HZN-825 QD for Sclerosis

1
Effectiveness
2
Safety
Organización Médica de Investigación, Ciudad Autónoma de Buenos Aires, Argentina
Sclerosis+4 More
HZN-825 QD - Drug
Eligibility
18+
All Sexes
Eligible conditions
Sclerosis

Study Summary

A Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis

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Eligible Conditions

  • Sclerosis
  • Scleroderma, Systemic
  • Scleroderma, Diffuse
  • Systemic; Sclerosis, Progressive
  • Diffuse Cutaneous Systemic Sclerosis

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether HZN-825 QD will improve 1 primary outcome and 8 secondary outcomes in patients with Sclerosis. Measurement will happen over the course of Baseline to Week 52.

Baseline to Week 52
Change from Baseline in HAQ-DI (Health Assessment Questionnaire - Disability Index) at Week 52
Change from Baseline in MDGA (Physician Global Assessment) at Week 52
Change from Baseline in PTGA (Patient Global Assessment) at Week 52
Change from Baseline in the Physical Effects subscale of the scleroderma skin patient-reported outcome (SSPRO-18) at Week 52
Change from Baseline in the Physical Limitations subscale of the scleroderma skin patient-reported outcome SSPRO-18 at Week 52
Change in FVC (forced vital capacity) percent predicted from Baseline to Week 52
Proportion of participants with an improvement in ≥3 of 5 core measures from Baseline: ≥20% in mRSS, ≥20% in HAQ-DI, ≥20% in PTGA, ≥20% in MDGA and ≥5% for FVC % predicted at Week 52 (ACR-CRISS-20)
Proportion of participants with an mRSS (modified Rodnan skin score) decrease of ≥5 points and 25% from Baseline at Week 52
Week 52
Responder rate (defined as ACR-CRISS [predicted probability] of at least 0.6) at Week 52

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

3 Treatment Groups

Placebo
HZN-825 300 mg twice daily (BID)
Placebo group

This trial requires 300 total participants across 3 different treatment groups

This trial involves 3 different treatments. HZN-825 QD is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

HZN-825 300 mg twice daily (BID)
Drug
300mg oral tablets given in the morning and evening
HZN-825 300 mg once daily (QD)
Drug
300mg oral tablets given in the morning and placebo in the evening
Placebo
Drug
Placebo will be given orally in the morning and evening

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: week 52
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly week 52 for reporting.

Closest Location

UT Physicians Rheumatology - Houston, TX

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 9 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
The person has symptoms that meet the criteria for scleroderma, as set by the American College of Rheumatology and the European League Against Rheumatism, and their total score is 9 or higher. show original
This patient has diffuse cutaneous SSc, which is a subset of scleroderma that affects the skin and is located proximal to the elbow and knee. show original
The patient must have been enrolled within 36 months of the first SSc symptom show original
The text states that the skin on the forearm of someone with SSc has thickened and is suitable for repeat biopsy. show original
Out of the people screened, those who had mRSS units ≥15 were more likely to have a relapse. show original
The person screened has a forced vital capacity (FVC) of at least 45% predicted. show original
The subject is willing and able to comply with the trial protocol and evaluations. show original
I consent to participate in this study show original
People aged 18 to 75 who are male or female. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can sclerosis be cured?

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Scleroderma not only prevents healing of broken or damaged tissues but also impairs tissue-repair mechanisms. Although no cure is known, early diagnosis and long-standing treatment have enabled many patients to maintain excellent health without disabling manifestations.

Unverified Answer

What are the signs of sclerosis?

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Sclerosis may be manifested through skin sores, decreased sensation, pain and loss of balance. It should be suspected if symptoms persist for more than 4 weeks in a person with an underlying systemic illness or history of joint pain.

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How many people get sclerosis a year in the United States?

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According to the results of this study, 0.8% of men and 1.1% of women got sclerosis during their lifetime in the United States. When analyzing the data by ethnicity, 0.7% of white men, 1.0% of black men and 0.3% of white women had gotten sclerosis. In the Asian population, the number of people who got sclerosis was marginally higher, at 0.8%. The number of Hispanics was the lowest at 0.1%. These data, unfortunately, are incomplete because we have not included self-reported cases. For this reason, we cannot make assumptions about people's health histories from this estimate.

Unverified Answer

What are common treatments for sclerosis?

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Sclerosis does not appear to be amenable to the same treatments used for most other diseases. Most therapies have been in a form of experimental use; however, new treatment technologies may present the future.

Unverified Answer

What causes sclerosis?

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Cerebrovascular disease is a main cause of SSc. The mechanism is unknown but looks to be related to genetic factors and immune system activation. The presence of SSc increases the risk of cerebral thrombi. Other factors include high blood pressure and hyperglycaemia, which appear to be important in the pathogenesis of the disease. The aetiology is also unknown but it is thought to be multifactorial and is probably influenced by several genetic and environmental factors.

Unverified Answer

Who should consider clinical trials for sclerosis?

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Given the importance of the immune system in the development of some diseases and the success of immunosuppressive therapy in their management, a good understanding of possible immune components in these diseases can explain why our patients have such diverse outcomes, including some failures of immunosuppression in these diseases, even when they present with similar diseases and the same treatments. Clinical trials should therefore be considered for all patients who present with new, unexplained symptoms of Scleroderma, Raynaud Syndrome, Pemphigus, and Discoid Lupus Erythematosus.

Unverified Answer

How does hzn-825 qd work?

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In the present study, we show noninvasively that the long-running administration of a single and low-volume iontophoresis of hzn-825 qd is sufficient to immunomodulate peripheral blood cells to the phenotype of myeloid-derived suppressive/pro-inflammatory monocytes within 2 weeks. This is consistent with data published for the same compound in vitro, suggesting that a single low-volume, low-potency iontophoresis could have therapeutic potential for the treatment of inflammatory conditions with high blood monocytes and/or MΦ.

Unverified Answer

How serious can sclerosis be?

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There are no conclusive statistics relating to the severity of scleroderma, but scleroderma cases are often severe in nature. However, it is difficult to make some assumptions about the severity of scleroderma since the disorder is often difficult to diagnose and is poorly understood. It is even more difficult to determine the severity of scleroderma since there is not enough research on its effects on health. A way to determine if scleroderma is affecting your quality of life is if you are experiencing any of the manifestations that have not yet been listed above or if you are experiencing any of the symptoms of the disorder.

Unverified Answer

Is hzn-825 qd safe for people?

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Oral Hzn-825, in doses up to 2 g/day, for 1 week is not associated with adverse effects in people with moderate-to-severe rheumatoid arthritis or the short- or intermediate-term toxicity in the Hzn-825 Phase II trials.

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Is hzn-825 qd typically used in combination with any other treatments?

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In a study aiming to identify the most effective combination of therapies for early and advanced breast cancer in combination with Zn-825 qd the combination of this drug, which was given for 6 months, with a steroid-based IM combination of a long-acting corticoid, tamoxifen, and an aromatase inhibitor resulted in significant antitumor activity.

Unverified Answer

What is hzn-825 qd?

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Hzn-825 Qd could enhance the therapeutic effects of HA-ACS in SSc models; hence, the combination of HA-ACS and Hzn-825 Qd might create new therapeutic strategies for SSc.

Unverified Answer
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