Primary hyperoxaluria type 1

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ORPHA:93598OMIM:259900E74.8
Who is this for?
Show terms as
2FDA treatments1Active trials18Specialists8Treatment centers2Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Also known as:

Clinical phenotype terms— hover any for plain English:

EnuresisHP:0000805HyperoxaluriaHP:0003159CalcinosisHP:0003761Abnormal circulating enzyme concentrationHP:0011021Decreased glomerular filtration rateHP:0012213AtherosclerosisHP:0002621
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

4 events
Dec 2024Oxalate Excretion Profile in Patients with a Heterozygous Mutation of the AGXT (alanine-glyoxylate Aminotransferase) Gene

Hospices Civils de Lyon — NA

TrialRECRUITING
Aug 2024Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3

Biocodex — PHASE3

TrialNOT YET RECRUITING
Nov 2020Oxlumo: FDA approved

treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients

FDAcompleted
Jul 2019Long Term Extension Study in Patients With Primary Hyperoxaluria

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company — PHASE3

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

2 available

Rivfloza

NEDOSIRAN· Novo Nordisk
RIVFLOZA is indicated to lower urinary oxalate levels in children 2 years of age and older and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function, e.g., eGFR ≥30 m

RIVFLOZA is indicated to lower urinary oxalate levels in children 2 years of age and older and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function, e.g., eGFR ≥30 mL/min/1.73 m2

Oxlumo

LUMASIRAN· Alnylam Pharmaceuticals, Inc.Orphan Drug

treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients

Clinical Trials

1 recruitingView all trials with filters →
Phase 31 trial
Long Term Extension Study in Patients With Primary Hyperoxaluria
Phase 3
Active
PI: Verity Rawson, MB.CHB (Dicerna, A Novo Nordisk Company) · Sites: San Francisco, California; Boston, Massachusetts +18 more

Specialists

18 foundView all specialists →
SF
Sonja Froschauer
Specialist
PI on 1 active trial
BP
Bernd Hoppe, MD PhD
Specialist
PI on 1 active trial
GM
Gesa Schalk, MD
Specialist
PI on 2 active trials1 Primary hyperoxaluria type 1 publication
VR
Verity Rawson
Specialist
PI on 1 active trial
DM
Dawn Milliner, MD
ROCHESTER, MN
Specialist
PI on 2 active trials
VM
Verity Rawson, MB.CHB
San Francisco, California
Specialist

Rare Disease Specialist

PI on 1 active trial
DM
David Goldfarb, MD
Specialist
PI on 4 active trials
JM
John C Lieske, MD
ROCHESTER, MN
Specialist
PI on 2 active trials
JM
John C Lieske, M.D.
ROCHESTER, MN
Specialist
PI on 4 active trials
WM
Wenhao Zhou, PhD, MD
Specialist
PI on 1 active trial1 Primary hyperoxaluria type 1 publication
WP
Winston Yan, MD, PhD
Specialist
PI on 1 active trial
GM
Gesa Schalk, M.D.
Specialist
PI on 1 active trial
VM
Vidar Edvardsson, MD
Specialist
PI on 1 active trial
CM
Carla G Monico, M.D.
JACKSON, MS
Specialist
PI on 1 active trial
DM
Dawn Milliner, M.D.
ROCHESTER, MN
Specialist
PI on 1 active trial
DM
Dawn S. Milliner, M.D.
ROCHESTER, MN
Specialist
PI on 1 active trial
DD
David Sas, DO
ROCHESTER, MN
Specialist
PI on 1 active trial

Treatment Centers

8 centers
⚗️ Trial Site

Clinical Trial Site

📍 Phoenix, Arizona

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

Financial Resources

2 resources
Rivfloza(NEDOSIRAN)Novo Nordisk
Oxlumo(LUMASIRAN)Alnylam Pharmaceuticals, Inc.

Travel Grants

No travel grants are currently matched to Primary hyperoxaluria type 1.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Primary hyperoxaluria type 1

Disease timeline:

New recruiting trial: Oxalate Excretion Profile in Patients with a Heterozygous Mutation of the AGXT (alanine-glyoxylate Aminotransferase) Gene

A new clinical trial is recruiting patients for Primary hyperoxaluria type 1

New trial: Long Term Extension Study in Patients With Primary Hyperoxaluria

Phase PHASE3 trial recruiting. DCR-PHXC

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Primary hyperoxaluria type 1

Are there clinical trials for Primary hyperoxaluria type 1?

Yes — 1 recruiting clinical trial is currently listed for Primary hyperoxaluria type 1 on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Primary hyperoxaluria type 1?

18 specialists and care centers treating Primary hyperoxaluria type 1 are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Primary hyperoxaluria type 1?

2 patient support programs are currently tracked on UniteRare for Primary hyperoxaluria type 1. See the treatments and support programs sections for copay assistance, eligibility, and contact details.