Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576

NCT03588026 | Completed Phase 3 DMC

• 1 other identifier: AK580
• Study Type: interventional
• Target: 9
• Locations: 3 countries
• Sites: 3

Brief Summary

rVA576 for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Outcome Measures

Primary Outcomes (1)

• 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.

  9 months

Secondary Outcomes (5)

• Number of units of packed red blood cells (PRBC) transfused

  Day 1 to Day 180

• Percentage of patients who achieve transfusion avoidance

  Day 1 to Day 180

• Change in (QOl) Quality of Life score

  Day 1 to Day 180

• AUC (LDH)

  Day 1 to Day 180

• CH50

  Day 1 to Day 180

Study Arms (2)

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).

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).

Drug: rVA576; Other: Standard of care (SOC)

Interventions

rVA576 DRUG

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.

Arm 1 - 9 months of treatment (rVA576 plus SOC); Arm 2 - 6 months on 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.

Arm 1 - 9 months of treatment (rVA576 plus SOC); Arm 2 - 6 months on SOC

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• AKARI Therapeutics: lead

Study Sites (3)

Almaty City Hospital No.7

Almaty, Microdistrict Kalkaman, 050006, Kazakhstan

Location

Vilnius University Hospital Santaros Klinikos , Santariškių St. 2, LT-08661,

Vilnius, LT-08661, Lithuania

Location

University of Kelaniya, Faculty of Medicine, Thalagolla Road

Colombo, Ragama/11010, Sri Lanka

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Study Officials

• Andrius Degulys, MBBS

  Vilnius University Hospital Santaros Klinikos

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 1, 2018
• First Posted: July 17, 2018
• Study Start: June 7, 2018
• Primary Completion: September 3, 2020
• Study Completion: September 3, 2020
• Last Updated: April 9, 2025

Record last verified: 2020-11

Data Sharing

• IPD Sharing: Will not share

Locations

SR (1); LI (1); KA (1)