Iron OverloadPhase 1NCT04741542
Safety of SP-420 in the Treatment of Transfusional Iron Overload
A Phase 1 study testing SP-420 in people with Iron Overload, run by The University of Texas Health Science Center at San Antonio at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron Overload
- What is being tested?
- SP-420
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Mar 2021to 10 Jan 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Mays Cancer Center, UT Health San Antonio
San Antonio, Texas, 78229
About this study
Official title: Phase 1, Open-Label, Dose Escalation Study to Assess the Safety of SP-420 in the Treatment of Transfusional Iron Overload
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.
- Conditions
- Iron Overload
- Being tested
- SP-420
- Medical subject headings
- Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SP-420Drug
Also called (4S)-4,5-dihydro-2-[2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl]-4-methyl-4-thiazolecarboxylic acid
This study aims to establish the safety of SP-420 administered orally three times per week (TIW).
| Study group | Role | What they receive |
|---|---|---|
| Group A | Experimental | Study subjects will receive a 14mg/kg starting dose of SP-420 three times a week Receives: Drug: SP-420 |
| Group B | Experimental | Study subjects will receive a 28mg/kg starting dose of SP-420 three times a week Receives: Drug: SP-420 |
| Group C | Experimental | Study subjects will receive a 42mg/kg starting dose of SP-420 three times a week Receives: Drug: SP-420 |
| Group D | Experimental | Study subjects will receive a 56mg/kg starting dose of SP-420 three times a week Receives: Drug: SP-420 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events
Count of adverse events induced by SP-420
Measured: 28 Days
Completion at original dose
Number of subjects that completed the study at the original starting dose of that group
Measured: 28 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2021
- Study start9 Mar 2021
- Primary completion10 Jan 2024
- Study completion10 Jan 2024
Why it stopped: Study is on hold by FDA in the US.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Iron OverloadStudies of the same condition, open and under way.
- More trials run by The University of Texas Health Science Center at San AntonioThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Abfero Pharmaceuticals, Inc
- Responsible party
- Sponsor
- Investigators
- Supreet Kaur, MD, Principal investigator, The University of Texas Health Science Center at San Antonio
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04741542
- Sponsor's protocol ID
- CTMS 20-0138
- Other IDs
- HSC20210039H
- Status last verified
- Dec 2024
- First submitted
- 19 Jan 2021
- Last updated
- 27 Dec 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1, Open-Label, Dose Escalation Study to Assess the Safety of SP-420 in the Treatment of Transfusional Iron Overload
- ClinicalTrials.gov ID
- NCT04741542
- Acronym / sponsor ID
- CTMS 20-0138
- Conditions
- Iron Overload
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study is on hold by FDA in the US.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- The University of Texas Health Science Center at San AntonioUniversity, hospital or other
- Collaborators
- Abfero Pharmaceuticals, Inc
- Study start
- 9 Mar 2021 (actual)
- Primary completion
- 10 Jan 2024 (actual)
- Study completion
- 10 Jan 2024 (actual)
- First posted
- 5 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Group A | Experimental | Drug: SP-420Study subjects will receive a 14mg/kg starting dose of SP-420 three times a week |
| Group B | Experimental | Drug: SP-420Study subjects will receive a 28mg/kg starting dose of SP-420 three times a week |
| Group C | Experimental | Drug: SP-420Study subjects will receive a 42mg/kg starting dose of SP-420 three times a week |
| Group D | Experimental | Drug: SP-420Study subjects will receive a 56mg/kg starting dose of SP-420 three times a week |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SP-420Also: (4S)-4,5-dihydro-2-[2-hydroxy-4-[2-(2-methoxyethoxy)ethoxy]phenyl]-4-methyl-4-thiazolecarboxylic acidThis study aims to establish the safety of SP-420 administered orally three times per week (TIW). | Drug | Group A, Group B, Group C, Group D |
Primary outcome
Number of adverse events
Time frame: 28 Days
Count of adverse events induced by SP-420
Completion at original dose
Time frame: 28 Days
Number of subjects that completed the study at the original starting dose of that group
Results and safety
The study was stopped early (reason given: Study is on hold by FDA in the US.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Dec 2024.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Supreet Kaur, MD principal investigator
The University of Texas Health Science Center at San Antonio
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SP-420 in people with Iron Overload, run by The University of Texas Health Science Center at San Antonio at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 27 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Study is on hold by FDA in the US.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse events (time frame: 28 Days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Dec 2024.