Clinical Trials

Research Information

Research is the driving force behind the OHF’s mission, because research provides hope to the thousands of people worldwide living with the effects of hyperoxaluria. OHF-funded research has led to groundbreaking discoveries that have unlocked the secrets of primary hyperoxaluria—providing hope, energizing science, and helping save lives around the world. We encourage people with hyperoxaluria and their families to consider taking part in clinical research trials and the patient registry to help researchers unlock the secrets of hyperoxaluria, and bring us one step closer to a cure.

Patients who volunteer to participate in clinical trials make it possible for new drugs and therapies to be developed. By enrolling in a trial or signing up for a patient registry, you will help researchers unlock the secrets of hyperoxaluria, and bring us one step closer to a cure.

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Clinical Trials 101

This is an exciting time in hyperoxaluria research. Not only are there several clinical trials studying therapies to treat the underlying cause of primary hyperoxaluria, many trials are also exploring ways to treat key symptoms of both primary and enteric (or secondary) hyperoxaluria and improve quality of life. If you are new to clinical research, this section will cover the basics of clinical trials. You will learn how your or your child's safety will be protected, why you might want to participate, and how to find a clinical trial.

Why participate in a clinical trial?

Patients who volunteer to participate in observational studies and clinical research trials make it possible for new drugs and therapies to be developed. From observational studies to clinical trials, you can help researchers unlock the secrets of hyperoxaluria.

By participating in a clinical trial, you may benefit by:

Gaining access to new treatments that are not yet available to the public
Obtaining expert medical care at a leading healthcare facility
Playing an active role in your own healthcare
Helping others by contributing to medical research

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How do clinical trials work?

When you or your child participate in a clinical trial, you will meet with doctors who will talk to you about what to expect. Doctors who are running the clinical trial have strict rules for how the treatments are given. This helps to keep you and your child safe. Before the trial begins, the doctors will discuss all risks with you, and they typically will perform certain tests to understand the health of you or your child.
Clinical trials often test experimental treatments. After you or your child have received these treatments for a certain period of time, the doctors will perform tests again. The results of these tests will be compared to the results from before the treatments are given. This helps doctors understand how effective and safe the treatments are.

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What are “phases” in a clinical trial?

Clinical trials are usually grouped into “phases,” which are stages of testing. Clinical trials in each phase are designed to answer certain questions, while trying to make sure the people taking part are kept as safe as possible. Every new treatment is tested in 3 or more phases of clinical trials before being considered reasonably safe and effective.

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Academic Clinical Trials

An academic clinical trial is a clinical trial that is not funded by a pharmaceutical or biotechnology company for commercial ends, but by public-good agencies to advance medicine.

Phases of a Clinical Trial

INITIAL RESEARCH

Funded by OHF and other organizations looking for treatments for hyperoxaluria

PHASE I

Evaluate safety
Determine safe dosage
Identify side effects

Approximately 70% of drugs move to the next phase

PHASE II

Test effectiveness
Further evaluate safety

Approximately 33% of drugs move to the next phase

PHASE III

Confirm effectiveness
Monitor side effects
Compare to other treatments
Collect information

Approximately 25-30% of drugs move to the next phase

FDA APPROVAL

Treatment is approved and becomes widely available

PHASE IV

Provide additional information after approval, including risks, benefits, and best use

Drug Development Pipeline

The Oxalosis & Hyperoxaluria Foundation facilitates the development of promising new drugs, and the drug development pipeline enables you to track the progress of these potential therapies.

Nearly every hyperoxaluria drug being investigated was made possible because of the OHF’s support and its ongoing work with researchers.

Products

www.alnylam.com

info@alnylam.com

Company: Alnylam

Indication: PH1

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

www.dcrphxc.com

info@dcrphxc.com

Company: Dicerna

Indication: PH1 & PH2

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

www.oxthera.com

info@oxthera.com

Company: OxThera

Indication: PH1

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

www.allena.com

info@allena.com

Company: Allena

Indication: Enteric hyperoxaluria

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

Company: Allena

Indication: Systemic oxalosis

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

Company: Allena

Indication: PH1

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

www.orfanbiotech.com

Company: Orfan, a BridgeBio company

Indication: PH1

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

www.novomebio.com

Company: Novome Biotechnologies

Indication: Secondary Hyperoxaluria

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

www.oxidien.com

Company: Oxidien Pharmaceuticals

Indication: Secondary Hyperoxaluria

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

www.oxidien.com

Company: Oxidien Pharmaceuticals

Indication: Secondary Hyperoxaluria

Discovery

Preclinical

Phase 1

Phase 2

Phase 3

To Patients

Product	Indication	Discovery	Preclinical	Phase 1	Phase 2	Phase 3	To Patients
Lumasiran	PH1
www.alnylam.com info@alnylam.com
Nedosiran	PH1, PH2, PH3
www.dicerna.com/pipeline/clinical-trials medicalinfo@dicerna.com
Oxabact OC5	PH1, PH2, PH3
www.oxthera.com info@oxthera.com
Reloxaliase www.allena.com info@allena.com	Enteric Hyperoxaluria Systemic Oxalosis Primary Hyperoxaluria Pediatric Hyperoxaluria
ORF-229	PH1
www.orfanbiotech.com
NOV-001	Enteric Hyperoxaluria
www.novomebio.com
AA-1	Enteric Hyperoxaluria
www.oxidien.com
OX1	Enteric Hyperoxaluria
www.oxidien.com

Research grants

Application deadline: December 15, 2019 at midnight ET

The OHF seeks to support research that will ultimately lead to new diagnostics, treatments, and a cure for primary hyperoxaluria and related hyperoxaluria conditions.

OHF will award up to $200,000 total cost per project after a competitive process to select the most meritorious projects.

APPLICATION Instructions

Short-term travel grant

Application deadline: Open

The OHF’s short-term travel grant supports postdoctoral and mid-career researchers to receive hands-on training for up to 3 months in advanced host institutions or laboratories.

The ultimate goal is to improve the standards in each recipient’s home institution upon their return, which will help them become leaders in their fields. 

Researchers receive hands-on training in outside laboratories, allowing them to acquire state-of-the-art knowledge of a new technique, methodology, or philosophical approach that would complement their own knowledge and experience and enhance their ability to carry out high-quality research compatible with the mission of the OHF.

Contact

OHF global registry initiative

Application deadline: Open

We are seeking proposals to develop international collaborative patient registries and databases to identify the determinants of the clinical manifestations and prognosis of patients with primary hyperoxaluria.

As part of its wider mission to improve the understanding and treatment of primary hyperoxaluria and related calcium oxalate kidney stone diseases, the Oxalosis & Hyperoxaluria Foundation is interested in receiving proposals from scientists and clinicians that address the issue of the influence of genetic, cultural, and environmental background on the clinical manifestations and prognosis of patients with primary hyperoxaluria. To this end, financial support will be given to international collaborative projects that develop and make use of well-defined patient cohorts covering a wide range of genetic and cultural backgrounds.

Possible questions to be addressed include, but are not limited to:

The influence of urinary oxalate level and other urine parameters on renal outcomes.
The influence of dietary intake on urinary oxalate level and renal outcomes.
Genotype-phenotype correlations within and between primary hyperoxaluria types.

In countries where no such databases exist, the OHF is willing to provide funding to help local clinicians and scientists to organize collections of primary hyperoxaluria patient information into standardized registries and databases and to make this information available for approved research projects that address the issues above. A required element of a successful response to this proposal will be a detailed data-sharing plan for key data elements that are collected under this program.

Proposed timelines can be 1 to 2 years; however, additional funds will be available for registry maintenance to sites that achieve predefined benchmarks (eg, patient enrollment targets) in the first year. Successful proposals will benefit clinicians in treating primary hyperoxaluria patients and provide a framework for the evaluation of existing and potential new therapies.

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Primary hyperoxaluria type 3 grant

Application deadline: Closed

Of the known genetic causes of primary hyperoxaluria, PH type 3 is the most recently identified and now known to be caused by mutations in the HOGA1 gene. Although the causative gene is known we still lack much basic information regarding this disease. Indeed, it is not entirely clear how mutations in this gene cause hyperoxaluria. It is not clear what the natural history is of this disorder or if there are treatment approaches that might be uniquely beneficial for PH3 over PH types 1 and 2.

To fill these gaps, the OHF specifically seeks funding applications to study PH type 3. Applications could be clinical, translational, or basic science in focus. Possible areas of study include, but are not limited to, the mechanism(s) of hyperoxaluria, novel treatment strategies and targets, novel biomarkers, and new information regarding risk factors for stone formation or chronic kidney disease in PH type 3.

OHF will award up to $200,000 total for up to 2 years. Indirect costs must be included in the maximum funding amount.

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Clinical Trials

We need your help.

Research Information

Clinical Trials 101

Academic Clinical Trials