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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04741542
Recruitment Status : Recruiting
First Posted : February 5, 2021
Last Update Posted : June 25, 2021
Sponsor:
Collaborator:
Abfero Pharmaceuticals, Inc
Information provided by (Responsible Party):
The University of Texas Health Science Center at San Antonio

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.

Condition or disease Intervention/treatment Phase
Iron Overload Drug: SP-420 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 28 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 1, Open-Label, Dose Escalation Study to Assess the Safety of Sp-420 in the Treatment of Transfusional Iron Overload in Patients With MDS(Myelodysplastic Syndrome), and MF (Myelofibrosis)
Actual Study Start Date : March 9, 2021
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : March 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron

Arm Intervention/treatment
Experimental: Group A
Study subjects will receive a 14mg/kg starting dose of SP-420 three times a week
Drug: SP-420
This study aims to establish the safety of SP-420 administered orally three times per week (TIW).
Other Name: (4S)-4,5-dihydro-2-[2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl]-4-methyl-4-thiazolecarboxylic acid

Experimental: Group B
Study subjects will receive a 28mg/kg starting dose of SP-420 three times a week
Drug: SP-420
This study aims to establish the safety of SP-420 administered orally three times per week (TIW).
Other Name: (4S)-4,5-dihydro-2-[2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl]-4-methyl-4-thiazolecarboxylic acid

Experimental: Group C
Study subjects will receive a 42mg/kg starting dose of SP-420 three times a week
Drug: SP-420
This study aims to establish the safety of SP-420 administered orally three times per week (TIW).
Other Name: (4S)-4,5-dihydro-2-[2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl]-4-methyl-4-thiazolecarboxylic acid

Experimental: Group D
Study subjects will receive a 56mg/kg starting dose of SP-420 three times a week
Drug: SP-420
This study aims to establish the safety of SP-420 administered orally three times per week (TIW).
Other Name: (4S)-4,5-dihydro-2-[2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl]-4-methyl-4-thiazolecarboxylic acid




Primary Outcome Measures :
  1. Number of adverse events [ Time Frame: 28 Days ]
    Count of adverse events induced by SP-420

  2. Completion at original dose [ Time Frame: 28 Days ]
    Number of subjects that completed the study at the original starting dose of that group



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04741542


Contacts
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Contact: Epp Goodwin 210-450-5798 goodwine@uthscsa.edu

Locations
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United States, Texas
Mays Cancer Center, UT Health San Antonio Recruiting
San Antonio, Texas, United States, 78229
Contact: Patricia Manea, RN    210-450-1821    maneap@uthscsa.edu   
Principal Investigator: Elizabeth Bowhay-Carnes, MD         
Sponsors and Collaborators
The University of Texas Health Science Center at San Antonio
Abfero Pharmaceuticals, Inc
Investigators
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Principal Investigator: Elizabeth Bowhay-Carnes, MD UT Health San Antonio
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Responsible Party: The University of Texas Health Science Center at San Antonio
ClinicalTrials.gov Identifier: NCT04741542    
Other Study ID Numbers: CTMS 20-0138
HSC20210039H ( Other Identifier: UT Health Science Center San Antonio )
First Posted: February 5, 2021    Key Record Dates
Last Update Posted: June 25, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Iron Overload
Iron Metabolism Disorders
Metabolic Diseases