Europas førende søgning efter kliniske forsøg

Human Apotransferrin

Clinical trials with Human Apotransferrin are studying whether replacement therapy can help people with congenital atransferrinaemia or hypotransferrinaemia. The trial data focus on safety, pharmacokinetics, and whether blood values such as hemoglobin and ferritin can return toward normal.

Table of contents

Trial overview

The available trial data describe one interventional study of Human Apotransferrin in patients with congenital atransferrinaemia or hypotransferrinaemia.[1] The study title says it is investigating treatment with apotransferrin in patients with atransferrinemia, and the brief summary says the goal is to study pharmacokinetics, efficacy, and safety of apotransferrin replacement therapy.[1]

Who is being studied

This trial focuses on a very rare patient group: people with congenital atransferrinaemia or hypotransferrinaemia.[1] “Congenital” means the condition is present from birth.[1] The planned enrollment is 5 participants, so this is a very small study designed for a rare disease population.[1]

Treatment and study design

The study uses Human Apotransferrin as the intervention, listed as a drug given by intravenous infusion, which means it is delivered into a vein.[1] The intervention is described as Human Apotransferrin (50 g/l) at 360 mg/kg milligram(s)/kilogram.[1] The trial is an interventional study, so researchers actively give the treatment and then observe the results.[1]

What the researchers measure

The primary outcomes are increase of hemoglobin to the normal range, pharmacokinetics of transferrin, and decrease of serum ferritin to the normal range.[1] Hemoglobin is a blood substance that helps carry oxygen, so an increase toward normal suggests the blood picture may improve.[1] Serum ferritin is a blood test linked to iron storage, and the study checks whether it moves down toward the normal range.[1] Pharmacokinetics means how the body handles the treatment, including how it is processed over time.[1]

Trial phase and status

The trial is in Phase 2, which is a stage that usually looks more closely at whether a treatment works while still monitoring safety.[1] The status is listed as Authorised.[1] Because the condition is rare and the enrollment is only 5 people, the study is likely designed to gather detailed information from a very small group of patients.[1]

Important terms for patients

  • Apotransferrin replacement therapy means giving the missing or low substance back to the body so researchers can see whether blood values improve.[1]

  • Efficacy means how well the treatment works in the people who receive it.[1]

  • Safety means whether the treatment can be given without causing unacceptable problems, although the trial data here do not list specific side effects.[1]

  • Normal range means the usual values seen in healthy blood tests, which the study uses as a target for hemoglobin and ferritin.[1]

Trial ID Phase Condition studied Status Enrollment
NCT01797055 Phase 2 Congenital atransferrinaemia / hypotransferrinaemia Authorised 5

FAQ

  • What is being studied in the Human Apotransferrin trial? The trial is studying apotransferrin replacement therapy in people with congenital atransferrinaemia or hypotransferrinaemia. It looks at whether treatment can improve blood test results and how the body handles the treatment.
  • Who can take part in these trials? The available trial data focus on patients with congenital atransferrinaemia or hypotransferrinaemia. The study is very small, so participation is limited to a specific rare patient group.
  • What phase is the Human Apotransferrin trial? The trial is in Phase 2. This means it is testing how well the treatment works and continuing to look at safety and how the body processes it.
  • What results are the researchers measuring? The main results are hemoglobin, transferrin pharmacokinetics, and serum ferritin. In simple terms, they want to see whether blood levels improve and whether the treatment moves ferritin toward the normal range.
  • How many people are in the study? The trial plans to include 5 participants. This small number fits the rare nature of the condition being studied.

Igangværende kliniske forsøg for Human Apotransferrin

  • Test af apotransferrin behandling hos patienter med medfødt mangel på transferrin i blodet

    Rekrutterer

    1 1
    Undersøgte sygdomme:
    Undersøgte lægemidler:
    Tyskland Italien Spanien

Ordliste

  • Atransferrinemia: A very rare inherited condition in which the body has too little transferrin, a blood protein. The trial studies people with this condition.
  • Hypotransferrinemia: A condition where transferrin levels are lower than normal. The trial includes patients with this rare condition.
  • Congenital: Present from birth. In this trial, the condition is congenital, meaning people are born with it.
  • Phase 2: A trial stage that looks more closely at whether a treatment works and continues to check safety.
  • Interventional study: A study where participants receive a treatment so researchers can measure its effects.
  • Pharmacokinetics: How the body takes in, moves, and removes a treatment. The trial measures this for transferrin.
  • Hemoglobin: A substance in red blood cells that carries oxygen. The study checks whether hemoglobin rises into the normal range.
  • Serum ferritin: A blood test that reflects iron storage in the body. The study looks for a decrease toward the normal range.
  • Replacement therapy: Treatment that replaces something the body does not have enough of. Here, the trial is testing apotransferrin replacement therapy.

Referencer

  1. https://kliniske-forsoeg.dk/forsog/test-af-apotransferrin-behandling-hos-patienter-med-medfodt-mangel-pa-transferrin-i-blodet/