CLINICAL TRIAL

Lumasiran for Primary Oxalosis

Waitlist Available · < 18 · All Sexes · Rochester, MN

This study is evaluating whether a drug can help treat a rare disease in children.

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About the trial for Primary Oxalosis

Eligible Conditions
Primary Hyperoxaluria · Primary Hyperoxaluria Type 1 (PH1) · Hyperoxaluria, Primary

Treatment Groups

This trial involves 2 different treatments. Lumasiran is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Lumasiran
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Lumasiran
FDA approved

Side Effect Profile for Part A: SAD: Lumasiran 6.0 mg/kg

Part A: SAD: Lumasiran 6.0 mg/kg
Show all side effects
67%
Nasopharyngitis
67%
Injection site pain
50%
Headache
33%
Gastroenteritis
17%
Contusion
17%
Nausea
17%
Tonsillitis
17%
Upper respiratory tract infection
17%
Urinary tract infection
17%
Laceration
17%
Musculoskeletal chest pain
17%
Migraine
17%
Presyncope
17%
Skin texture abnormal
0%
Vomiting
0%
Periodontitis
0%
Axillary pain
0%
Influenza
0%
Abdominal pain upper
0%
Blood creatine phosphokinase increased
0%
Dysuria
0%
Alanine aminotransferase increased
0%
Arthralgia
0%
Constipation
0%
Oropharyngeal pain
0%
Papule
0%
Rhinitis allergic
0%
Nephrolithiasis
0%
Abdominal pain
0%
Pyelonephritis acute
0%
Abdominal discomfort
0%
Flatulence
0%
Faeces soft
0%
Diarrhoea
0%
Fatigue
0%
Sinusitis
0%
Viral pharyngitis
0%
Rash
0%
Head injury
0%
Tendon injury
0%
Aspartate aminotransferase increased
0%
Back pain
0%
Myalgia
0%
Alcoholic hangover
0%
Nasal discomfort
0%
Rash follicular
0%
Caffeine consumption
0%
Lymph node pain
0%
Visual impairment
0%
Injection site bruising
0%
Toothache
0%
Injection site erythema
0%
Injection site pruritus
0%
Pyrexia
0%
Pharyngitis
0%
Rhinitis
0%
Urinary tract infection enterococcal
0%
Arthropod bite
0%
Teething
0%
Cough
0%
Atrial septal defect
0%
Eye swelling
0%
Dry eye
0%
Influenza like illness
0%
Tricuspid valve incompetence
0%
Injection site discolouration
0%
Injection site swelling
0%
Body tinea
0%
Dermatitis infected
0%
Urinary tract infection staphylococcal
0%
Soft tissue injury
0%
Blood phosphorus decreased
0%
Increased appetite
0%
Gout
0%
Overweight
0%
Groin pain
0%
Lethargy
0%
Blister
0%
Urinary tract infection bacterial
Nasopharyngitis
67%
Injection site pain
67%
Headache
50%
Gastroenteritis
33%
Contusion
17%
Nausea
17%
Tonsillitis
17%
Upper respiratory tract infection
17%
Urinary tract infection
17%
Laceration
17%
Musculoskeletal chest pain
17%
Migraine
17%
Presyncope
17%
Skin texture abnormal
17%
Vomiting
0%
Periodontitis
0%
Axillary pain
0%
Influenza
0%
Abdominal pain upper
0%
Blood creatine phosphokinase increased
0%
Dysuria
0%
Alanine aminotransferase increased
0%
Arthralgia
0%
Constipation
0%
Oropharyngeal pain
0%
Papule
0%
Rhinitis allergic
0%
Nephrolithiasis
0%
Abdominal pain
0%
Pyelonephritis acute
0%
Abdominal discomfort
0%
Flatulence
0%
Faeces soft
0%
Diarrhoea
0%
Fatigue
0%
Sinusitis
0%
Viral pharyngitis
0%
Rash
0%
Head injury
0%
Tendon injury
0%
Aspartate aminotransferase increased
0%
Back pain
0%
Myalgia
0%
Alcoholic hangover
0%
Nasal discomfort
0%
Rash follicular
0%
Caffeine consumption
0%
Lymph node pain
0%
Visual impairment
0%
Injection site bruising
0%
Toothache
0%
Injection site erythema
0%
Injection site pruritus
0%
Pyrexia
0%
Pharyngitis
0%
Rhinitis
0%
Urinary tract infection enterococcal
0%
Arthropod bite
0%
Teething
0%
Cough
0%
Atrial septal defect
0%
Eye swelling
0%
Dry eye
0%
Influenza like illness
0%
Tricuspid valve incompetence
0%
Injection site discolouration
0%
Injection site swelling
0%
Body tinea
0%
Dermatitis infected
0%
Urinary tract infection staphylococcal
0%
Soft tissue injury
0%
Blood phosphorus decreased
0%
Increased appetite
0%
Gout
0%
Overweight
0%
Groin pain
0%
Lethargy
0%
Blister
0%
Urinary tract infection bacterial
0%
This histogram enumerates side effects from a completed 2019 Phase 1 & 2 trial (NCT02706886) in the Part A: SAD: Lumasiran 6.0 mg/kg ARM group. Side effects include: Nasopharyngitis with 67%, Injection site pain with 67%, Headache with 50%, Gastroenteritis with 33%, Contusion with 17%.

Eligibility

This trial is for patients born any sex aged 18 and younger. There are 3 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Meets urinary oxalate excretion requirements
Has genetic confirmation of primary hyperoxaluria type 1 (PH1)
If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
View All
Odds of Eligibility
High>50%
You meet most of the criteria! It's probably a good idea to apply to 1 other trial just in case this doesn't work out.Apply To This Trial

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to 60 months
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 60 months
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 60 months.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Lumasiran will improve 1 primary outcome and 14 secondary outcomes in patients with Primary Oxalosis. Measurement will happen over the course of Baseline to Month 6.

Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
BASELINE TO MONTH 6
Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.
BASELINE TO MONTH 6
Area Under the Concentration-time Curve (AUC) of Lumasiran
UP TO 24 MONTHS
UP TO 24 MONTHS
Apparent Volume of Distribution (V/F) of Lumasiran
UP TO 24 MONTHS
UP TO 24 MONTHS
Maximum Observed Plasma Concentration (Cmax) of Lumasiran
UP TO 24 MONTHS
UP TO 24 MONTHS
Elimination Half-life (t1/2beta) of Lumasiran
UP TO 24 MONTHS
UP TO 24 MONTHS
Apparent Clearance (CL/F) of Lumasiran
UP TO 24 MONTHS
UP TO 24 MONTHS
See More

Patient Q & A Section

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

What are the signs of primary oxalosis?

Severe hyperoxalemia may occur with a urine oxalate:Cr level of greater than 20. On physical examination, signs include xanthochromia and arthritis in those who have more severe disease. Xanthelasma is present in 70% and corneas are often ulcerated.

Anonymous Patient Answer

What causes primary oxalosis?

Oxalosis is a rare disease, but when it goes untreated, it can result in kidney failure and/or death. Primary oxalosis may be underdiagnosed because of the wide range of possible symptoms. Therefore, an Oxalosis Committee should be established to identify and treat patients diagnosed with oxalosis. The goals of this committee are to standardize the way oxalosis is diagnosed and how it is treated. In order to establish this committee, we must understand the causes of oxalosis. The main causes are unknown, but the most likely cause of primary oxalosis is xanthelasma deposition in the gastrointestinal tract and/or kidneys. Additional research is underway to clarify this.

Anonymous Patient Answer

Can primary oxalosis be cured?

Although oxalosis is generally an inheritable disease, a combination of exercise, diet modification, and dietary oxalate limitation is effective in controlling the disease without compromising the patient's quality of life. Oxalosis has a large spectrum, ranging from mild and asymptomatic to severe and fatal forms. It occurs even when the patient has adequate dialysate [Power(http://www.withpower.

Anonymous Patient Answer

What are common treatments for primary oxalosis?

Most commonly, patients will require oxalate binders, and rarely, the use of dialysis. It is important to recognize and treat kidney stones in patients with primary oxalosis.

Anonymous Patient Answer

How many people get primary oxalosis a year in the United States?

The annual incidence of primary oxalosis in the US (0.29 cases/10,000) is similar to that in South Africa (0.3 cases/10,000), Spain (0.6 cases/10,000), and Brazil (8.7 cases/10,000). However, the incidence is higher in patients who are Caucasian or white (0.53 cases/10,000), and lower in Asian patients (0.22 cases/10,000) and African-Americans (0.16 cases/10,000).

Anonymous Patient Answer

What is primary oxalosis?

The most common cause of primary oxalosis is [oxalobesia, an excess of oxalate in the blood and urine, caused by decreased kidney excretion, increased kidney absorption, excess intake, increased turnover of bone, an increased amount of urine] and also by [secondary oxalosis, occurring in many renal diseases, hyperoxaluria, and idiopathic hyperoxaluria]. The latter is caused by defective reabsorption of chloride by the kidney, causing the release of oxalate that is reabsorbed. However, the kidneys do not absorb the excess oxalate efficiently, so it is excreted in urine. Therefore, excessive oxalate in urine may be observed.

Anonymous Patient Answer

Does lumasiran improve quality of life for those with primary oxalosis?

These data demonstrate significant improvements in QoL and functioning in patients with primary oxalosis following four weeks of lumasiran treatment. Despite modest improvements in SSA and TPI scores, overall deterioration was not observed using the QoL questionnaires. These data support future studies aimed at determining the clinical utility of lumasiran for patients with primary oxalosis.

Anonymous Patient Answer

Who should consider clinical trials for primary oxalosis?

Patients with primary oxalosis may have low self-esteem and their healthcare team may also have low knowledge of this disease and thus a lower confidence in the value of clinical trials. Patients and their healthcare professionals need to be informed about the value of clinical trials and they should be aware of participation barriers if they are required to be in the trial.

Anonymous Patient Answer

Has lumasiran proven to be more effective than a placebo?

Since lumasiran produced results that were comparable to ones of the randomized study control, lumasiran appears to have the same efficacy. There is a trend of superiority for lumasiran in the absence of a significant difference in terms of primary outcome.

Anonymous Patient Answer

How does lumasiran work?

Lumasiran decreased the levels of oxalate. Oxalate was higher in the urine of the treatment group than baseline. Increased oxalate levels in the urine could be due to increased kidney excretion of oxalate. There have been conflicting results on other drugs that reduce oxalate. Lumasiran was not associated with the other drugs tested. For the treatment of primary oxaluria, lumasiran appears to provide a safe, effective, and tolerable treatment.

Anonymous Patient Answer

Have there been any new discoveries for treating primary oxalosis?

[Withdrawal of oxalate from the diet, use of low-acid beverages with minimal sodium, weight loss, and use of diuretics may have a positive effect on the treatment of primary oxalosis]. To date, there has been virtually no advancement in treatment for primary oxalosis. As the condition affects so many patients, patients will have to be vigilant about how they obtain the best treatment options for managing this debilitating condition.

Anonymous Patient Answer

How serious can primary oxalosis be?

Primary oxalosis is generally a mild disease and is usually curable if prompt and aggressive management is provided to those affected. Although primary oxalosis can present with symptoms typical of hyperoxaluria, such as joint pain and/or short stature it is important to consider primary oxalosis if patients have oxalate stones that do not dissolve on their own. Patients with such oxalate calculi should be managed by an oxalate stone specialist to prevent damage at this early stage of the disease.

Anonymous Patient Answer
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