Frequently Asked Questions
About Clinical Trials
1. What is a clinical trial?
- A research study that explores whether a medical strategy, investigational treatment, or device is safe and effective for humans.
2. What are the phases of clinical development?
- All potential new treatments go through a set of clinical trial phases under controlled conditions.
- There are 4 phases of clinical research.
- Phase I tests safety in humans by giving the treatment or drug to healthy volunteers.
- Phase II tests safety and effectiveness in patients living with the disease being studied.
- Phase III tests safety and confirms effectiveness in patients with the disease and studies a larger group of people than the Phase II trial.
- Phase IV tests the drug's effect in various populations and any side effects associated with long-term use after it is approved.
- When these phases are complete, regulatory agencies – for example, U.S. Food & Drug Administration (FDA) or the European Medicines Agency (EMA) – determine whether the treatment can be prescribed by physicians to patients.
3. Why should I participate in a clinical trial?
People choose whether or not to participate in clinical trials for various reasons, including:
- Gaining early access to treatments being studied for safety and effectiveness
- Receiving expert medical care
- Contributing to medical research
- Potentially helping future generations of people
Talk to your doctor if you are interested in participating in a clinical trial.
4. How is a rare disease clinical trial different than larger clinical trials?
- The main difference between rare disease clinical trials and clinical trials in larger patient populations, such as diabetes is that the population for the specific rare disease is much smaller.
- There are many implications due to a smaller patient population
- Generally, these trials are small in scope
- They tend to move more quickly through the development stages
- There is a higher dependence on a larger percentage of patients to participate
- It’s important for as many patients as possible to participate due to the low number of patients in the entire population.
5. How are study participants protected? What is informed consent?
- An independent group called an Institutional Review Board (IRB) in the United States or Independent Ethics Committee (IEC) in Europe protects the rights and welfare of people who participate in clinical trials.
- The IRB/EC monitors the ethical aspects, progress, and results of the clinical trial from start to finish.
- The regulatory agency (either the Food and Drug Administration, FDA in the U.S., or individual country health agencies in the EU) are also required to review and approve of how a clinical trial is designed before it begins.
6. What is informed consent?
- Informed consent is when the patient/caregiver/legal guardian is provided clear, easy-to-understand information as he/she is deciding whether to participate in a clinical trial.
- Both the Institutional Review Board (IRB) and International Ethics Committee (IEC) and the regulatory agency ensure that each clinical trial participant receives and understands the informed consent in order to make the most informed decision.
1. What is DCR-PHXC?
- DCR-PHXC utilizes Dicerna’s GalXC™ RNAi technology to develop products.
- The GalXC RNAi technology harnesses the body’s natural biological pathways to silence or “turn off” disease-driving genes with a high degree of selectivity and specificity.
2. What is RNAi?
- Ribonucleic acid interference (RNAi) is a biological process designed to “stop” or “turn off” destructive diseases by “silencing” the genes underlying these conditions.
- RNAi is not gene therapy and does not result in permanent alteration of genes. When the drug is no longer available, the disease returns to its native state.
3. How is an investigational therapy delivered to the liver using RNAi?
- Using RNAi, investigational therapies need a delivery system to transport the medicine to the diseased cells, thus silencing the disease-driving genes.
- Dicerna attaches a specialized type of sugar molecule to the investigational therapy and uses the sugar molecule as the delivery system to send the intended therapy to the correct target.
- Once the therapy makes it to the correct target, then the therapy silences the disease-driving gene by turning it off.
4. What is subcutaneous delivery?
- Subcutaneous means “under the skin.” So, a therapy that is delivered subcutaneously is delivered with a needle under the skin before it enters the bloodstream.
ABOUT THE PHYOX TRIAL
1. What is the PHYOX Clinical Trial?
- The PHYOX clinical trial of DCR-PHXC is a Phase I, single-ascending dose study of DCR-PHXC in patients with primary hyperoxaluria (PH) types 1 (PH1) and 2 (PH2).
- A single-ascending dose study is when a single dose of a treatment is given to a small group of participants. If the dose is well-tolerated, researchers may raise the dose in order to determine the highest safe dose.
2. What is the purpose of this trial?
The primary objective for the trial:
- Evaluate the safety and tolerability of single doses of DCR-PHXC
Secondary objectives include:
- Evaluate the pharmacodynamic (PD) effects of single doses of DCR-PHXC on biochemical markers including, but not limited to, changes in urine oxalate concentrations
- Characterize the pharmacokinetics (PK) of single doses of DCR-PHXC in patients with PH
Pharmacodynamics is the study of how a drug affects the body, whereas pharmacokinetics is the study of how drugs move within the body.
3. What is the study design?
The study is an open-label, multi-center study in patients with PH1 and PH2.
- Open-label means that both the researcher and the participant know that the treatment is being given. There is no placebo in the Group B study.
4. Who is eligible to participate in this study?
You may be eligible to participate in Group B (patients with primary hyperoxaluria) if you:
- Are male or female, 6 years or older
- Have a minimum body weight of 25 kg
- Are a non-smoker, at least 1-month tobacco free, and willing to remain tobacco free for the entire study
- Are diagnosed with PH1 or PH2
- Meet the 24-hour urine oxalate excretion requirements
5. Are there any reasons a patient may not be allowed to participate in this trial?
There are several reasons that patients with primary hyperoxaluria (PH) might not eligible to participate in Group B of the trial. Examples include (but are not limited to) if a person:
- Is currently receiving dialysis
- Is a female who is pregnant or plans to become pregnant during or within 90 days of the study
- Has participated in a clinical trial where they received an investigational treatment within 3 months before enrollment
- Has liver function test abnormalities
6. If patients are not eligible for the clinical trial, can they still get access to the medication?
- Dicerna, the sponsor of the PHYOX trial, believes that the best way for patients with primary hyperoxaluria (PH) to receive access to investigational treatments before they are approved is for those patients to participate in clinical trials. In some extreme circumstances when this is not possible, patients with life-threatening diseases or conditions may seek special access to investigational medicines outside of a clinical trial setting. These situations are typically referred to as compassionate use, but can also be known as expanded access, early access, pre-approval access, and emergency use.
7. What kinds of side effects might be associated with DCR-PHXC?
- The safety of study participants is a priority for Dicerna, the sponsor of the study. Researchers will monitor for potential side effects of DCR-PHXC and will report serious side effects to the Institutional Review Board (IRB) for further review. Safety is the most important aspect of the clinical trial, and Dicerna will monitor all participants’ safety very closely.
8. What happens if a trial participant has side effects from DCR-PHXC?
- The safety of study participants is a priority for Dicerna, the sponsor of the study. Researchers will monitor for potential side effects of DCR-PHXC and will report any to the Institutional Review Board (IRB) for further review. Safety is the most important aspect of the clinical trial, and Dicerna will monitor all participants’ safety very closely.
9. Is there any cost to participate in this study?
- No, there is no cost to participate in this study.
10. Where can I participate in the trial?
- For a complete list of clinical trial locations for the PHYOX study, please visit our Clinical Trial Sites page.
11. Will transportation and housing related to trial participation be covered?
- Dicerna is dedicated to helping individuals who might live far away from trial sites by covering transportation and lodging expenses. Our reimbursement plans include travel, meals, lodging and stipend based on a set of requirements and financial/medical needs. Contact your study center to learn more about reimbursement and travel agencies.
12. Who is sponsoring the trial?
- The trial is sponsored by Dicerna Pharmaceuticals, Inc. For more information, visit Dicerna’s website.
13. Where can I learn more?
- Learn more about the clinical trial by reaching out to a clinical trial site near you, or by visiting www.clinicaltrials.gov and entering the identifier number NCT03392896 into the search bar.
1. Who is Dicerna?
- Dicerna Pharmaceuticals, Inc. is a biopharmaceutical company focused on the discovery and development of innovative ribonucleic acid interference (RNAi)-based therapeutics for diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases.
- The Company is using its proprietary GalXC™ RNAi technology platform to build a broad product pipeline in these core therapeutic areas.
- DCR-PHXC is Dicerna’s most advanced product, which is being developed for the treatment of all forms of primary hyperoxaluria (PH).
2. Where is Dicerna located?
- Dicerna is headquartered in Cambridge, Massachusetts (USA).
3. Where can I find out more about Dicerna
- To learn more about Dicerna and its pipeline, management team, and recent news, please visit www.dicerna.com.