Paroxysmal Nocturnal HemoglobinuriaPhase 3DAHLIANCT03818607
A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
A Phase 3 study testing ABP 959 and Eculizumab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Amgen at 24 sites in 14 countries.
Trial summary
This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- ABP 959 and Eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 24 sitesin 14 countries
- Study dates
- 22 Jan 2019to 12 Jul 2022
- Phase
- Phase 3
- 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
- Men and women ≥ 18 years of age.
- Historical diagnosis of PNH.
- Administration of eculizumab for ≥ 6 months and currently receiving 900 mg of eculizumab.
- Hemoglobin ≥ 9.0 g/dL for at least 6 weeks before randomization.
- Lactate dehydrogenase < 1.5 × the upper limit of normal at screening.
- Platelet count ≥ 50 × 10^9/L.
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- Absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L (500/μL).
- Participants must be vaccinated against Neisseria meningitidis.
- Participants must sign an IRB/IEC-approved ICF before participation in any procedures.
Who cannot join
- Known or suspected hereditary complement deficiency.
- Clinically significant cardiovascular disease (including myocardial infarction, unstable angina, symptomatic congestive heart failure [New York Heart Association ≥ Class III], serious uncontrolled cardiac arrhythmia), peripheral vascular disease, cerebrovascular accident, or transient ischemic attack in the previous 6 months.
- Evidence of acute thrombosis (liver Doppler ultrasound of hepatic and portal veins).
- Known to be positive for human immunodeficiency virus.
- Woman who is pregnant or breastfeeding.
- Participant is currently enrolled in or has not yet completed at least 30 days since ending other investigational device or drug study(s), or participant is receiving other investigational agent(s).
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- Participant has known sensitivity to any of the products to be administered during the study, including mammalian cell-derived drug products.
- History of meningococcal infection.
- Presence or suspicion of active bacterial infection, or recurrent bacterial infection.
- History of bone marrow transplantation.
- Red blood cell transfusion required within 12 weeks before randomization.
- Participant experienced ≥ 2 breakthrough events, (ie, signs and symptoms of intravascular hemolysis, that require dose and/or schedule adjustments of eculizumab) in the previous 12 months before screening.
Trial locations and contacts
24 sites in 14 countries.
24 sites in 14 countries
Italy5 sites
Azienda Ospedaliera S. Croce e Carle Cuneo
Cuneo, 12100
Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
Meldola, Forli-cesena, 47014
Azienda Ospedaliera San Gerardo di Monza
Monza, Monza Brianza, 20052
Azienda USL della Romagna
Ravenna, 48121
Fondazione Policlinico Universitario Agostino Gemelli
Roma, 00168
Czechia3 sites
Fakultní Nemocnice Brno
Brno, Jihormoravsky KRAJ, 625 00
Fakultní Nemocnice Olomouc
Olomouc, 772 00
Fakultní Nemocnice Ostrava
Ostrava-Poruba, 708 52
Finland2 sites
Keski-Suomen keskussairaala Jyväskylä
Jyväskylä, FI-40620
Päijät-Häme Central Hospital
Lahti, FI-15850
Spain2 sites
Hospital Universitario de Salamanca
Salamanca, 37007
Hospital Universitario La Fe
Valencia, 46026
Turkey (Türkiye)2 sites
Ege Universitesi Hastanesi - Sağlık Uygulama ve Araştırma Merkezi
Bornova, İzmir, 35100
Mersin Universitesi Tip Fakultesi
Mersin, 33110
United Kingdom2 sites
The Leeds Teaching Hospitals NHS Trust
Leeds, England, LS9 7TF
King's College Hospital NHS Foundation Trust
London, England, SE5 9RS
United States1 site
Children's Healthcare of Atlanta at Egleston
Atlanta, Georgia, 30322
Netherlands1 site
Radboud Universitair Medisch Centrum
Nijmegen, Gelderland, 6525 GA
Portugal1 site
Instituto Português de Oncologia do Porto Francisco Gentil
Porto, 4200-072
About this study
Official title: A Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- ABP 959, Eculizumab
- Keywords
- Marchiafava-Micheli Syndrome, Paroxysmal Cold 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.
ABP 959Drug
Also called Treatment T
intravenous infusion
EculizumabDrug
Also called Soliris, Treatment R
intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| T (ABP 959) / R (eculizumab) | Other | ABP 959 for 52 weeks in Period 1 followed by eculizumab for 26 weeks in Period 2 Receives: Drug: ABP 959; Drug: Eculizumab |
| R (eculizumab) / T (ABP 959) | Other | Eculizumab for 52 weeks in Period 1 followed by ABP 959 for 26 weeks in Period 2 Receives: Drug: ABP 959; Drug: Eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
LDH Level at Week 27 (Parallel Comparison)
The primary analysis for the parallel comparison was hemolysis as measured by LDH at Week 27 by initial treatment received (Period 1).
Measured: Week 27
Time-adjusted Area Under the Effect Curve (AUEC) of LDH (Crossover Comparison Per Assigned Treatment)
The primary analysis for the crossover comparison was hemolysis, as measured by the time-adjusted AUEC of LDH, according to treatment assigned during each of the 14-week assessments during Periods 1 and 2.
Measured: From Week 13 to Week 27, from Week 39 to Week 53, and from Week 65 to Week 79
Secondary outcomes (14)
Mean Total Complement (50% Total Hemolytic Complement Activity [CH50])
Total complement (%) was measured in serum using an assay method and compared the total hemolytic complement activity to the lower limit of the normal human reference (LLN) of 58 U/mL for all CH50 values. The percent of LLN of CH50 at each time point was calculated as mean CH50 results/LLN x 100%.
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Total Hemoglobin Levels
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Serum-free Hemoglobin Levels
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Haptoglobin Levels
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Bilirubin Levels
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Degree of Hemoglobinuria
The degree of hemoglobinuria was categorized as negative, trace, small, moderate, and large based on the analysis of urine samples collected from each participant at the specified time points.
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Percentage of Type III Erythrocytes
As a measure of hemolysis the mean percentage of Type III erythrocytes was measured at the specified timepoints.
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline, Week 27, Week 39, Week 53, Week 65 and Week 79
LDH Levels at Week 53 and Week 79
The analysis of the crossover comparison of hemolysis, as measured by LDH at Week 53 and Week 79.
Measured: Week 53 (first week of Period 2) and Week 79 (last week of Period 2)
Mean LDH Levels by Visit up to Week 79
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline, Week 3, Week 7, Week 13, Week 15, Week 19, Week 25, Week 27, Week 29, Week 33, Week 39, Week 41, Week 43, Week 45, Week 47, Week 49, Week 51, Week 53, Week 55, Week 59, Week 65, Week 67, Week 69, Week 71, Week 73, Week 75, Week 77, and Week 79
Mean Number of Packed RBC Units Transfused Per Month
Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Baseline to End of Study (up to Week 79)
Total and Unbound Pharmacokinetics (PK) Area Under the Curve (AUC) of ABP 959 and Eculizumab From Week 13 to Week 15 (Period 1)
The total and unbound PK concentration AUC values from Week 13 to Week 15 in Period 1 are presented by actual treatment received.
Measured: PK samples were collected predose and immediately postdose Week 13, 7 days post the Week 13 dose (Week 14), and predose at Week 15
Total and Unbound Trough Serum Concentrations of ABP 959 and Eculizumab
The total and unbound serum trough concentrations are presented by treatment sequence received for the prespecified time points. Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: PK samples were collected predose at the prespecified timepoints: baseline, Week 3, Week 7, Week 13, Week 15, Week 19, Week 27, Week 33, Week 39, Week 45, Week 51, Week 53, Week 55, Week 59, Week 65, Week 71, Week 77, and Week 79
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
TEAEs are defined as any adverse event (AE) that began or increased in severity or frequency at or after the time of first treatment up to end of study (up to Week 79). A treatment-emergent serious adverse event (SAE) was a TEAE that met at least 1 of the following criteria: was fatal, life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or was another medically important serious event.
The treatment-emergent events of interest (EOI) prespecified for this study included serious infections (meningococcus aspergillus, and other serious infections/sepsis), and infusion reactions.
Measured: Day 1 to End of Study (up to Week 79)
Number of Participants With Antidrug Antibodies (ADAs)
Any samples that tested positive for binding antibodies were also tested for neutralizing antibodies. Treatment boosted ADAs were defined as a positive immunoassay result at baseline and at least 1 postbaseline immunoassay result that was ≥ 4 times the magnitude of the baseline result. Baseline was defined as the last non-missing assessment taken prior to the first dose of IP.
Measured: Blood samples for ADA assessments were taken predose at baseline, Week 3, Week 7, Week 13, Week 19, Week 25, Week 27, Week 33, Week 39, Week 45, Week 51, Week 53, Week 55, Week 59, Week 65, Week 71, Week 77 and Week 79.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2019
- Study start22 Jan 2019
- Primary completion12 Jul 2022
- Study completion12 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by AmgenThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03818607
- Sponsor's protocol ID
- 20150168
- Acronym
- DAHLIA
- Status last verified
- Apr 2023
- First submitted
- 17 Jan 2019
- Last updated
- 23 May 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Publications
Kulasekararaj A, Lanza F, Arvanitakis A, Langemeijer S, Chonat S, Tombak A, Hanes V, Cao J, Miller MJ, Colbert A, Shander B, Mytych DT, Chow V, Henary H. Comparative clinical efficacy and safety of biosimilar ABP 959 and eculizumab reference product in patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol. 2024 Nov;99(11):2108-2117. doi: 10.1002/ajh.27456. Epub 2024 Aug 22.
PubMed 39171864
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT03818607
- Acronym / sponsor ID
- DAHLIA / 20150168
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- Marchiafava-Micheli Syndrome, Paroxysmal Cold Hemoglobinuria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 22 Jan 2019 (actual)
- Primary completion
- 12 Jul 2022 (actual)
- Study completion
- 12 Jul 2022 (actual)
- First posted
- 28 Jan 2019 (actual)
- Results
- Posted 23 May 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| T (ABP 959) / R (eculizumab) | Other | Drug: ABP 959, Drug: EculizumabABP 959 for 52 weeks in Period 1 followed by eculizumab for 26 weeks in Period 2 |
| R (eculizumab) / T (ABP 959) | Other | Drug: ABP 959, Drug: EculizumabEculizumab for 52 weeks in Period 1 followed by ABP 959 for 26 weeks in Period 2 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Men and women ≥ 18 years of age.
- Historical diagnosis of PNH.
- Administration of eculizumab for ≥ 6 months and currently receiving 900 mg of eculizumab.
- Hemoglobin ≥ 9.0 g/dL for at least 6 weeks before randomization.
- Lactate dehydrogenase < 1.5 × the upper limit of normal at screening.
Key exclusion criteria (12 listed)
- Known or suspected hereditary complement deficiency.
- Clinically significant cardiovascular disease (including myocardial infarction, unstable angina, symptomatic congestive heart failure [New York Heart Association ≥ Class III], serious uncontrolled cardiac arrhythmia), peripheral vascular disease, cerebrovascular accident, or transient ischemic attack in the previous 6 months.
- Evidence of acute thrombosis (liver Doppler ultrasound of hepatic and portal veins).
- Known to be positive for human immunodeficiency virus.
- Woman who is pregnant or breastfeeding.
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 |
|---|---|---|
| ABP 959Also: Treatment Tintravenous infusion | Drug | R (eculizumab) / T (ABP 959), T (ABP 959) / R (eculizumab) |
| EculizumabAlso: Soliris, Treatment Rintravenous infusion | Drug | R (eculizumab) / T (ABP 959), T (ABP 959) / R (eculizumab) |
Primary outcome
LDH Level at Week 27 (Parallel Comparison)
Time frame: Week 27
The primary analysis for the parallel comparison was hemolysis as measured by LDH at Week 27 by initial treatment received (Period 1).
Time-adjusted Area Under the Effect Curve (AUEC) of LDH (Crossover Comparison Per Assigned Treatment)
Time frame: From Week 13 to Week 27, from Week 39 to Week 53, and from Week 65 to Week 79
The primary analysis for the crossover comparison was hemolysis, as measured by the time-adjusted AUEC of LDH, according to treatment assigned during each of the 14-week assessments during Periods 1 and 2.
14 secondary outcomes
Mean Total Complement (50% Total Hemolytic Complement Activity [CH50])
Time frame: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Total Hemoglobin Levels
Time frame: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Serum-free Hemoglobin Levels
Time frame: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Haptoglobin Levels
Time frame: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Bilirubin Levels
Time frame: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Degree of Hemoglobinuria
Time frame: Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Percentage of Type III Erythrocytes
Time frame: Baseline, Week 27, Week 39, Week 53, Week 65 and Week 79
LDH Levels at Week 53 and Week 79
Time frame: Week 53 (first week of Period 2) and Week 79 (last week of Period 2)
Mean LDH Levels by Visit up to Week 79
Time frame: Baseline, Week 3, Week 7, Week 13, Week 15, Week 19, Week 25, Week 27, Week 29, Week 33, Week 39, Week 41, Week 43, Week 45, Week 47, Week 49, Week 51, Week 53, Week 55, Week 59, Week 65, Week 67, Week 69, Week 71, Week 73, Week 75, Week 77, and Week 79
Mean Number of Packed RBC Units Transfused Per Month
Time frame: Baseline to End of Study (up to Week 79)
Total and Unbound Pharmacokinetics (PK) Area Under the Curve (AUC) of ABP 959 and Eculizumab From Week 13 to Week 15 (Period 1)
Time frame: PK samples were collected predose and immediately postdose Week 13, 7 days post the Week 13 dose (Week 14), and predose at Week 15
Total and Unbound Trough Serum Concentrations of ABP 959 and Eculizumab
Time frame: PK samples were collected predose at the prespecified timepoints: baseline, Week 3, Week 7, Week 13, Week 15, Week 19, Week 27, Week 33, Week 39, Week 45, Week 51, Week 53, Week 55, Week 59, Week 65, Week 71, Week 77, and Week 79
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: Day 1 to End of Study (up to Week 79)
Number of Participants With Antidrug Antibodies (ADAs)
Time frame: Blood samples for ADA assessments were taken predose at baseline, Week 3, Week 7, Week 13, Week 19, Week 25, Week 27, Week 33, Week 39, Week 45, Week 51, Week 53, Week 55, Week 59, Week 65, Week 71, Week 77 and Week 79.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39171864Kulasekararaj A, Lanza F, Arvanitakis A, Langemeijer S, Chonat S, Tombak A, Hanes V, Cao J, Miller MJ, Colbert A, Shander B, Mytych DT, Chow V, Henary H. Comparative clinical efficacy and safety of biosimilar ABP 959 and eculizumab reference product in patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol. 2024 Nov;99(11):2108-2117. doi: 10.1002/ajh.27456. Epub 2024 Aug 22.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
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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.
MD study director
Amgen
24 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ABP 959 and Eculizumab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Amgen at 24 sites in 14 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 9 inclusion and 12 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: finished, as of the sponsor's last update on 23 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in Italy, Czechia, Finland, Spain, Turkey (Türkiye) and 9 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 23 May 2023). 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?
LDH Level at Week 27 (Parallel Comparison) (time frame: Week 27). 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?
On the ClinicalTrials.gov record for NCT03818607, which also lists 1 linked publication.
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 23 May 2023.