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Apadamtase Alfa

Clinical trials investigating Apadamtase Alfa are studying whether it is safe and effective for people with thrombotic thrombocytopenic purpura, including immune-mediated and congenital forms. The trials look at treatment given either as prevention or on demand, and they measure safety, tolerability, and other outcomes.

Table of Contents

Trial overview

Two clinical trials investigated Apadamtase Alfa in people with thrombotic thrombocytopenic purpura, a rare blood disorder. One trial studied immune-mediated TTP, and the other studied congenital TTP.[1][2]

Both studies were interventional, which means researchers gave a treatment and then checked the results.[1][2]

Conditions studied

The first trial focused on immune-mediated thrombotic thrombocytopenic purpura (iTTP), a form of TTP linked to the immune system.[1]

The second trial focused on congenital thrombotic thrombocytopenic purpura (cTTP), which is the inherited form of the disease and is present from birth.[2]

These trials did not study other conditions, based on the source data provided.[1][2]

Study designs and phases

NCT05714969 was a Phase 2 study and looked at treatment with minimal to no plasma exchange in patients with iTTP.[1]

NCT04683003 was a Phase 3 continuation study and evaluated prophylactic and on-demand treatment in people with cTTP.[2]

In the source data, the Phase 3 study states that there was no primary efficacy endpoint because the main objective was long-term safety.[2]

Who participated

The Phase 2 iTTP study enrolled 36 participants.[1]

The Phase 3 cTTP study enrolled 75 participants.[2]

From the trial records provided, the target groups were people with either immune-mediated TTP or congenital TTP.[1][2]

What was measured

The main outcome in the Phase 2 study was the incidence of adverse events, serious adverse events, and special interest adverse events after any dose of the study treatment.[1]

The Phase 3 study measured long-term safety and tolerability, including treatment-emergent adverse events and serious adverse events in both the prophylactic and on-demand groups.[2]

These outcomes show that the trials were mainly designed to learn how safe the treatment was in these patient groups, rather than to compare many different disease outcomes.[1][2]

Key points for patients

Apadamtase Alfa was studied in two completed trials, both in rare forms of TTP.[1][2]

One study tested a Phase 2 approach in iTTP with minimal to no plasma exchange, while the other tested Phase 3 long-term use in cTTP with preventive and on-demand treatment.[1][2]

The main focus across both studies was safety, including adverse events and serious adverse events.[1][2]

Because the source data are limited to these two trials, this article only reflects the trial details provided here.[1][2]

Trial ID Phase Condition studied Status Enrollment
NCT05714969 Phase 2 Immune-mediated thrombotic thrombocytopenic purpura (iTTP) Completed 36
NCT04683003 Phase 3 Congenital thrombotic thrombocytopenic purpura (cTTP) Completed 75

FAQ

  • What conditions are being studied in Apadamtase Alfa trials? The trials study thrombotic thrombocytopenic purpura, also called TTP. One trial focuses on immune-mediated TTP, and another focuses on congenital TTP.
  • What is the main goal of these trials? The main goal is to check safety and long-term tolerability. One study also looks at treatment use with minimal to no plasma exchange.
  • What phases are the Apadamtase Alfa trials? One trial is Phase 2, and the other is Phase 3. These phases help researchers learn more about safety and how the treatment performs in the target group.
  • Who took part in these studies? One study enrolled 36 patients with immune-mediated TTP. The other enrolled 75 patients with congenital TTP.
  • What outcomes were measured? The studies measured safety outcomes such as adverse events, serious adverse events, and other treatment-related events. The Phase 3 study did not use a primary efficacy endpoint because its main aim was long-term safety.

Ongoing Clinical Trials on Apadamtase Alfa

  • Study on the Safety and Effectiveness of TAK-755 (Apadamtase Alfa) for Patients with Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Austria France Germany Italy Poland Spain

  • Study on the Safety of TAK-755, Rituximab, and Glucocorticoids for Patients with Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP)

    Not recruiting

    2 1 1 1
    Investigated drugs:
    Austria Greece Italy Poland Spain

Glossary

  • Thrombotic thrombocytopenic purpura (TTP): A rare blood disorder where tiny clots can form in small blood vessels. This can lower the number of platelets, which are cells that help blood clot.
  • Immune-mediated TTP (iTTP): A form of TTP caused by the immune system. The immune system attacks a normal body process and leads to the disease.
  • Congenital TTP (cTTP): A form of TTP that is present from birth. It is linked to an inherited problem.
  • Phase 2: A trial stage that usually looks more closely at safety and early signs of benefit in a smaller group of people.
  • Phase 3: A later trial stage that often includes more people and helps researchers learn more about safety and how well a treatment works.
  • Interventional study: A study in which researchers give a treatment or procedure and then measure the effects.
  • Plasma exchange: A procedure that removes part of the blood plasma and replaces it. It is used in some blood disorders.
  • Prophylactic treatment: Treatment given to help prevent a disease flare or new episode before it happens.
  • On-demand treatment: Treatment given when symptoms or a disease episode appears, rather than on a regular schedule.
  • Adverse event (AE): Any unwanted medical problem that happens during a study, whether or not it is caused by the treatment.
  • Serious adverse event (SAE): A more severe unwanted medical problem, such as one that causes hospital care, major disability, or is life-threatening.
  • Treatment-emergent adverse event (TEAE): An unwanted medical problem that starts or gets worse after treatment begins.

References