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(R)‑Tetrahydrofuran‑3‑Yl 4‑(6‑(5‑(4‑Ethoxy‑1‑Isopropylpiperidin‑4‑Yl)Pyridin-2-Yl)Pyrrolo[1,2-B]Pyridazin-4-Yl)Piperazine-1-Carboxylate Sesquisuccinate

This article discusses the ongoing clinical trials of fidrisertib (IPN60130), a promising drug being studied for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). FOP is a rare genetic disorder characterized by the formation of extra bone in muscles and connective tissues. The trials aim to assess the safety and effectiveness of fidrisertib in both children and adults with FOP, focusing on its ability to inhibit new bone formation and improve patients’ quality of life.

Table of Contents

What is Fidrisertib?

Fidrisertib, also known as IPN60130, is an experimental medication being developed to treat a rare genetic condition called Fibrodysplasia Ossificans Progressiva (FOP). The scientific name for this drug is (R)‑tetrahydrofuran‑3‑yl 4‑(6‑(5‑(4‑ethoxy‑1‑isopropylpiperidin‑4‑yl)pyridin-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate. It comes in the form of hard capsules that are taken by mouth.[1]

What is Fibrodysplasia Ossificans Progressiva (FOP)?

Fibrodysplasia Ossificans Progressiva (FOP) is a very rare and serious genetic disorder. In this condition, the body’s soft tissues (like muscles, tendons, and ligaments) gradually turn into bone over time. This process is called heterotopic ossification (HO). FOP can severely limit a person’s movement and cause significant pain and disability.[1]

How Fidrisertib Works

While the exact mechanism is not fully explained in the provided information, Fidrisertib is designed to target the underlying cause of FOP. It aims to inhibit or slow down the process of heterotopic ossification, potentially preventing new bone formation in soft tissues.[1]

Current Clinical Trial

Fidrisertib is currently being studied in a Phase 2 clinical trial. This trial is designed to evaluate how well the drug works (its efficacy) and how safe it is when used to treat both children and adults with FOP. The study will compare two different dosing regimens of Fidrisertib against a placebo (a substance with no active medication).[1]

Who Can Participate in the Trial?

The trial is open to both male and female patients with FOP, including children and adults. Here are some key eligibility criteria:

  • Participants must be at least 5 years old (younger children may be included later in the study)
  • Must have a clinical diagnosis of FOP with a specific genetic mutation (R206H ACVR1 or other FOP variants)
  • Must have shown disease progression in the year before screening
  • Must weigh at least 10 kg (about 22 pounds)
  • Must be able to undergo certain medical tests without sedation

There are also several factors that might prevent someone from participating, such as certain heart conditions, liver problems, or use of specific medications.[1]

What the Trial Aims to Achieve

The main goals of this clinical trial are:

  1. To see if Fidrisertib can slow down or stop new bone formation in soft tissues (heterotopic ossification) compared to placebo
  2. To evaluate how safe Fidrisertib is for people with FOP

The researchers will use special low-dose whole-body CT scans to measure new bone formation. They will also look at other factors such as:

  • The number of disease flare-ups
  • Changes in pain levels
  • Effects on joint movement
  • Impact on overall physical function

These measurements will help determine how effective Fidrisertib is in managing FOP symptoms and progression.[1]

Potential Benefits of Fidrisertib

While it’s important to note that the benefits of Fidrisertib are still being studied, the researchers hope that it might:

  • Reduce the formation of new bone in soft tissues
  • Decrease the number and severity of disease flare-ups
  • Improve or maintain joint movement
  • Reduce pain associated with FOP
  • Improve overall physical function and quality of life for people with FOP

However, these potential benefits are not guaranteed and will be carefully evaluated during the clinical trial.[1]

Safety Considerations

As with any experimental treatment, there may be risks and side effects associated with Fidrisertib. The clinical trial will closely monitor participants for any adverse effects. Some areas of particular focus include:

  • Heart function (through ECGs and echocardiograms)
  • Liver function
  • Blood cell counts
  • Eye health
  • Overall physical health

Participants in the trial will have regular check-ups and tests to ensure their safety throughout the study period.[1]

Aspect Details
Drug Name Fidrisertib (IPN60130)
Condition Studied Fibrodysplasia Ossificans Progressiva (FOP)
Trial Phase Phase 2
Participant Age Range 5 years and older (adults and children)
Primary Objectives 1. Evaluate efficacy in inhibiting new heterotopic ossification
2. Assess safety in FOP patients
Key Measurements 1. Change in heterotopic ossification volume
2. Number of new bone lesions
3. Flare-up frequency
4. Pain intensity
5. Physical function
Imaging Technique Low-dose whole-body computed tomography (WBCT)
Treatment Duration Up to 60 months
Administration Route Oral

FAQ

  • What is Fibrodysplasia Ossificans Progressiva (FOP)? Fibrodysplasia Ossificans Progressiva (FOP) is a rare genetic disorder where the body forms extra bone in muscles, tendons, and other connective tissues. This can lead to restricted movement and other complications.
  • What is fidrisertib (IPN60130)? Fidrisertib (IPN60130) is an investigational drug being studied for the treatment of FOP. It is designed to inhibit the formation of new heterotopic ossification (extra bone growth) in patients with FOP.
  • Who can participate in these clinical trials? The trials include both children (as young as 5 years old) and adults with FOP who have the R206H ACVR1 mutation or other FOP variants associated with progressive heterotopic ossification. Participants must have shown disease progression in the year before screening.
  • What are the main objectives of the clinical trials? The main objectives are to evaluate the efficacy of fidrisertib in inhibiting new heterotopic ossification compared to placebo, and to assess its safety in both adult and pediatric FOP patients.
  • How is the effectiveness of fidrisertib being measured? The effectiveness is primarily measured by the change in heterotopic ossification volume using low-dose whole-body computed tomography (WBCT). Other measures include the number of new bone lesions, flare-up frequency, pain intensity, and physical function assessments.

Ongoing Clinical Trials on (R)‑Tetrahydrofuran‑3‑Yl 4‑(6‑(5‑(4‑Ethoxy‑1‑Isopropylpiperidin‑4‑Yl)Pyridin-2-Yl)Pyrrolo[1,2-B]Pyridazin-4-Yl)Piperazine-1-Carboxylate Sesquisuccinate

  • Study on the Effects of Fidrisertib for Treating Fibrodysplasia Ossificans Progressiva in Children and Adults

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany Italy The Netherlands Portugal +2

Glossary

  • Fibrodysplasia Ossificans Progressiva (FOP): A rare genetic disorder characterized by the formation of extra bone in muscles, tendons, and other connective tissues, leading to restricted movement and other complications.
  • Heterotopic Ossification (HO): The formation of bone tissue outside of the skeleton, which is a key feature of FOP.
  • ACVR1 mutation: A genetic mutation in the ACVR1 gene that is associated with FOP. The R206H mutation is the most common variant.
  • Flare-up: A period of increased FOP activity, often characterized by swelling, pain, and potential new bone formation.
  • Whole Body Computed Tomography (WBCT): An imaging technique used to assess the extent of heterotopic ossification throughout the body, excluding the head.
  • Cumulative Analogue Joint Involvement Scale (CAJIS): A measurement tool used to assess joint function and mobility in FOP patients.
  • FOP-PFQ: Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire, a tool used to evaluate physical function in FOP patients.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Exposure-Response Relationship: The association between the amount of drug a patient receives (exposure) and the observed effects (response) of the medication.
  • Echocardiography: An ultrasound-based imaging technique used to assess heart structure and function.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effects-of-fidrisertib-for-treating-fibrodysplasia-ossificans-progressiva-in-children-and-adults/