Study of ianalumab for adults with primary immune thrombocytopenia or warm-antibody autoimmune hemolytic anemia who previously responded to ianalumab

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What is this study about?

This study involves two blood disorders. Immune thrombocytopenia is a condition where the immune system mistakenly destroys platelets, which are blood cells that help stop bleeding, leading to low platelet counts and increased risk of bruising and bleeding. Warm-antibody autoimmune hemolytic anemia is a condition where the immune system destroys red blood cells, which carry oxygen throughout the body, causing anemia with symptoms like fatigue and weakness. The study will use a medication called ianalumab, also known as VAY736, which is given through a vein. This medication is designed to affect certain immune system cells that contribute to these blood disorders. Participants may also receive other medications during the study including pain relievers, entecavir which is an antiviral medication, medications that help blood clotting, antihistamines for allergic reactions, danazol which affects hormone production and immune function, and glucocorticoids which are steroid medications that reduce inflammation.

The purpose of this study is to see if a second course of treatment with ianalumab can help people who previously received this medication and experienced improvement but then had their condition return. For people with immune thrombocytopenia, the study will look at whether the second treatment can maintain platelet counts and reduce the need for additional treatments. For people with warm-antibody autoimmune hemolytic anemia, the study will check if the second treatment can maintain hemoglobin levels, which reflects the amount of red blood cells in the blood, and reduce symptoms of anemia.

Participants in this study must have previously taken part in earlier research studies with ianalumab and must have experienced benefit from the treatment but later had their condition worsen. The study will involve receiving ianalumab infusions and regular monitoring visits where blood samples will be taken to measure blood cell counts and check for any side effects. The treatment period will last up to 16 weeks, and participants will be followed for several months afterward to assess how long the benefits last and to monitor safety. During the study, some participants may receive additional supportive treatments or rescue medications if needed to manage their symptoms.

1 Screening and enrollment

Your doctor will review your medical history and confirm that you previously participated in a study with ianalumab and had a positive response to the treatment.

If you have immune thrombocytopenia, a condition where your body does not have enough platelets to help blood clot properly, your platelet count must be checked. The count should be below 30,000 per microliter of blood.

If you have warm-antibody autoimmune hemolytic anemia, a condition where your immune system destroys your red blood cells, your hemoglobin level must be checked. Hemoglobin is a protein in red blood cells that carries oxygen. Your level should be at least 5 grams per deciliter but less than 10 grams per deciliter, and you should have symptoms related to anemia.

Your doctor may have started you on rescue medication or bridging therapy within 28 days before screening. These are temporary treatments used to manage your condition.

If you were already taking supportive care treatment in the previous study when your condition returned, you should continue this treatment at the same dose for at least 4 weeks before screening.

2 Treatment period with ianalumab

You will receive a second course of treatment with ianalumab, which is given through a vein in your arm. This is called an intravenous infusion.

The study will monitor how well this second course of treatment works for your condition.

The specific dosing schedule and number of infusions will be determined by the study protocol.

3 Monitoring during treatment for immune thrombocytopenia

If you have immune thrombocytopenia, your doctor will monitor your platelet count regularly throughout the study.

The goal is to keep your platelet count above 30,000 per microliter of blood after 8 weeks from the start of treatment.

Your doctor will check whether you need any rescue treatments after the first 8 weeks of treatment.

If you are taking a medication called thrombopoietin receptor agonist, which helps your body make more platelets, your doctor will try to reduce the dose by week 24.

4 Monitoring during treatment for warm-antibody autoimmune hemolytic anemia

If you have warm-antibody autoimmune hemolytic anemia, your doctor will monitor your hemoglobin levels regularly throughout the study.

The goal is to achieve a hemoglobin level of at least 10 grams per deciliter, with an increase of at least 2 grams per deciliter from your starting level.

This improvement should last for at least 8 consecutive weeks between week 9 and week 25 of treatment.

Your doctor will monitor whether you need any rescue treatments or new medications for your condition.

5 Safety monitoring throughout the study

Your doctor will monitor you for any side effects or adverse events throughout the study.

Special attention will be given to monitoring for severe infections.

Blood samples will be taken to measure the level of ianalumab in your blood.

Blood samples will also be taken to check if your body develops antibodies against ianalumab. Antibodies are proteins your immune system makes in response to substances it recognizes.

6 Follow-up period

The study will continue to monitor your condition for up to 12 months after you start the second course of ianalumab.

Your doctor will assess whether the treatment has been successful in managing your condition without the need for additional rescue treatments or new medications.

Regular check-ups will be scheduled to evaluate your response to treatment and overall health.

Who Can Join the Study?

You must sign a document showing you agree to join the study before any study activities begin
You must be 18 years old or older when you sign the agreement form
If you have primary ITP (a condition where your immune system destroys blood cells called platelets, which help your blood clot): You must have been in a previous study where you received the study medicine called ianalumab or a placebo (a substance with no active medicine) along with other treatments, and your condition came back at least 2 years after your last treatment dose in that study
You may use emergency medicines or temporary treatments to help manage your condition within 28 days before joining this study. However, your platelet count (number of platelets in your blood) must be measured before starting these medicines and within 30 days before joining
If you have wAIHA (a condition where your immune system destroys your red blood cells, causing anemia, which means low levels of a protein called hemoglobin that carries oxygen in your blood): You must have had a positive test called DAT that confirms your condition, been in a previous study with ianalumab or placebo, and had a good response that lasted more than 2 years after your last dose
If you have wAIHA: Your condition must have come back with hemoglobin levels between 5 and 10 grams per deciliter, and you must have symptoms of anemia (such as tiredness or weakness) during the screening period or within 14 days before, or within 28 days before if you started emergency medicines
You may use emergency medicines or temporary treatments during the screening period or within 28 days before joining. Your hemoglobin level must be measured before starting these treatments and within 30 days before joining
If you were taking supportive care medicines in the previous study when your condition came back, you may continue them, but they must have stayed at the same dose for at least 4 weeks before joining this study

Who Cannot Join the Study?

The source data does not contain specific exclusion criteria for this clinical trial
This study is for people with immune thrombocytopenia (a condition where the body attacks its own blood cells that help with clotting) or warm autoimmune hemolytic anemia (a condition where the body destroys its own red blood cells)
The trial is designed for patients who previously received treatment with ianalumab, had improvement, but then had their condition return
Without detailed exclusion criteria listed in the source data, specific reasons why someone cannot participate are not available

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
University Hospital Jena KöR	Jena	Germany
Hospital Universitario De Salamanca	Salamanca	Spain
Centre Hospitalier Universitaire De Lille	Lille	France

Other Sites

Site Name	City	Country
Azienda Sanitaria Universitaria Giuliano Isontina	Trieste	Italy
ASST Fatebenefratelli Sacco	Milan	Italy
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hospital General Universitario Morales Meseguer	Murcia	Spain
Algemeen Ziekenhuis Delta	Roeselare	Belgium
Azienda Unita Locale Socio Sanitaria N 8 Berica	Vicenza	Italy
University Of Debrecen	Debrecen	Hungary
Spitalul Clinic Coltea	Bucharest	Romania
Fakultni Nemocnice Kralovske Vinohrady	Prague	Czechia
Universidade De Santiago De Compostela	Santiago De Compostela	Spain
Centre Hospitalier Universitaire De Caen Normandie	Caen	France
University Hospital Ostrava	Ostrava	Czechia
Apqelft Upxby Shxfixmbe Lltjyt Dx Baipigv	Bologna	Italy
Htcnxrth Vvbi dzwuqaux	Barcelona	Spain
Devbfjhvolknsiefoamkhyr Cmhtuy I Pnimeus Evvv	Plovdiv	Bulgaria

Trial status

Country	Status	Recruitment Start
Belgium	Not yet recruiting	01.11.2025
Bulgaria	Not yet recruiting	01.11.2025
Czechia	Not yet recruiting	01.11.2025
France	Not yet recruiting	01.11.2025
Germany	Not yet recruiting	01.11.2025
Hungary	Not yet recruiting	01.11.2025
Italy	Not yet recruiting	01.11.2025
Romania	Not yet recruiting	01.11.2025
Spain	Not yet recruiting	01.11.2025

Trial locations

Investigated drugs:

Ianalumab is an investigational medication being studied for adults with two blood disorders: primary immune thrombocytopenia (ITP), which is a condition where the body destroys its own blood platelets, and warm-antibody autoimmune hemolytic anemia (wAIHA), which is a condition where the body destroys its own red blood cells. This medication works by targeting specific cells in the immune system that may be causing these blood disorders. In this study, ianalumab is being given to patients who previously received this medication and had improvement in their condition, but later experienced a return of their symptoms.

Investigated diseases:

Immune Thrombocytopenia – Immune thrombocytopenia is a blood disorder in which the immune system mistakenly destroys platelets, which are cell fragments necessary for normal blood clotting. People with this condition have an unusually low number of platelets in their blood. The disease can develop suddenly or gradually over time. When platelet counts drop significantly, affected individuals may experience easy bruising, bleeding gums, or small red spots on the skin. The condition may occur without any known cause or may develop following infections or other triggering events. Some people experience periods when the platelet count improves, followed by times when it drops again.

Warm Autoimmune Hemolytic Anemia – Warm autoimmune hemolytic anemia is a condition in which the immune system produces antibodies that attack and destroy red blood cells at body temperature. The destruction of red blood cells occurs faster than the body can replace them, leading to anemia. People with this condition may feel tired, weak, and short of breath due to the reduced number of red blood cells. The skin and eyes may appear pale or yellowish as red blood cells break down. The spleen, which filters damaged blood cells, may become enlarged as it works to remove the damaged cells. Symptoms can develop gradually or appear suddenly, and the severity can vary from mild to more serious forms.

Trial ID:

2024-518231-11-00

Protocol code:

CVAY736Q12202B

NCT ID:

NCT07039422

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of ianalumab for adults with primary immune thrombocytopenia or warm-antibody autoimmune hemolytic anemia who previously responded to ianalumab

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?