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Study on the Effectiveness of INCB000928 for Patients with Fibrodysplasia Ossificans Progressiva

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What is this study about?

This clinical trial is focused on studying a rare disease called Fibrodysplasia Ossificans Progressiva (FOP). FOP is a condition where the body’s soft tissues, like muscles and tendons, gradually turn into bone, leading to stiffness and movement difficulties. The study is testing a new treatment called INCB000928, which is taken as a film-coated tablet. This medication is an ALK2 inhibitor, which means it is designed to block a specific protein that may play a role in the abnormal bone growth seen in FOP.

The purpose of the study is to evaluate how effective and safe INCB000928 is in preventing new bone growth in people with FOP. Participants in the study will be randomly assigned to receive either the medication or a placebo, which looks like the medication but does not contain the active ingredient. The study is conducted in a double-blind manner, meaning neither the participants nor the researchers know who is receiving the actual medication or the placebo, to ensure unbiased results.

Throughout the study, participants will take the medication or placebo for a period of time and will have regular check-ups to monitor their health and any changes in their condition. The study will assess the total volume of new bone growth and the number of new flare-ups, which are episodes of swelling and pain. The study aims to provide valuable information on whether INCB000928 can help manage FOP and improve the quality of life for those affected by this challenging condition.

1 joining the study

Upon joining the study, participants are required to provide informed consent. For adults, this involves signing a consent form. For adolescents aged 12 to 18, consent from a parent or guardian and assent from the adolescent are necessary.

Participants must be able to swallow tablets or receive them through a feeding tube.

2 initial assessment

Participants undergo an initial assessment to confirm a clinical diagnosis of fibrodysplasia ossificans progressiva (FOP). This includes checking for congenital malformation of the great toes, episodic soft-tissue swelling, or progressive heterotopic ossification (HO).

Participants must report FOP disease activity within one year of the screening visit.

3 medication administration

Participants are randomly assigned to receive either the medication INCB000928 or a placebo. The medication is administered orally in the form of a film-coated tablet.

The dosage and frequency of administration are determined by the study protocol and are consistent throughout the trial period.

4 monitoring and assessments

Participants undergo regular monitoring and assessments to evaluate the efficacy and safety of the treatment. This includes low-dose whole-body computed tomography (WBCT) scans, excluding the head, to assess the total volume of new HO at Week 24.

Other assessments include monitoring the number of new flares, evaluating flare-related symptoms, and checking for any adverse events (AEs) or serious adverse events (SAEs).

5 follow-up period

The study includes a follow-up period from Week 24 to Week 48 for participants initially randomized to placebo. During this period, the total volume and number of new HO lesions are assessed again.

Participants continue to be monitored for any new flares and the proportion of participants with new HO is evaluated.

Who Can Join the Study?

  • Must provide informed consent or assent:
    • For adults (18 years or older), must understand and be willing to sign a consent form.
    • For adolescents (12 to under 18 years), a parent or legal guardian must provide written consent, and the adolescent must give written agreement.
  • Must be a female or male adult or adolescent aged 12 years or older.
  • Must have a clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP), which is based on certain physical findings like toe malformations, soft-tissue swelling, or progressive bone formation in muscles.
  • Must have reported FOP disease activity within one year before the screening visit.
  • Must be able to swallow and keep down tablets taken by mouth, either whole or crushed and mixed with food or liquids, or be able to receive crushed tablets through a feeding tube.
  • Must agree to avoid becoming pregnant or fathering children during the study.
  • Must be willing and able to undergo a low-dose whole-body CT scan (excluding the head) without needing a breathing tube.
  • Must be willing and able to follow all study procedures and requirements and attend all study visits as outlined in the study plan.

Who Cannot Join the Study?

  • Participants who have any other serious health condition that might interfere with the study.
  • Individuals who are currently taking medications that could affect the study results.
  • People who have participated in another clinical trial recently.
  • Pregnant or breastfeeding women.
  • Individuals who are unable to follow the study procedures or attend study visits.
  • Participants who have allergies to the study medication or its ingredients.
  • Anyone who has a history of drug or alcohol abuse.
  • Individuals with a mental health condition that might affect their ability to participate.
  • People who have had a recent surgery or are planning to have surgery during the study.
  • Participants who have an infection or illness that requires treatment with antibiotics.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy
Unidade Local De Saúde De Santa Maria, E.P.E. Lisbon Portugal

Other Sites

Site Name City Country Status
Hospital Universitario Ramon Y Cajal Madrid Spain
Hopital Beaujon Clichy France
Ufruibtmkj Hnxaldfv Cewbmft Cologne Germany
Angevzchc Uqg Amsterdam The Netherlands

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Recruiting
12.04.2022
Germany Germany
Recruiting
12.04.2022
Italy Italy
Recruiting
12.04.2022
Portugal Portugal
Not recruiting
12.04.2022
Spain Spain
Recruiting
12.04.2022
The Netherlands The Netherlands
Recruiting
12.04.2022

Trial locations

Investigated drugs:

INCB000928 is a medication being studied for its potential to prevent new bone formation in people with a rare condition called Fibrodysplasia Ossificans Progressiva (FOP). This condition causes soft tissues to gradually turn into bone, leading to severe movement restrictions. The trial aims to evaluate how effective, safe, and tolerable this medication is for patients with FOP.

Investigated diseases:

Fibrodysplasia Ossificans Progressiva – This is a rare genetic disorder characterized by the abnormal development of bone in areas where bone should not form, such as muscles, tendons, and other connective tissues. The disease progresses through episodes known as “flare-ups,” where new bone forms, leading to joint immobility and fusion of bones. Over time, these flare-ups can result in significant physical limitations as the body becomes increasingly encased in bone. The condition is progressive, meaning it worsens over time, and each flare-up can lead to more extensive bone formation. The disease is caused by a mutation in the ACVR1 gene, which affects bone growth and development.

Trial ID:
2023-504129-38-00
Protocol code:
INCB 00928-201
NCT ID:
NCT05090891
Trial Phase:
Therapeutic exploratory (Phase II)

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