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NCT04580420RECRUITINGIRB Ready

Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD

(PHYOX7)

Sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
📊
1 of 28 participants interested
4% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.

Who Can Participate

Inclusion Criteria

Four age groups of participants will be enrolled: 1. adults and adolescents (aged ≥ 12 years) 2. children 6 to 11 years of age 3. children 2 to 5 years of age 4. infants and newborns from birth to \< 2 years of age 2. . Documented diagnosis of PH1, confirmed by genotyping 3. Estimated GFR at Screening \<30mL/min normalized to 1.73m\^2 BSA 4. Mean of 2 Plasma Oxalate \>20μmol/L during screening 5. For participants receiving hemodialysis or peritoneal dialysis total duration of hemodialysis or peritoneal dialysis must be less than 24 months and hemodialysis or peritoneal dialysis regimen must have been stable for at least 2 weeks prior to Screening. 6. Male or Female 1. Male participants:
A male participant with a female partner of childbearing potential must agree to use contraception during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period. 2. F

❓ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

28 participants

Gender

ALL

🏢

Sponsor

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Study Locations

Choose your preferred location or select flexible during enrollment

ACTIVE_NOT_RECRUITING

San Francisco

California

Location available
ACTIVE_NOT_RECRUITING

Boston

Massachusetts

Location available
RECRUITING

Rochester

Minnesota

Location available
ACTIVE_NOT_RECRUITING

New York

New York

Location available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

🔒 Secure · 🏥 Expert Care

Looking for Primary Hyperoxaluria Type 1 Treatment in San Francisco?

Join others in California exploring innovative treatment options through clinical research

Primary Hyperoxaluria Type 1 Treatment Options in San Francisco, California

If you're searching for Primary Hyperoxaluria Type 1 treatment in San Francisco, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in San Francisco, Boston, Rochester and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Primary Hyperoxaluria Type 1. All study-related care is provided at no cost to participants.

Local Sites
3 locations in California
Now Enrolling
Up to 28 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Primary Hyperoxaluria Type 1?

Potential Benefits

  • Access to new treatment approaches before public availability
  • Close monitoring by experienced medical professionals
  • Study-related care provided at no cost
  • Contribute to medical research for Primary Hyperoxaluria Type 1

What to Expect

  • Initial screening to determine eligibility
  • Regular check-ups and monitoring visits
  • Possible compensation for time and travel
  • You can withdraw at any time

Frequently Asked Questions About This Primary Hyperoxaluria Type 1 Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT04580420. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.