Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 3CAPSTONENCT03588026
Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576
A Phase 3 study testing rVA576 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by AKARI Therapeutics at 3 sites in 3 countries.
Trial summary
rVA576 for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- What is being tested?
- rVA576
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 7 Jun 2018to 3 Sep 2020
- Phase
- Phase 3
- 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
- Willing to give informed consent to treatment with rVA576
- Diagnosed with paroxysmal nocturnal haemoglobinuria (PNH)
- Have not received any complement inhibitor within the 4 months prior to screening
- ≥ 18 years of age at the time of screening
- Weight ≥50kg
- Complete transfusion medical history for 12 months
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- Transfusion dependent
- LDH ≥1.5 x the ULN
- Willing to receive appropriate prophylaxis against Neisseria meningitidis infection, by both immunisation and continuous or intermittent antibiotics
- Willing to avoid prohibited medications such as other complement inhibitors and chemotherapeutic agents
- Patients must agree to avoid pregnancy and fathering children from the time of signing the Informed Consent Form until 90 days after the last dose of rVA576.
- Patients who are on erythropoietin and/or immunosuppressant treatment should be on stable doses for at least 6 months.
- Patients who are taking systemic corticosteroids should be on a stable dose for at least 4 weeks.
- Patients on anticoagulant therapy should be well-controlled prior to entry.
- Patients taking iron and/or folic acid supplements should be on a stable dose for at least 4 weeks
Who cannot join
- Patients whose mean haemoglobin level over the previous 12 months prior to screening was greater than 105 g/L (10.5g/dL)
- Severe bone marrow failure
- Patients with a platelet count of ≤ 70 x 109/L
- Patients with known or suspected acquired somatic mutations affecting the bone marrow (e.g. acute myeloid leukaemia) which may be associated with PNH
- Chemotherapy within 3 months of screening visit
- History of recurrent bacterial infections or suspicion of active bacterial infections requiring antibiotic therapy
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- Planned or actual pregnancy or breast feeding (females)
- Known allergy to ticks or severe reaction to arthropod venom (e.g. bee or wasp venom)
- Unresolved N. meningitidis infection.
- Patients who are not willing to receive adequate immunisation against N. meningitidis unless, in the opinion of the investigator, the risks of delaying therapy outweigh the risks of developing a meningococcal infection
- Impaired hepatic function unless, in the opinion of the investigator, the risks of delaying therapy outweigh the risks of treatment in the presence of impaired hepatic function
- Patients with a glomerular filtration rate (GFR) of <30mL/min/1.73m2 unless, in the opinion of the investigator, the risks of delaying therapy outweigh the risks of treatment in the presence of impaired renal function
- Participation in other clinical trials within 4 weeks of signing the consent form
- History of active systemic autoimmune diseases.
- Any other systemic disorders that could interfere with the evaluation of the study treatment
- Failure to comply with protocol requirements
- Known Hepatitis B or Hepatitis C
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
Kazakhstan1 site
Almaty City Hospital No.7
Almaty, Microdistrict Kalkaman, 050006
Lithuania1 site
Vilnius University Hospital Santaros Klinikos , Santariškių St. 2, LT-08661,
Vilnius, LT-08661
Sri Lanka1 site
University of Kelaniya, Faculty of Medicine, Thalagolla Road
Colombo, Ragama/11010
About this study
Official title: Investigational Product ; Coversin. Phase III Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH)
rVA576 for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
Read the detailed description
rVA576, a small protein complement C5 inhibitor which prevents the cleavage of C5 by C5 convertase into C5a and C5b, will be used in an open label, non-comparative clinical trial in patients with PNH.
Patients will be treated with rVA576 by daily subcutaneous injection in order to determine the safety and efficacy of the drug in these circumstances.
If satisfactory control of the PNH is achieved, and at the discretion of the Principal Investigator (PI), patients will have the option of remaining on rVA576 and being entered into the long term follow-up study.
- Being tested
- rVA576, Standard of care (SOC)
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rVA576Drug
6 months of treatment, rVA576 plus SOC. Followed by a further 3 months of rVA576 plus SOC. In total, 9 months on rVA576 plus SOC.
Standard of care (SOC)Other
6 months on SOC followed by 3 months of treatment with rVA576 plus SOC. In total, 3 months on rVA576 plus SOC.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 - 9 months of treatment (rVA576 plus SOC) | Experimental | 6 months (SOC plus rVA576), Followed by a further 3 months of (SOC plus rVA576). Receives: Drug: rVA576; Other: Standard of care (SOC) |
| Arm 2 - 6 months on SOC | Experimental | 6 months on SOC only. Followed by 3 months (SOC plus rVA576). Receives: Drug: rVA576; Other: Standard of care (SOC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
HB (Haemoglobin) stabilisation rate and the avoidance of packed red blood cells (PRBC) transfusions
Haemoglobin stabilisation rate defined as haemoglobin greater than the set point for each patient during the pre-study randomisation period and the avoidance of PRBC transfusions during the treatment period.
Measured: 9 months
Secondary outcomes (5)
Number of units of packed red blood cells (PRBC) transfused
Number of units of packed red blood cells (PRBC) transfused from Baseline Day 1 to Day 180
Measured: Day 1 to Day 180
Percentage of patients who achieve transfusion avoidance
Percentage of patients who achieve transfusion avoidance
Measured: Day 1 to Day 180
Change in (QOl) Quality of Life score
Change in Quality of Life score
Measured: Day 1 to Day 180
AUC (LDH)
AUC (Area under the curve) (LDH) Lactate Dehydrogenase
Measured: Day 1 to Day 180
CH50
CH50 (Classical haemolytic 50% lysis)
Measured: Day 1 to Day 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2018
- Study start7 Jun 2018
- Primary completion3 Sep 2020
- Study completion3 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies of the same condition, open and under way.
- 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
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- AKARI Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Andrius Degulys, MBBS, Principal investigator, Vilnius University Hospital Santaros Klinikos
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03588026
- Sponsor's protocol ID
- AK580
- Acronym
- CAPSTONE
- Status last verified
- Nov 2020
- First submitted
- 1 Jun 2018
- Last updated
- 9 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigational Product ; Coversin. Phase III Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT03588026
- Acronym / sponsor ID
- CAPSTONE / AK580
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- AKARI TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 7 Jun 2018 (actual)
- Primary completion
- 3 Sep 2020 (actual)
- Study completion
- 3 Sep 2020 (actual)
- First posted
- 17 Jul 2018 (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.
rVA576 for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
Detailed description
rVA576, a small protein complement C5 inhibitor which prevents the cleavage of C5 by C5 convertase into C5a and C5b, will be used in an open label, non-comparative clinical trial in patients with PNH.
Patients will be treated with rVA576 by daily subcutaneous injection in order to determine the safety and efficacy of the drug in these circumstances.
If satisfactory control of the PNH is achieved, and at the discretion of the Principal Investigator (PI), patients will have the option of remaining on rVA576 and being entered into the long term follow-up study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1 - 9 months of treatment (rVA576 plus SOC) | Experimental | Drug: rVA576, Other: Standard of care (SOC)6 months (SOC plus rVA576), Followed by a further 3 months of (SOC plus rVA576). |
| Arm 2 - 6 months on SOC | Experimental | Drug: rVA576, Other: Standard of care (SOC)6 months on SOC only. Followed by 3 months (SOC plus rVA576). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Willing to give informed consent to treatment with rVA576
- Diagnosed with paroxysmal nocturnal haemoglobinuria (PNH)
- Have not received any complement inhibitor within the 4 months prior to screening
- ≥ 18 years of age at the time of screening
- Weight ≥50kg
Key exclusion criteria (17 listed)
- Patients whose mean haemoglobin level over the previous 12 months prior to screening was greater than 105 g/L (10.5g/dL)
- Severe bone marrow failure
- Patients with a platelet count of ≤ 70 x 109/L
- Patients with known or suspected acquired somatic mutations affecting the bone marrow (e.g. acute myeloid leukaemia) which may be associated with PNH
- Chemotherapy within 3 months of screening visit
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 |
|---|---|---|
| rVA5766 months of treatment, rVA576 plus SOC. Followed by a further 3 months of rVA576 plus SOC. In total, 9 months on rVA576 plus SOC. | Drug | Arm 1 - 9 months of treatment (rVA576 plus SOC), Arm 2 - 6 months on SOC |
| Standard of care (SOC)6 months on SOC followed by 3 months of treatment with rVA576 plus SOC. In total, 3 months on rVA576 plus SOC. | Other | Arm 1 - 9 months of treatment (rVA576 plus SOC), Arm 2 - 6 months on SOC |
Primary outcome
HB (Haemoglobin) stabilisation rate and the avoidance of packed red blood cells (PRBC) transfusions
Time frame: 9 months
Haemoglobin stabilisation rate defined as haemoglobin greater than the set point for each patient during the pre-study randomisation period and the avoidance of PRBC transfusions during the treatment period.
5 secondary outcomes
Number of units of packed red blood cells (PRBC) transfused
Time frame: Day 1 to Day 180
Percentage of patients who achieve transfusion avoidance
Time frame: Day 1 to Day 180
Change in (QOl) Quality of Life score
Time frame: Day 1 to Day 180
AUC (LDH)
Time frame: Day 1 to Day 180
CH50
Time frame: Day 1 to Day 180
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2025.
- Individual participant dataSharing planned: No
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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.
Andrius Degulys, MBBS principal investigator
Vilnius University Hospital Santaros Klinikos
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing rVA576 in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by AKARI Therapeutics at 3 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 15 inclusion and 17 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 9 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Kazakhstan, Lithuania, Sri Lanka. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
HB (Haemoglobin) stabilisation rate and the avoidance of packed red blood cells (PRBC) transfusions (time frame: 9 months). 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 9 Apr 2025.