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Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 2REDEEM-1NCT05116774

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy

A Phase 2 study testing Eculizumab, Ravulizumab and BCX9930 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by BioCryst Pharmaceuticals at 7 sites in 5 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH participants with residual anemia despite treatment with a C5 inhibitor. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Eculizumab, Ravulizumab and BCX9930
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
7 sitesin 5 countries
Study dates
6 Dec 2021to 14 Sep 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Male or female, aged ≥ 18 years old
  • Body weight ≥ 40 kg
  • Documented diagnosis of PNH
  • Currently being treated with a stable C5 inhibitor regimen
  • Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willing to start vaccination series
  • At screening: PNH clone size of ≥ 10% and hemoglobin ≤ 10.5 g/dL

Who cannot join

  • Known history of or existing diagnosis of hereditary complement deficiency
  • History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation
  • Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition
  • History of malignancy within 5 years prior to the screening visit
  • Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening
  • Treatment with anti-thymocyte globulin within 180 days prior to screening
  • Initiation of treatment with an erythrocyte or platelet growth factor, or danazol within 28 days prior to screening
  • Receiving iron supplementation with an unstable dose in the 28 days prior to screening

Trial locations and contacts

7 sites in 5 countries.

7 sites in 5 countries

Spain2 sites

  • Investigative Site

    Barcelona

  • Investigative Site

    Valencia

Other trials in Spain

United Kingdom2 sites

  • Investigative Site

    Leeds

  • Investigative Site

    London

Other trials in United Kingdom

France1 site

  • Investigative Site

    Paris

Other trials in France

Hungary1 site

  • Investigative Site

    Budapest

Other trials in Hungary

Italy1 site

  • Investigative Site

    Rome

Other trials in Italy

About this study

Official title: A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects With Inadequate Response to C5 Inhibitor Therapy

The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH participants with residual anemia despite treatment with a C5 inhibitor.

Read the detailed description

This was a randomized, active comparator-controlled, open-label, parallel-group, 2-part study. Parts 1 and 2 were conducted in the same participants.

Part 1 of the study was designed to evaluate the efficacy, safety, and tolerability of oral BCX9930 monotherapy for 24 weeks versus continuing C5 inhibitor therapy in participants with PNH with inadequate response to their current C5 inhibitor therapy. Participants were randomized to either discontinue C5 inhibitor therapy and start BCX9930 monotherapy or to continue C5 inhibitor therapy for the 24-week randomized treatment period. The primary efficacy and safety analyses were based on Part 1.

Part 2 of the study was designed to evaluate the long-term safety, tolerability, and effectiveness of BCX9930 monotherapy when administered through Week 52. All participants in Part 2 received BCX9930. Participants who were randomized to BCX9930 monotherapy in Part 1 continued to receive BCX9930 in Part 2. Participants who were randomized to C5 inhibitor therapy in Part 1 discontinued that therapy at the Week 24 visit and received BCX9930 in Part 2.

Being tested
Eculizumab, Ravulizumab, BCX9930
Keywords
BCX9930, factor D inhibitor, proximal complement inhibitor, oral therapy, paroxysmal nocturnal hemoglobinuria, inadequate response to C5 inhibitor, C5 inhibitor, eculizumab, ravulizumab
Medical subject headings
Hemoglobinuria, Paroxysmal

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EculizumabDrug

    Also called Soliris

    Administered by intravenous infusion per current dose regimen

  • RavulizumabDrug

    Also called Ultomiris, ALXN1210, ravulizumab-cwvz

    Administered by intravenous infusion per current dose regimen

  • BCX9930Drug

    Administered orally twice daily

Study groups in NCT05116774
Study groupRoleWhat they receive
BCX9930/BCX9930ExperimentalParticipants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.
Receives: Drug: BCX9930
C5-Inhibitor (C5-INH)/BCX9930Active comparatorParticipants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.
Receives: Drug: Eculizumab; Drug: Ravulizumab; Drug: BCX9930

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part 1: Change From Baseline in Hemoglobin at Week 24

    Measured: Baseline, Week 24

Secondary outcomes (15)

  1. Part 1: Number of Participants Who Were Transfusion-free

    The number of participants who did not receive any transfusions (packed red blood cells [pRBCs] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free.

    Measured: From Week 4 to Week 24

  2. Part 1: Number of Units of pRBCs Transfused

    Measured: From Week 4 to Week 24

  3. Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

    The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 ("not at all") to 4 ("very much"), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.

    Measured: Baseline, Week 24

  4. Part 1: Percent Change From Baseline in Lactate Dehydrogenase

    Measured: Baseline, Week 24

  5. Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused

    The rate of pRBC units transfused from Week 4 to Week 24 was calculated and compared to the rate of pRBC units transfused prestudy during the 12 months prior to screening. The percent reduction in rate of pRBC units transfused was the percent difference in rate relative to the prestudy rate, calculated as: (current rate - prestudy rate)/prestudy rate * 100%. Total rate among all participants was evaluated here. Rate of pRBC units transfusion was defined as the percentage of participants who received pRBC transfusions.

    Measured: Prestudy (12 months prior to screening) and from Week 4 to Week 24

  6. Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL)

    Measured: Week 24

  7. Part 1: Number of Participants Who Achieved Hemoglobin Stabilization

    Hemoglobin stabilization was defined as the participants who avoided 2 g/dL or greater decrease in hemoglobin in the absence of transfusion from Week 4 to Week 24.

    Measured: From Week 4 to Week 24

  8. Part 1: Change From Baseline in Complement Component 3 (C3)-Opsonized Red Blood Cells (RBCs)

    Red blood cells opsonized by C3 were to be assessed by flow cytometry .

    Measured: Baseline, Week 24

  9. Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size

    The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBC cells within the total RBC population.

    Measured: Baseline, Week 24

  10. Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size

    The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBCs within the total RBC population. The PNH WBC clone size refers to the percentage of PNH-affected WBCs within the total WBC population. The ratio of total PNH RBC clone size to PNH WBC clone size = ratio of percent total PNH RBCs / percent PNH WBCs.

    Measured: Baseline, Week 24

  11. Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)

    Measured: Baseline, Week 24

  12. Part 1: Number of Participants With ARC in the Normal Range

    Number of participants with ARC in the normal range (0.03 - 0.12 10^6 cells/uL) were reported.

    Measured: Week 24

  13. Part 1: Change From Baseline in Haptoglobin

    Measured: Baseline, Week 24

  14. Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range

    Number of Participants With Haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported.

    Measured: Week 24

  15. Part 1: Change From Baseline in Total Bilirubin

    Measured: Baseline, Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Nov 2021
  2. Study start6 Dec 2021
  3. Primary completion14 Sep 2023
  4. Study completion14 Sep 2023

Why it stopped: Sponsor decision

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05116774
Sponsor's protocol ID
BCX9930-202
Other IDs
2020-004438-39
Acronym
REDEEM-1
Status last verified
Mar 2025
First submitted
2 Nov 2021
Last updated
7 Mar 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Eculizumab, Ravulizumab and BCX9930 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by BioCryst Pharmaceuticals at 7 sites in 5 countries.

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 6 inclusion and 8 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 7 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in Spain, United Kingdom, France, Hungary, Italy. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Part 1: Change From Baseline in Hemoglobin at Week 24 (time frame: Baseline, Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05116774.

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 7 Mar 2025.

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