AuthorisedTherapeutic exploratory (Phase II)Interventional

A Phase IIb, Monocentric, Non-Profit, Open-label Trial for the Intrathecal Administration of AAV9/AP4M1 for Hereditary Spastic Paraplegia Type 50 (SPG50)

This research study, called a Phase IIb trial, is investigating a new treatment named Melpida for a genetic condition known as Hereditary Spastic Paraplegia Type 50 (SPG50). SPG50 affects how people move, causing muscle stiffness and weakness. The main goal is to see if Melpida can improve important physical skills, like walking and other movements, over three years. Researchers will use special tests to measure movement and development as well as looking at overall well-being. This study helps doctors understand if Melpida is safe and effective for people living with SPG50, aiming to improve their quality of life.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii

Enrolment target

Start

06 Feb 2026

Hereditary Spastic Paraplegia Type 50 (SPG50)

What is this study about?

This study is a research project focusing on Hereditary Spastic Paraplegia Type 50, often shortened to SPG50. SPG50 is a rare genetic condition that primarily affects the nervous system, leading to issues with movement such as muscle stiffness (spasticity) and weakness, particularly in the legs. It can also impact other developmental areas. The new treatment being tested in this study is called Melpida.

The main purpose of this study is to find out if Melpida can help improve the major physical skills of people with SPG50. Researchers will be carefully observing and measuring changes in things like walking, standing, and other important movements over a period of about three years. They'll use special checklists designed to track these physical milestones.

Beyond just movement, the study will also look at other aspects of health and development. This includes assessing cognitive skills (like thinking and learning), overall disease severity, and how the condition affects daily life and well-being, both from the patient's and caregiver's perspective. They will also collect blood and spinal fluid samples to look for specific markers that might indicate changes related to the treatment.

Key takeaways

This study investigates Melpida for SPG50.
The goal is to improve movement and physical skills.
It will involve regular clinic visits over about three years.
Adults aged 18 and over with SPG50 are eligible.
Participation is voluntary, and you can withdraw anytime.

Who may be eligible?

To be considered for this study, you would need to be diagnosed with Hereditary Spastic Paraplegia Type 50 (SPG50).

Only adults aged 18 years and older can take part in this particular study. There are no restrictions based on whether you are male or female.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

I have been diagnosed with Hereditary Spastic Paraplegia Type 50 (SPG50).
I am 18 years old or older.
I understand the study involves a new treatment called Melpida.
I am able and willing to attend regular hospital visits for assessments.

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the study medication, Melpida. You would have regular visits to the study clinic where doctors will conduct various assessments. These assessments include physical examinations, tests to measure your movement and physical abilities (like walking and coordination), and evaluations of your cognitive development and overall well-being. You might also have blood tests and possibly spinal fluid tests. These visits will be spread out over approximately three years. The research team will explain the full schedule of visits and tests during an initial screening.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as access to a new treatment that isn't widely available yet, and contributes to understanding SPG50 better. However, there are also potential risks involved, which will be fully explained by the study team. These could include side effects from the medication or discomfort from tests. It's important to remember that participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Italy

Common questions

What is SPG50?

SPG50 is a rare genetic condition that primarily affects movement, causing muscle stiffness and difficulty with walking and other physical skills.

What is Melpida?

Melpida is the new treatment being tested in this study to see if it can help improve movement in people with SPG50.

How long will the study last?

The main part of the study where physical improvements are measured will last for about three years.

Will taking part in the study affect my current medical care?

No, your decision to join or leave the study will not affect the standard medical care you receive for SPG50.

What kind of tests will I have?

You will have physical exams, tests to measure your movement and development, and might have blood and spinal fluid tests.