baseline in urinary HSD-1 ratio
Description
The urinary HSD-1 ratio (tetrahydrocortisol + allotetrahydrocortisol ) / tetrahydrocortisone will be used as a biomarker of HSD-1 activity in liver. The primary analysis will include only subjects with Cushing's disease.
Time Frame
Baseline to 6 weeks
Secondary Outcome Measure Information:
Title
Treatment emergent adverse events
Description
Adverse events including clinically significant abnormal values on clinical laboratory evaluations, continuous glucose monitoring (CGM), 12-lead ECGs, vital signs measurements (including orthostatic vital sign measurements), physical examinations, and HPA and HPG axis biomarkers
Time Frame
Baseline through 24 weeks of treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or non-menstruating female 18 years or older Active and consistent cortisol excess Documented diagnosis of ACTH-dependent Cushing's syndrome including Cushing's disease, ectopic ACTH secretion, and ectopic CRH secretion. Exclusion Criteria: Recent (within 6 weeks) surgery for Cushing's or surgery planned within 24 weeks of randomization. History of any fractionated radiation therapy for Cushing's within the past 2 years or conventional radiation therapy within 4 years. History of bilateral adrenalectomy or exogenous, pseudo, cyclic, or non-ACTH-dependent Cushing's syndrome (including certain inherited conditions). High risk of acute morbidity from corticotroph adenoma growth (similar to that which occurs with Nelson's syndrome) defined as current evidence of macroadenoma at risk of impingement of vital structures. Any current or prior medical condition, medical or surgical therapies, or clinical trial participation expected to interfere with the conduct of the study or the evaluation of its results, including but not limited to poor venous access or recent receipt or donation of blood products. Women who are currently pregnant, lactating or planning fertility and unwilling to adhere to approved contraceptives or abstinence.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Frank Czerwiec, MD
Phone
+1-617-465-0328
Email
fczerwiec@sparrowpharma.com
First Name & Middle Initial & Last Name or Official Title & Degree
Sarah Hooper
Phone
+1-617-465-0328
Email
shooper@sparrowpharma.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Frank Czerwiec, MD
Organizational Affiliation
Sparrow Pharmaceuticals (info@sparrowpharma.com)
Official's Role
Study Director
Facility Information:
Facility Name
St. Joseph's Hospital and Medical Center - Barrow Neurological Institute (BNI) - Pituitary Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Cathy O'Hagan
Email
mariecatherine.ohagan@dignityhealth.org
First Name & Middle Initial & Last Name & Degree
Kevin Yuen, MD
Facility Name
University of California Los Angeles - Gonda (Goldschmied) Diabetes Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Individual Site Status
Withdrawn
Facility Name
Southwest General Healthcare Center
City
Fort Myers
State/Province
Florida
ZIP/Postal Code
33907
Country
United States
Individual Site Status
Terminated
Facility Name
Indiana University of Clinical Medicine
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Julie Raters
Email
jraters@iu.edu
First Name & Middle Initial & Last Name & Degree
Cary Mariash, MD
Facility Name
Mayo Clinic Cancer Center (MCCC) - Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jasmine Sexton
Email
sexton.jasmine@mayo.edu
First Name & Middle Initial & Last Name & Degree
Irina Bancos, MD
Facility Name
Washington University School of Medicine - Center for Advanced Medicine (CAM)
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Cameron Smith
Email
camerons@wustl.edu
First Name & Middle Initial & Last Name & Degree
Julie Silverstein, MD
Facility Name
Comprehensive and Interventional Pain Management Llp
City
Henderson
State/Province
Nevada
ZIP/Postal Code
89052
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mailyn Marta
Email
mailyn@conductclinicaltrials.com
First Name & Middle Initial & Last Name & Degree
Claudia Vogel, MD
Facility Name
Memorial Sloan-Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Isabelle Grillo
Email
grilloi@mskcc.org
First Name & Middle Initial & Last Name & Degree
Eliza Geer, MD
Facility Name
Diabetes and Endocrinology Associates of Stark County, Inc
City
Canton
State/Province
Ohio
ZIP/Postal Code
44718
Country
United States
Individual Site Status
Terminated
Facility Name
Oregon Health & Science University (OHSU) - Northwest Pituitary Center
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Samantha Yau
Email
yaus@ohsu.edu
First Name & Middle Initial & Last Name & Degree
Maria Fleseriu, MD
Facility Name
University of Pittsburgh Medical Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Natalie David
Email
nld37@pitt.edu
First Name & Middle Initial & Last Name & Degree
Pouneh Fazeli, MD, MPH
Facility Name
Medical University of Plovdiv
City
Plovdiv
ZIP/Postal Code
4002
Country
Bulgaria
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Naira Kisyova
Email
naira.kisyova@clineca.net
First Name & Middle Initial & Last Name & Degree
Maria Orbetzova, MD
Facility Name
Clinical Center of Endocrinology and Gerontology, University Hospital of Endocrinology, Medical University Sofia (USHATE)
City
Sofia
ZIP/Postal Code
1431
Country
Bulgaria
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Milena Paynova
Email
milena_paynova@abv.bg
First Name & Middle Initial & Last Name & Degree
Ivayla Rumenova Uzunova, MD
Facility Name
Medical University of Sofia
City
Sofia
ZIP/Postal Code
1431
Country
Bulgaria
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ginka Todorova
Email
ginka.todorova@clineca.net
First Name & Middle Initial & Last Name & Degree
Zdravko Kamenov, MD
Facility Name
University Hospital Stara Zagora
City
Stara Zagora
ZIP/Postal Code
6003
Country
Bulgaria
Individual Site Status
Withdrawn
Facility Name
Carol Davila University of Medicine and Pharmacy
City
Bucharest
ZIP/Postal Code
050474
Country
Romania
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Paul Lungu
Email
paul.lungu25@yahoo.com
First Name & Middle Initial & Last Name & Degree
Corin Badiu, MD
Facility Name
Iuliu Hațieganu University of Medicine and Pharmacy
City
Cluj-Napoca
ZIP/Postal Code
400347
Country
Romania
Individual Site Status
Withdrawn
Facility Name
Victor Babes University of Medicine and Pharmacy Timisoara
City
Timişoara
ZIP/Postal Code
300041
Country
Romania
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ioana Golu
Email
igolu25@yahoo.com
First Name & Middle Initial & Last Name & Degree
Melania-Olga Balas, MD

12. IPD Sharing Statement

Learn more about this trial

SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome

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