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Study · Non-randomized trial · n = 16 · 2023

10 results

Rusfertide for the treatment of iron overload in HFE-related haemochromatosis: an open-label, multicentre, proof-of-concept phase 2 trial.

Kowdley KV, Modi NB, Peltekian K, Vierling JM, Ferris C, Valone FH, Gupta S

The lancet. Gastroenterology & hepatology17 Oct 2023Cited by 26 (Europe PMC)

PMID37863080 (opens in a new tab)DOI10.1016/s2468-1253(23)00250-9 (opens in a new tab)PepBankPUB-37863080
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study10

10 results from studies that gave the peptide. 10 results from people.

  1. 01HumanNon-randomized trialEfficacy outcome

    In HFE-related haemochromatosis, mean phlebotomies fell from 2·31 before treatment to 0·06 during rusfertide treatment.

    In plain wordsIn HFE-related haemochromatosis, mean blood removal procedures fell from 2·31 before treatment to 0·06 during rusfertide treatment.

    Peptide
    Rusfertide
    Outcome
    DecreasePhlebotomy frequency
    Study

    n = 16

    Comparator
    24 weeks before study
    Outcome group
    Blood biochemical markers · Whole body
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    Mean number of phlebotomies
    0.06 phlebotomies
    24-week rusfertide treatment
    Mean number of phlebotomies
    2.31 phlebotomies
    24 weeks pre-study
    P value
    < 0.0001
    treatment versus pre-study phlebotomies

    Source sentences

    Summary
    The mean number of phlebotomies was significantly reduced during the 24-week rusfertide treatment (0·06 phlebotomies [95% CI -0·07 to 0·20]) compared with 24 weeks pre-study (2·31 phlebotomies [95% CI 1·77 to 2·85]; p<0·0001).
    3 context sentences
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  2. 02HumanNon-randomized trialEfficacy outcome

    In HFE-related haemochromatosis, 15 (94%) of 16 patients were phlebotomy-free during rusfertide treatment.

    In plain wordsIn HFE-related haemochromatosis, 94% of patients needed no blood removal during rusfertide treatment.

    Peptide
    Rusfertide
    Outcome
    Not applicablePhlebotomy independence
    Study

    n = 16

    Outcome group
    Blood biochemical markers · Whole body
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    Phlebotomy-free patients
    94%
    24-week treatment period, 15 of 16 patients
    Phlebotomy-free patients
    15 patients
    24-week treatment period, of 16

    Source sentences

    Summary
    15 (94%) of 16 patients were phlebotomy-free during the treatment period.
    3 context sentences
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  3. 03HumanNon-randomized trialSafety outcome

    In HFE-related haemochromatosis, 12 (75%) patients had at least one treatment-emergent adverse event with rusfertide.

    In plain wordsIn HFE-related haemochromatosis, 75% had at least one adverse event during rusfertide treatment.

    Peptide
    Rusfertide
    Outcome
    Not applicableTreatment-emergent adverse events
    Study

    n = 16

    Outcome group
    Adverse events · Whole body
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    Patients with at least one TEAE
    12 patients
    all treated patients
    Patients with at least one TEAE
    75%
    all treated patients

    Source sentences

    Summary
    12 (75%) patients had at least one TEAE, the most common of which was injection site pain (five [31%] patients).
    3 context sentences
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  4. 04HumanNon-randomized trialSafety outcome

    In HFE-related haemochromatosis, injection site pain occurred in five (31%) patients during rusfertide treatment.

    In plain wordsIn HFE-related haemochromatosis, 31% had injection site pain during rusfertide treatment.

    Peptide
    Rusfertide
    Outcome
    Not applicableInjection site pain
    Study

    n = 16

    Outcome group
    Adverse events · Skin and wound
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    Patients with injection site pain
    5 patients
    all treated patients
    Patients with injection site pain
    31%
    all treated patients

    Source sentences

    Summary
    12 (75%) patients had at least one TEAE, the most common of which was injection site pain (five [31%] patients).
    3 context sentences
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  5. 05HumanNon-randomized trialSafety outcome

    In HFE-related haemochromatosis, adverse events were mild or moderate except for severe, pre-existing pancreatic adenocarcinoma considered unrelated to rusfertide.

    In plain wordsDuring rusfertide treatment for haemochromatosis, adverse events were mild or moderate except for severe, pre-existing pancreatic cancer considered unrelated to treatment.

    Peptide
    Rusfertide
    Outcome
    Not applicableAdverse event severity
    Study

    n = 16

    Outcome group
    Adverse events · Whole body
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Source sentences

    Summary
    All TEAEs were mild or moderate in severity, except for a serious adverse event of pancreatic adenocarcinoma, which was considered severe and unrelated to treatment and was pre-existing and diagnosed 21 days after starting rusfertide treatment.
    3 context sentences
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  6. 06HumanNon-randomized trialEfficacy outcome

    In HFE-related haemochromatosis, liver iron concentration changed from 1·4 to 1·1 mg iron per g dry liver weight with rusfertide, without significance.

    In plain wordsIn HFE-related haemochromatosis, liver iron changed from 1·4 to 1·1 mg per g with rusfertide, without a statistically significant change.

    Peptide
    Rusfertide
    Outcome
    No significant changeLiver iron concentration
    Study

    n = 14

    Comparator
    Screening
    Outcome group
    Liver fat and liver function · Liver
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    Mean liver iron concentration
    1.4 mg iron per g dry liver weight
    screening, n=14
    Mean liver iron concentration
    1.1 mg iron per g dry liver weight
    end of treatment, n=14
    P value
    0.068
    LIC change from screening to end of treatment

    Source sentences

    Summary
    Mean LIC in the 14 patients in the intention-to-treat population was 1·4 mg iron per g dry liver weight (95% CI 1·0 to 1·8) at screening and 1·1 mg iron per g dry liver weight (95% CI 0·9 to 1·3) at the end of treatment (p=0·068).
    3 context sentences
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  7. 07HumanNon-randomized trialEfficacy outcome

    In HFE-related haemochromatosis, mean TSAT was 45·3% at screening, 21·8% after the first rusfertide dose, and 40·4% at treatment end.

    In plain wordsIn HFE-related haemochromatosis, iron saturation fell after the first rusfertide dose, then increased by treatment end.

    Peptide
    Rusfertide
    Outcome
    MixedTransferrin iron saturation
    Study

    n: not reported in source

    Outcome group
    Blood biochemical markers · Whole body
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    TSAT
    45.3%
    screening
    TSAT
    36.7%
    after pretreatment phlebotomy
    TSAT
    21.8%
    24 h after first dose
    TSAT
    40.4%
    end of treatment
    TSAT
    32.6%
    over treatment duration

    Source sentences

    Summary
    Mean TSAT was 45·3% (95% CI 33·2 to 57·3) at screening, 36·7% (24·2 to 49·2) after the pretreatment phlebotomy, 21·8% (15·8 to 27·9) 24 h after the first dose of rusfertide, 40·4% (27·1 to 53·8) at the end of treatment, and 32·6% (25·0 to 40·1) over the treatment duration.
    3 context sentences
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  8. 08HumanNon-randomized trialEfficacy outcome

    In HFE-related haemochromatosis, mean serum iron was 24·6 μmol/L at screening and 22·5 μmol/L at rusfertide treatment end.

    In plain wordsIn HFE-related haemochromatosis, mean blood iron was lower at rusfertide treatment end than at screening.

    Peptide
    Rusfertide
    Outcome
    MixedSerum iron
    Study

    n: not reported in source

    Outcome group
    Blood biochemical markers · Whole body
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    Serum iron
    24.6 μmol/L
    screening
    Serum iron
    20.1 μmol/L
    after pretreatment phlebotomy
    Serum iron
    11.9 μmol/L
    24 h after first dose
    Serum iron
    22.5 μmol/L
    end of treatment
    Serum iron
    19 μmol/L
    over treatment duration

    Source sentences

    Summary
    Mean serum iron was 24·6 μmol/L (95% CI 18·6 to 30·6), 20·1 μmol/L (14·8 to 25·3), 11·9 μmol/L (9·2 to 14·7), 22·5 μmol/L (15·9 to 29·1), and 19·0 μmol/L (15·3 to 22·6) at these same timepoints, respectively.
    4 context sentences
    Summary · context
    at screening, 36·7% (24·2 to 49·2) after the pretreatment phlebotomy, 21·8% (15·8 to 27·9) 24 h after the first dose of rusfertide, 40·4% (27·1 to 53·8) at the end of treatment, and 32·6% (25·0 to 40·1) over the treatment duration.
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  9. 09HumanNon-randomized trialEfficacy outcome

    In HFE-related haemochromatosis, mean serum ferritin was 83·3 μg/L at screening and 150·0 μg/L at rusfertide treatment end.

    In plain wordsIn HFE-related haemochromatosis, mean ferritin was higher at rusfertide treatment end than at screening.

    Peptide
    Rusfertide
    Outcome
    MixedSerum ferritin
    Study

    n: not reported in source

    Outcome group
    Blood biochemical markers · Whole body
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Measurements

    Serum ferritin
    83.3 μg/L
    screening
    Serum ferritin
    65.5 μg/L
    after pretreatment phlebotomy
    Serum ferritin
    62.8 μg/L
    24 h after first dose
    Serum ferritin
    150 μg/L
    end of treatment
    Serum ferritin
    94.3 μg/L
    over treatment duration

    Source sentences

    Summary
    Mean serum ferritin was 83·3 μg/L (52·2 to 114.4), 65·5 μg/L (32·1 to 98·9), 62·8 μg/L (33·8 to 91·9), 150·0 μg/L (86·6 to 213.3), and 94·3 μg/L (54·9 to 133.6) at these same timepoints, respectively.
    4 context sentences
    Summary · context
    at screening, 36·7% (24·2 to 49·2) after the pretreatment phlebotomy, 21·8% (15·8 to 27·9) 24 h after the first dose of rusfertide, 40·4% (27·1 to 53·8) at the end of treatment, and 32·6% (25·0 to 40·1) over the treatment duration.
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.
  10. 10HumanNon-randomized trialEfficacy outcome

    In HFE-related haemochromatosis, serum transferrin concentration showed only minor changes during rusfertide treatment.

    In plain wordsIn HFE-related haemochromatosis, the iron transport protein transferrin changed only slightly during rusfertide treatment.

    Peptide
    Rusfertide
    Outcome
    Direction not reportedSerum transferrin
    Study

    n: not reported in source

    Outcome group
    Blood biochemical markers · Whole body
    Studied dose
    Initially 10 mg once weekly, with dose and schedule adjustment · subcutaneous · 24 weeksAs given in this study (human).

    Source sentences

    Summary
    There were only minor changes in serum transferrin concentration.
    3 context sentences
    Summary · context
    This open-label, multicentre, proof-of-concept phase 2 trial was done across nine academic and community centres in the USA and Canada.
    Summary · context
    Patients initiated 24 weeks of subcutaneous rusfertide treatment within 7 days of a scheduled phlebotomy at 10 mg once weekly.
    Summary · context
    Rusfertide doses and dosing schedules could be adjusted to maintain serum transferrin iron saturation (TSAT) at less than 40%.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 completed · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 2Results postedNCT04202965

PTG-300 in Subjects With Hereditary Hemochromatosis

Protagonist Therapeutics, Inc.n = 1619 Mar 2020 to 15 Feb 2022

Hereditary Hemochromatosis

Trial pageClinicalTrials.govUpdated 20 Aug 2025

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    27 Oct 2026, 18:20 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:32 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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