RecruitingOBSERVATIONAL

Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).

This study is a long-term registry designed to keep an eye on the safety and how well Increlex® works for children and teenagers with a condition called Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD). Essentially, it's a way for doctors and researchers to gather information over time about patients who are already taking Increlex® as part of their regular treatment. The goal is to understand the medication's effects, especially its safety, for at least five years after treatment ends. This is not a clinical trial where new treatments are tested, but rather an observational study that collects real-world data from patients in several countries, including the UK, to ensure the medication is used safely and effectively as approved.

At a glance

Status

Recruiting

Sponsor

Esteve Pharmaceuticals, S.A.

Enrolment target

500

Start

09 Dec 2008

Estimated completion

31 Dec 2027

IGF1 Deficiency

What is this study about?

This study is called the Increlex® Global Registry. It's not a typical clinical trial where new medicines are tested. Instead, it's a way for healthcare professionals to keep a close watch on the long-term safety and how well a medicine called Increlex® works in children and teenagers. This particular medicine is used to treat a specific growth condition known as Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD). Essentially, this condition means the body doesn't produce enough of a certain hormone needed for growth.

The main reasons for this registry are to gather important information about any potential side effects of Increlex® over time, and to also understand how effective it is in helping children and teenagers with SPIGFD grow and develop. Data will be collected for at least five years after a patient finishes their treatment, giving a comprehensive picture of its long-term impact. This information is crucial for ensuring the medication continues to be used safely and effectively, benefiting those who need it.

Patients who are already taking Increlex® can be included in this registry. The information gathered helps the medical community understand the real-world experience of using this medication outside of a controlled trial setting. This is important for guiding doctors on how to best prescribe and monitor patients receiving Increlex® for SPIGFD, ultimately leading to better care.

Key takeaways

This is a long-term study monitoring an existing medicine, not a new drug trial.
It collects real-world information on Increlex® for children and teenagers with SPIGFD.
The main goals are to track long-term safety and effectiveness.
Participation involves sharing medical information from your routine care.
You won't be asked to change your treatment or have extra appointments.
Your data helps improve understanding and care for others with SPIGFD.

Who may be eligible?

This study is open to children and teenagers aged between 2 and 18 years old.

You might be able to take part if you are currently taking Increlex®, or are about to start taking it, for a condition called Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD). This applies if your doctor has prescribed Increlex® according to the approved guidelines for the medicine in your country, such as the UK.

However, you cannot join if you are already involved in another clinical study looking into Increlex® or any other treatment for growth problems. Also, you can't participate if you have a medical condition that means Increlex® isn't suitable for you, or if you're allergic to any of its ingredients. This includes if you have or are suspected of having cancer, or if your growth plates (the parts of your bones where growth happens) have already closed.

Quick self-check

Are you between 2 and 18 years old?
Are you currently taking, or planning to start, Increlex® for SPIGFD?
Are you not currently in another medical study for growth problems or Increlex®?
Do you not have any medical conditions or allergies that would prevent you from safely taking Increlex®?
Are your parents or legal guardians willing to give permission for you to participate?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to participate in this registry, it means that information about your Increlex® treatment will be collected and recorded. This is an "observational" study, which means you won't be given any new treatments or asked to do anything differently than your usual medical care. The study aims to gather data from your routine doctor visits and treatment with Increlex®, as prescribed by your doctor.

There are no specific extra visits or special assessments required just for this registry. The duration of your participation will depend on how long you are on Increlex® treatment and will continue for at least five years after your treatment ends to monitor long-term effects. The information will be collected from your existing medical records and follow-up appointments.

Potential risks and benefits

The potential benefits of participating include contributing valuable information that helps medical professionals understand the long-term safety and effectiveness of Increlex® for other children and teenagers with SPIGFD. This can improve future care and treatment guidelines. As this is an observational study, there are no additional medical risks beyond those already associated with taking Increlex® as prescribed by your doctor. You always have the right to withdraw from the registry at any time, for any reason, without it affecting your medical care.

Locations (58)

Children's Hospital of Orange County
Orange, United States· Recruiting
University of Miami Leonard M Miller
Miami, United States· Recruiting
University Of Miami Leonard M. Miller
Miami, United States· Recruiting
D&H National Research Centers
Miami, United States· Withdrawn
Cincinnati Children's Hospital Medical Center
Cincinnati, United States· Recruiting
UT Southwestern Medical Center
Dallas, United States· Not yet recruiting
Children's Health Specialty Center West Plano
Plano, United States· Withdrawn
Salzkammergut-Klinik Vöcklabruck
Vöcklabruck, Austria· Recruiting
Hôpital Amiens-Picardie
Amiens, France· Terminated
Centre Hospitalier de Blois
Blois, France· Recruiting
Hôpital Jean Verdier
Bondy, France· Recruiting
Hôpital Femme Mère-Enfant
Bron, France· Recruiting

+46 more sites — see the official record for the full list.

Common questions

What is SPIGFD?

SPIGFD stands for Severe Primary Insulin-like Growth Factor-1 Deficiency. It's a condition where the body doesn't produce enough of a specific hormone needed for normal growth.

Is this study testing a new medicine?

No, this study is not testing a new medicine. It's a registry that collects information about Increlex®, a medicine that is already approved and used by patients.

What information will be collected about me?

The registry will collect information about your health, your treatment with Increlex®, and how you respond to it over time. This helps doctors understand its safety and effectiveness.

Do I have to do anything extra if I join?

No, you don't have to do anything extra. The registry will collect information from your usual doctor's appointments and treatments, as prescribed by your medical team.

Can I stop participating in the registry at any time?

Yes, you can choose to stop participating in the registry at any time, for any reason, and it will not affect your medical care or treatment.

How to find out more

Esteve Global Clinical Development

increlexregistry@esteve.com Official trial record

Always speak to your GP or specialist before deciding to take part in a study.