CLINICAL TRIAL

Ravulizumab for Paroxysmal Nocturnal Haemoglobinuria (PNH)

Recruiting · 18+ · All Sexes · Whittier, CA

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

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About the trial for Paroxysmal Nocturnal Haemoglobinuria (PNH)

Eligible Conditions
Paroxysmal Nocturnal Haemoglobinuria (PNH) · Hemoglobinuria · Hemoglobinuria, Paroxysmal

Treatment Groups

This trial involves 2 different treatments. Ravulizumab is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

Experimental Group 1
Ravulizumab
DRUG
+
Eculizumab
DRUG
Experimental Group 2
Ravulizumab
DRUG
+
Eculizumab
DRUG
+
Pozelimab
DRUG
+
Cemdisiran
DRUG

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ravulizumab
FDA approved
Eculizumab
FDA approved
Pozelimab
Not yet FDA approved
Cemdisiran
Not yet FDA approved

Eligibility

This trial is for patients born any sex aged 18 and older. There are 2 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing
Treated with eculizumab or ravulizumab prior to screening visit as described in the protocol Note: Biosimilars are not permitted, unless approved by the Sponsor Key
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Week 8 (day 57) through week 36
Screening: ~3 weeks
Treatment: Varies
Reporting: Week 8 (day 57) through week 36
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Week 8 (day 57) through week 36.
View detailed reporting requirements
Trial Expert
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- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Ravulizumab will improve 1 primary outcome and 23 secondary outcomes in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH). Measurement will happen over the course of Through week 32.

Concentrations of total cemdisiran in plasma
THROUGH WEEK 32
Treatment period
THROUGH WEEK 32
Change in global health status (GHS)/QoL scale score on the EORTC-QLQ-C30
FROM BASELINE TO WEEK 36
EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
FROM BASELINE TO WEEK 36
Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale
FROM BASELINE TO WEEK 36
The FACIT-Fatigue is a 13 item, self-administered clinical outcome assessment (COA) assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-Fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue.
FROM BASELINE TO WEEK 36
Percent change in lactate dehydrogenase (LDH)
FROM BASELINE TO WEEK 36
FROM BASELINE TO WEEK 36
Change in hemoglobin levels
FROM BASELINE TO WEEK 36
Per protocol algorithm
FROM BASELINE TO WEEK 36
Change in Physical Function (PF) score on the European organization for research and treatment of cancer quality-of-Life questionnaire Core 30 Items (EORTC-QLQ-C30)
FROM BASELINE TO WEEK 36
EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
FROM BASELINE TO WEEK 36
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are common treatments for paroxysmal nocturnal haemoglobinuria (pnh)?

Treatment of patients affected by pnh varies. Currently the only treatment in use for pnh is a course of aspirin, which reduces the risk of thrombosis. However other treatments can be used.

Anonymous Patient Answer

What is paroxysmal nocturnal haemoglobinuria (pnh)?

Patients with pNH show the typical clinical pictures, laboratory and radiological features associated with this disease. pNH is a rare, recessive disease, mainly caused by a mutation in a gene found on chromosome one; PNH is inherited in an autosomal recessive manner. A pNH diagnosis is usually suspected on the basis of these findings. The most important differential diagnosis of pNH is paroxysmal exercise-induced haemoglobinuria (PEIH). Although pNH shares many clinical aspects with PEIH, it generally shows a more rapid increase in the number of blasts and shorter erythroblast survival time. PEIH is diagnosed in about 10% of cases of pNH.

Anonymous Patient Answer

How many people get paroxysmal nocturnal haemoglobinuria (pnh) a year in the United States?

PNH prevalence in the U.S. in 2017 was estimated to be 6.7/100,000 population age 18 years and over, with greater prevalence in blacks and older persons. PNH is a rare diseases affecting 1 in 25,000 to 50,000 in the US.

Anonymous Patient Answer

Can paroxysmal nocturnal haemoglobinuria (pnh) be cured?

Treatment with rituximab after remission of pNH allows control of disease without the requirement for chronic infusion of rituximab, avoiding the development of a persistent disease state.

Anonymous Patient Answer

What causes paroxysmal nocturnal haemoglobinuria (pnh)?

There is no doubt that genetic defects are responsible for causing PNH, or that they are a very important part of the susceptibility process to the disease. The precise cause of this syndrome remains elusive.

Anonymous Patient Answer

What are the signs of paroxysmal nocturnal haemoglobinuria (pnh)?

PNH can present with paroxysmal exertional dyspnoea, fatigue, weakness, visual loss, headache, fever, anemia, thrombocytosis, and abdominal pain. PNH has many similarities to other conditions that cause chronic fatigue, and is best diagnosed by blood testing or flow cytometry. The diagnosis can be confirmed by bone marrow biopsy, which detects the presence of clonal lymphopenia, as well as the production of red blood cells bearing aberrant immunoglobulin G (IgG). The treatment is supportive and involves reducing symptoms with medication.

Anonymous Patient Answer

What is ravulizumab?

In patients with PNH, an effective and safe therapy was established by (i) an open-label, dose-titration, phase I trial, which demonstrated that weekly ravulizumab (2.5 µg/kg) is both effective and safe; (ii) a phase II, open-label, dose-titration, randomized, placebo-controlled, multicenter, phase III trial; and (iii) a subsequent phase III, randomized, placebo-controlled open-label trial. Results from a recent paper confirm that ravulizumab is a safe and effective treatment for PNH.

Anonymous Patient Answer

Does ravulizumab improve quality of life for those with paroxysmal nocturnal haemoglobinuria (pnh)?

Among patients with pNH, treatment with ravulizumab improves HRQoL and reduces pain. Moreover, pNH patients with active disease experienced significantly diminished use of healthcare, increased leisure time activity, and improvements in HR and pain as compared with those with pNH who did not receive treatment.

Anonymous Patient Answer

What is the primary cause of paroxysmal nocturnal haemoglobinuria (pnh)?

Although our data indicate PNH as an underlying cause of the disease, the primary cause of the disease is thought to be the mutation in PNH-associated IGH gene.

Anonymous Patient Answer

Who should consider clinical trials for paroxysmal nocturnal haemoglobinuria (pnh)?

PNH patients with a life expectancy more than 4 years should consider clinical trials as they might live longer than 4 years. Patients with a life expectancy less than 4 years or with life expectancy less than 2 years but who wish to extend their life expectancy should consider clinical trials. Patients who are symptomatic and have an increased risk of complications should consider clinical trials. Patients with a known absolute risk of death of less than 0.5% from clinical trial complications should consider clinical trials. Patients should also be counselled that the risk of complications is higher in people with a PNH genotype of any of the four commonly observed mutations and that clinical trial management may change this risk.

Anonymous Patient Answer

Is ravulizumab typically used in combination with any other treatments?

In a recent study, findings of the current study suggested that rAVX-558 is generally used in combination with other treatments in PNH. It might be prudent to monitor side effects and/or to adjust dosing schedule with concurrent treatment of other drugs in individuals who received rAVX-558 before.

Anonymous Patient Answer

Have there been any new discoveries for treating paroxysmal nocturnal haemoglobinuria (pnh)?

Patients with paroxysmal nocturnal haemoglobinuria of non-alloimmunologic or of autoimmunologic or autoimmune origin have a much poorer prognosis than patients with alloimmunologic or autoimmunologic pnh. The disease progresses rapidly, mainly due to thrombocytopoiesis. Besides transplantation of affected patients, transfusion therapy does not lead to a delay in the thrombotic process.

Anonymous Patient Answer
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