Understanding Scleroderma: An In-Depth Overview

Scleroderma

Scleroderma is multifaceted, manifesting in a spectrum of symptoms that vary across individuals. At its core, the disease is characterised by inflammation, vascular issues, and fibrosis. These manifestations can translate to symptoms like tightening of the skin, ulcers on digits, and various internal complications.

Research methodologies globally

Research on Scleroderma has diverse methodologies across the world.

Australia has one of the highest prevalence rates of systemic sclerosis (SSc) worldwide and predominantly investigates the disease’s epidemiology—understanding its patterns, distribution, and determinants.

Clinical trials, both for new treatments and repurposing existing ones, stand out in their rigorous demand for evidence and specifically defined outcomes.

Clinical trials

Clinical trials are structured investigations, designed meticulously to balance patient safety with determining a drug’s efficacy. The phases include:

Phase One:

The first time a substance is given to a human.
Usually involves normal, healthy volunteers.
Administered in a special laboratory.
Participants are often monitored in a hospital-like setting for around seven days after a single dose.
Aimed at observing immediate side effects.
Starts with one person, waits, then progresses to another.
Doses increase incrementally with each new participant.
Long-term side effects are also observed.
After determining a safe dose and understanding metabolism, multiple doses are given over extended periods.
Generally involves a small number of young, healthy participants.

Phase Two:

Involves testing the substance in individuals with the target disease.
Mainly a safety study with some focus on effectiveness.
Typical study size for diseases like scleroderma is between 30 to 80 patients.
Looks at how the substance is excreted, its absorption, and its duration in the body.
Participants often undergo numerous blood tests.
Some studies may involve different doses versus a placebo.
Duration can range from six to twelve months with frequent visits.
Drug absorption and clearance are studied in detail.

Phase Three:

Larger studies focusing on the substance’s effectiveness.
Often ‘double-blind, placebo-controlled’ means neither participants nor doctors know who gets the active substance or placebo.
For diseases like scleroderma, trials often require three or four hundred participants.
Global multi-centre trials might be needed.
Duration often lasts a year or more.
Open-label extensions might be included where all participants receive the active drug.

Phase Four:

Focuses on long-term safety and can span several years.
Often large open-label studies.
May compare two different treatments.
Used to assess long-term risks such as cardiovascular or cancer risks.
The substance might already be on the market or about to be.
Less common in diseases like scleroderma as fewer effective treatments exist.
Blinding in Clinical Trials:

Single Blind: Only participants do not know what they’re receiving.

Double Blind: Neither participants nor doctors know who’s receiving what.

Purpose of Blinding:

Minimise bias in results.
Especially important when outcomes are subjective (e.g., quality of life questionnaires).
Ensures neither participants’ nor doctors’ expectations influence the trial outcomes.
Pharmacy typically manages blinding with randomized procedures.

Placebos play an integral role, allowing researchers to distinguish the genuine effects of a drug from the disease’s natural trajectory. Choosing the right participants is also critical to ensure valid results.

Engaging in clinical trials

Participation in a clinical trial follows a set journey.

It begins with recruitment and progresses to a detailed breakdown of the trial’s processes. After initial screenings, participants are categorised into groups, and the trial commences.

Potential participants are encouraged to understand every facet of the trial, from the number of required visits to potential side effects.

Maintaining discretion about trial details, especially when sharing on platforms such as social media, is also emphasised.

Being involved in a trial offers an opportunity to be at the forefront of medical advancements.

However, it’s essential to remember that new treatments, while promising, come without guarantees.

Scleroderma-Specific clinical trials

A range of clinical trials are tailored specifically for those with scleroderma.

For instance, one trial focuses on a novel drug from Boehringer Ingelheim, aimed at those with early-stage diffuse scleroderma. Various other trials have distinct criteria and objectives.

Final thoughts

Scleroderma, with its varied manifestations and complexities, continues to pose challenges in both understanding and treatment.

With ongoing research and clinical trials, there’s hope for enhanced understanding and more effective treatments.

For those living with scleroderma, staying updated and informed can make a world of difference. Connecting with others can also be hugely beneficial. Click here to find out more about support in your area.

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