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Safety of SP-420 in the Treatment of Transfusional Iron Overload

Primary Purpose

Iron Overload

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
SP-420
Sponsored by
The University of Texas Health Science Center at San Antonio
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Iron Overload

Eligibility Criteria

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

Inclusion Criteria:

  1. Age ≥18
  2. Diagnosis of MDS or MF with transfusional iron overload
  3. Patients with MDS, will include only those with MDS Revised international prognostic scoring system (IPSS-R) risk group of intermediate, high, or very high.
  4. Patients with MF, will include only those with Dynamic International Prognostic Scoring System-Plus (DIPSS=Plus) risk category of intermediate-1, intermediate-2, and high risk.
  5. Not appropriate for other iron chelation therapy, per physician
  6. Received 10 or more units of packed red blood cells in the preceding 24 months and remains red cell transfusion dependent
  7. ECOG ≤ 3
  8. ALT ≤ 3 times the upper limit of the normal range
  9. Estimate glomerular filtration rate calculated using Cockroft Gault of ≥ 60 mL/min/1.73m2
  10. Serum ferritin ≥1000 ng/ml
  11. Willing to comply with all study procedures and be available for the duration of the study
  12. Able to take oral medication and be willing to adhere to study medication for 28 days
  13. Female patient must be post-menopausal (no menses for > 12 consecutive months) or surgically sterile (i.e., bilateral oophorectomy, hysterectomy, or tubal sterilization; must agree to completely abstain for heterosexual intercourse; or, if sexually active, must agree to use 1 of the following methods for birth control from the date she signs the consent form until 30 days after final dose of the study drug.

    • Progesterone implant
    • Intrauterine device
    • Combination of 2 highly effective birth control methods (e.g., diaphragm/or cervical cap with spermicide plus a condom, hormonal contraception plus a barrier method, partner with vasectomy conducted >60 days before screening visit plus a hormone or barrier method
  14. Male patients must agree to use 1 of the following methods for birth control from the date he signs the consent form until 30 days after final dose of the study drug: be surgically sterile by vasectomy conducted > 60 days before screening visit plus use a barrier method, or, must agree to completely abstain from heterosexual intercourse, or must agree to use a combination of 2 highly effective birth control methods (e.g., diaphragm/or cervical cap with spermicide plus a condom, hormonal contraception plus a barrier method), or have a post-menopausal partner plus barrier method.

Exclusion Criteria:

  1. History of kidney disease including the renal Fanconi syndrome
  2. Proteinuria on urine dipstick greater than trace positive
  3. Pregnant, intending to become pregnant during the study, or breastfeeding
  4. Receiving another investigational drug within 30 days or 3 half-lives of the discontinued investigational agent, whichever is greater, of signing consent
  5. History of significant hepatic impairment, defined by Child-Pugh class C
  6. Active hepatitis B or C disease, evidenced by positive viral PCR
  7. Symptomatic heart failure
  8. Receiving active cytotoxic chemotherapy or radiation therapy for a second malignancy (hormonal therapy or topical therapy for squamous cell/basal cell cutaneous tumors are allowed). Treatment of the underlying hematologic malignancy with azacytidine, decitabine, venetoclax, lenalidomide, or ruxolitinib is permitted. Treatment with the supportive care agents luspatercept or erythropoietin agonists is permitted.
  9. Concurrent treatment with Exjade/Jadenu (deferasirox), Desferal (deferoxamine), or Ferriprox (deferiprone) are not permitted. Patients are allowed to stop these chelators and participate in this trial 14 days after discontinuation of the other chelator.

Sites / Locations

  • Mays Cancer Center, UT Health San AntonioRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

Group A

Group B

Group C

Group D

Arm Description

Study subjects will receive a 14mg/kg starting dose of SP-420 three times a week

Study subjects will receive a 28mg/kg starting dose of SP-420 three times a week

Study subjects will receive a 42mg/kg starting dose of SP-420 three times a week

Study subjects will receive a 56mg/kg starting dose of SP-420 three times a week

Outcomes

Primary Outcome Measures

Number of adverse events
Count of adverse events induced by SP-420
Completion at original dose
Number of subjects that completed the study at the original starting dose of that group

Secondary Outcome Measures

Full Information

First Posted
January 19, 2021
Last Updated
January 27, 2023
Sponsor
The University of Texas Health Science Center at San Antonio
Collaborators
Abfero Pharmaceuticals, Inc
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1. Study Identification

Unique Protocol Identification Number
NCT04741542
Brief Title
Safety of SP-420 in the Treatment of Transfusional Iron Overload
Official Title
Phase 1, Open-Label, Dose Escalation Study to Assess the Safety of SP-420 in the Treatment of Transfusional Iron Overload
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 9, 2021 (Actual)
Primary Completion Date
August 2023 (Anticipated)
Study Completion Date
August 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
The University of Texas Health Science Center at San Antonio
Collaborators
Abfero Pharmaceuticals, Inc

4. Oversight

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

5. Study Description

Brief Summary
This study enrolls patients with myelodysplastic syndrome (MDS) and myelofibrosis (MFS), with transfusional iron overload and treats them with the investigational iron chelator, SP-420. SP-420 may be better tolerated and safer than commercially available iron chelators. Iron chelation therapy (ICT) has been shown to improve outcomes in iron overload, but adherence is poor due to problems related to ease of administration, tolerability, and safety.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
28 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Group A
Arm Type
Experimental
Arm Description
Study subjects will receive a 14mg/kg starting dose of SP-420 three times a week
Arm Title
Group B
Arm Type
Experimental
Arm Description
Study subjects will receive a 28mg/kg starting dose of SP-420 three times a week
Arm Title
Group C
Arm Type
Experimental
Arm Description
Study subjects will receive a 42mg/kg starting dose of SP-420 three times a week
Arm Title
Group D
Arm Type
Experimental
Arm Description
Study subjects will receive a 56mg/kg starting dose of SP-420 three times a week
Intervention Type
Drug
Intervention Name(s)
SP-420
Other Intervention Name(s)
(4S)-4,5-dihydro-2-[2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl]-4-methyl-4-thiazolecarboxylic acid
Intervention Description
This study aims to establish the safety of SP-420 administered orally three times per week (TIW).
Primary Outcome Measure Information:
Title
Number of adverse events
Description
Count of adverse events induced by SP-420
Time Frame
28 Days
Title
Completion at original dose
Description
Number of subjects that completed the study at the original starting dose of that group
Time Frame
28 Days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age ≥18 Diagnosis of MDS or MF with transfusional iron overload Patients with MDS, will include only those with MDS Revised international prognostic scoring system (IPSS-R) risk group of intermediate, high, or very high. Patients with MF, will include only those with Dynamic International Prognostic Scoring System-Plus (DIPSS=Plus) risk category of intermediate-1, intermediate-2, and high risk. Patients with sickle cell disease and transfusional iron overload Not appropriate for other iron chelation therapy, per physician Received 10 or more units of packed red blood cells in the preceding 24 months and remains red cell transfusion dependent ECOG ≤ 3 ALT ≤ 3 times the upper limit of the normal range Estimate glomerular filtration rate calculated using Cockroft Gault of ≥ 60 mL/min/1.73m2 Serum ferritin ≥1000 ng/ml Willing to comply with all study procedures and be available for the duration of the study Able to take oral medication and be willing to adhere to study medication for 28 days Female patient must be post-menopausal (no menses for > 12 consecutive months) or surgically sterile (i.e., bilateral oophorectomy, hysterectomy, or tubal sterilization; must agree to completely abstain for heterosexual intercourse; or, if sexually active, must agree to use 1 of the following methods for birth control from the date she signs the consent form until 30 days after final dose of the study drug. Progesterone implant Intrauterine device Combination of 2 highly effective birth control methods (e.g., diaphragm/or cervical cap with spermicide plus a condom, hormonal contraception plus a barrier method, partner with vasectomy conducted >60 days before screening visit plus a hormone or barrier method Male patients must agree to use 1 of the following methods for birth control from the date he signs the consent form until 30 days after final dose of the study drug: be surgically sterile by vasectomy conducted > 60 days before screening visit plus use a barrier method, or, must agree to completely abstain from heterosexual intercourse, or must agree to use a combination of 2 highly effective birth control methods (e.g., diaphragm/or cervical cap with spermicide plus a condom, hormonal contraception plus a barrier method), or have a post-menopausal partner plus barrier method. Exclusion Criteria: History of kidney disease including the renal Fanconi syndrome Proteinuria on urine dipstick greater than trace positive Pregnant, intending to become pregnant during the study, or breastfeeding Receiving another investigational drug within 30 days or 3 half-lives of the discontinued investigational agent, whichever is greater, of signing consent History of significant hepatic impairment, defined by Child-Pugh class C Active hepatitis B or C disease, evidenced by positive viral PCR Symptomatic heart failure Receiving active cytotoxic chemotherapy or radiation therapy for a second malignancy (hormonal therapy or topical therapy for squamous cell/basal cell cutaneous tumors are allowed). Treatment of the underlying hematologic malignancy with azacytidine, decitabine, venetoclax, lenalidomide, or ruxolitinib is permitted. Treatment with the supportive care agents luspatercept or erythropoietin agonists is permitted. Concurrent treatment with Exjade/Jadenu (deferasirox), Desferal (deferoxamine), or Ferriprox (deferiprone) are not permitted. Patients are allowed to stop these chelators and participate in this trial 14 days after discontinuation of the other chelator.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Epp Goodwin
Phone
210-450-5798
Email
goodwine@uthscsa.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Supreet Kaur, MD
Organizational Affiliation
UT Health San Antonio
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mays Cancer Center, UT Health San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Patricia Manea, RN
Phone
210-450-1821
Email
maneap@uthscsa.edu
First Name & Middle Initial & Last Name & Degree
Supreet Kaur, MD

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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Safety of SP-420 in the Treatment of Transfusional Iron Overload

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