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Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 2REDEEM-2NCT05116787

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

A Phase 2 study testing BCX9930 monotherapy in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by BioCryst Pharmaceuticals at 5 sites in 3 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 adult participants with PNH not currently receiving complement inhibitor therapy. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
BCX9930 monotherapy
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
12 participants
Where
5 sitesin 3 countries
Study dates
26 Oct 2021to 18 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
  • No complement inhibitor therapy for ≥ 12 months prior to screening
  • Contraindication to or no access to approved (C3 or C5) complement inhibitor therapies
  • Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series
  • At screening: PNH clone of ≥ 10%, hemoglobin ≤ 10.5 g/dL and lactate dehydrogenase ≥ 2 × upper limit of normal

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

5 sites in 3 countries.

5 sites in 3 countries

South Africa3 sites

  • Investigative Site

    Bloemfontein

  • Investigative Site

    Cape Town

  • Investigative Site

    Pretoria

Other trials in South Africa

Malaysia1 site

  • Investigative Site

    Ampang

Other trials in Malaysia

South Korea1 site

  • InvestigativeSite

    Daejeon

Other trials in South Korea

About this study

Official title: A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult participants with PNH not currently receiving complement inhibitor therapy.

Read the detailed description

This was a randomized, placebo-controlled, double-blind, parallel-group, 2-part study. Parts 1 and 2 was to be conducted in the same participants.

Part 1 of the study was designed to evaluate the efficacy, safety, and tolerability of treatment with oral BCX9930 monotherapy for 12 weeks versus placebo in participants with PNH who were not currently receiving treatment with complement inhibitor therapy. Participants were randomized to receive BCX9930 or placebo under double blind conditions for the 12-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 open-label 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 placebo in Part 1 discontinued that therapy at the Week 12 visit and received BCX9930 in Part 2.

Being tested
Placebo, BCX9930 monotherapy
Keywords
BCX9930, factor D inhibitor, proximal complement inhibitor, oral therapy, paroxysmal nocturnal hemoglobinuria
Medical subject headings
Hemoglobinuria, Paroxysmal

What participants receive

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

  • PlaceboDrug

    Administered orally twice daily

  • BCX9930 monotherapyDrug

    Administered orally twice daily

Study groups in NCT05116787
Study groupRoleWhat they receive
BCX9930/BCX9930ExperimentalParticipants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. Initially participants were to receive BCX9930 500 mg twice daily (BID) orally. Per protocol amendment, participants who previously received 500 mg 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 in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days.
Receives: Drug: BCX9930 monotherapy
Placebo/BCX9930Placebo comparatorParticipants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days.
Receives: Drug: Placebo; Drug: BCX9930 monotherapy

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 12

    Measured: Baseline, Week 12

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 12, 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 12

  2. Part 1: Number of Units of pRBCs Transfused

    Measured: From Week 4 to Week 12

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

    Measured: Baseline, Week 12

  4. 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 12

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

    The rate of pRBC units transfused from Week 4 to Week 12 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 12

  6. Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL

    Measured: At Week 12

  7. Part 1: Number of Participants Achieving 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 12.

    Measured: From Week 4 to Week 12

  8. Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (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 12

  9. 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 (i.e, 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 12

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

    Measured: Baseline, Week 12

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

    Number of participants with ARC in the normal range (50 - 100 x 10^9 cells/L) were reported.

    Measured: Week 12

  12. Part 1: Change From Baseline in Haptoglobin

    Measured: Baseline, Week 12

  13. 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 12

  14. Part 1: Change From Baseline in Total Bilirubin

    Measured: Baseline, Week 12

  15. Part 1: Change From Baseline in Aspartate Aminotransferase (AST)

    Measured: Baseline, Week 12

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 start26 Oct 2021
  3. Primary completion18 Sep 2023
  4. Study completion18 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
Double blind
Who is masked
Participant, Investigator
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05116787
Sponsor's protocol ID
BCX9930-203
Other IDs
2020-004403-14
Acronym
REDEEM-2
Status last verified
Dec 2024
First submitted
2 Nov 2021
Last updated
29 Oct 2026
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 BCX9930 monotherapy in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by BioCryst Pharmaceuticals at 5 sites in 3 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 7 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 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in South Africa, Malaysia, South Korea. 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. Some participants in this study may receive a placebo. 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 3 Jan 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 12 (time frame: Baseline, Week 12). 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 NCT05116787.

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 29 Oct 2026.

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