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Clinical Trials

Clinical Trials. J. Jill Hopkins, M.D. Retina Vitreous Associates Los Angeles, CA. OVERVIEW. General introduction to Clinical Trials What they are, what you need to know CNTF Clinical Trials Illustrating how clinical trials bring new treatments to patients Questions and Answers.

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Clinical Trials

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  1. Clinical Trials J. Jill Hopkins, M.D. Retina Vitreous Associates Los Angeles, CA

  2. OVERVIEW • General introduction to Clinical Trials • What they are, what you need to know • CNTF Clinical Trials • Illustrating how clinical trials bring new treatments to patients • Questions and Answers

  3. What is a Clinical Trial? • National Institutes of Health • www.clinicaltrials.gov • Biomedical or health-related research studies in human beings that follow a pre-defined protocol • Interventional or Observational

  4. Clinical Trials • Interventional studies: • research subjects are assigned by the investigator to a treatment (or other intervention) • outcomes are measured

  5. Clinical Trials • Observational studies: • individuals are observed and their outcomes are measured by the investigators

  6. Types of Clinical Trials • Treatment trials • Prevention trials • Diagnostic trials • Screening trials • Quality of Life trials

  7. Treatment Trials • Test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy

  8. Prevention Trials • Investigate ways to prevent a disease in people who have never had it or to prevent a disease from returning • Approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes

  9. Diagnostic Trials • Investigate better tests or procedures for diagnosing a particular disease or condition

  10. Screening Trials • Investigate the best way to detect certain diseases or health conditions

  11. Quality of Life trials • Supportive Care trials • Explore ways to improve comfort/quality of life for individuals with a chronic disease

  12. KEY DEFINITIONS - PROTOCOL • A study plan on which all clinical trials are based • The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions

  13. Protocol • A protocol describes: • what types of people may participate in the trial • the schedule of tests, procedures, medications, and dosages • the length of the study

  14. Protocol • While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment

  15. KEY DEFINITIONS - PLACEBO • an inactive pill, liquid, or powder that has no treatment value. • experimental treatments are often compared with placebos to assess the experimental treatment's effectiveness. • In some studies, participants in the control group will receive placebo instead of active drug or experimental treatment.

  16. What is a control/control group? • A control is the standard by which experimental observations are evaluated. • one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

  17. Important Definitions • Primary Outcome: the main efficacy variable being studied • Visual acuity • Secondary Outcome: additional variables thought to be important • Quality of Life

  18. Phases of Clinical Trials • Phase I - researchers test an experimental drug or treatment • small group of people (10-80) • first time to be evaluated • determine safety, safe dosage range, and identify side effects • Phase II - experimental study drug or treatment given to a larger group of people (100-300) • see if it is effective, further evaluate its safety

  19. Phases of Clinical Trials • Phase III - experimental study drug or treatment given to large groups of people (1,000-3,000) • confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely • Phase IV - post marketing studies delineate additional information including the drug's risks, benefits, and optimal use

  20. Participating in a Clinical Trial • INCLUSION/EXCLUSION CRITERIA: • medical or social standards determining whether a person may or may not be allowed to enter a clinical trial

  21. INCLUSION/EXCLUSION • Criteria based on such factors as age, gender, type and stage of a disease, previous treatment history, and other medical conditions • Criteria not used to reject people personally, but rather to identify appropriate participants and keep them safe

  22. Participating in a Clinical Trial • What is Informed Consent? • the process of learning the key facts about a clinical trial before deciding whether or not to participate • a continuing process throughout the study • doctors and nurses involved in the trial explain the details of the study

  23. Informed Consent • the research team provides an informed consent document that includes details about the study including purpose, duration, required procedures, and key contacts

  24. Informed Consent • Risks and potential benefits are explained in the informed consent document • The participant then decides whether or not to sign the document • Informed consent is not a contract, and the participant may withdraw from the trial at any time

  25. Clinical Trials - Benefits • Clinical trials are an approach for eligible participants to: • Play an active role in their own health care • Gain access to new research treatments before they are widely available • Obtain expert medical care at leading health care facilities during the trial • Help others by contributing to medical research

  26. Clinical Trials - Risks • Risks • May be unpleasant, serious or even life-threatening side effects to experimental treatment • The experimental treatment may not be effective for the participant • The protocol may require more time and attention than a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements

  27. Important Considerations • What is the purpose of the study? • Who is going to be in the study? • Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?

  28. Important Considerations • What kinds of tests and experimental treatments are involved? • How do the possible risks, side effects, and benefits in the study compare with my current treatment? • How might this trial affect my daily life?

  29. Important Considerations • How long will the trial last? • Will hospitalization be required? • Who will pay for the experimental treatment? • Will I be reimbursed for other expenses?

  30. Important Considerations • What type of long-term follow up care is part of this study? • How will I know that the experimental treatment is working? Will results of the trials be provided to me? • Who will be in charge of my care?

  31. Developing Clinical Trials • Repair/Prevent Initial Insult • Gene Therapy • Protect cells from Programmed death • Neuronal Survival Agents • Replace or Bypass Abnormal Structure and Function • Transplantation/Implantation

  32. CNTF Trials in RP and AMDNeuronal Survival Agents RENOIR STUDIES by NEUROTECH

  33. Neuronal Survival Agents • Not cure (restoration) but perhaps stabilization (preservation) over the lifetime of an individual • “If I could just stay where I am” • Can have quite advanced degeneration and still see effects of therapy

  34. Ciliary Neurotrophic Factor • Extensively studied growth factor • Retinal cell rescue capabilities across 13 different animal models of retinal degeneration • Increase in outer nuclear layer cell counts from 1 to greater than 6 layers

  35. FIGURE 3. Retinal • photomicrographs • rats with rhodopsin • mutation S334ter • S334ter untreated eye • NTC-200 parental cell– treated eye • NTC-201 cell–treated eye.

  36. CNTF Device • Surgically implanted • Local anaesthetic/15 minute procedure • Achieves controlled, continuous, sustained delivery of ciliary neurotrophic factor (CNTF

  37. Neurotech CNTF Trials • 2003 – Phase 1A Safety of Device • 5 patients with advanced RP • 2004 – Phase 1B Safety of Therapeutic Doses • 5 patients with advanced RP

  38. Safety Profile • No systemic or ocular complications ensued • Shallow choroidal detachment in one eye receiving the lower-dose device

  39. Efficacy Results • Three of seven study eyes for which acuity could be tracked showed increases of 10–15 letters over baseline • Equivalent to 2–3 lines of conventional Snellen acuity • These increases were maintained 6 months after the implants had been removed

  40. Neurotech CNTF Trials • Phase I Studies – excellent Safety profile • Improvement experienced by some • Phase II/III Studies – Efficacy design • Can CNTF improve: • Visual Acuity, Visual Field in Retinitis Pigmentosa • Visual acuity in Geographic Atrophy ARMD

  41. Retinal Neurotrophic Ocular Implant Research (RENOIR) for ARMD

  42. Inclusion Criteria: • Best-corrected visual acuity in the study eye < 20/63 - > 20/160 (58->39 letter score) as measured using EVA • Presence in the study and/or fellow eye of geographic atrophy (GA) compatible with category 3 or 4 age-related macular degeneration (AMD) • GA must be at least 175 microns in area

  43. Exclusion Criteria: • Participant < 50 years of age • Evidence of classic or occult choroidal neovascularization in either eye, which may include serous RPE detachment, stippling on fluorescein angiogram, macular edema, evidence of hemorrhage and lipid, and disciform scar

  44. CNTF 2 – ARMD Geographic Atrophy • Treatment/Placebo Trial – N=32 • 1 eye randomized to low dose, high dose or placebo • Primary outcome measure – • Visual acuity at 4 months

  45. Retinal Neurotrophic Ocular Implant Research (RENOIR) for RP

  46. CNTF 3 – RP Acuity • Inclusion Criteria: • Participant must be older than 18, but less than 65 years of age • Each eye has a visual acuity score of at least 24 (20/320) and no more than 58 (20/80) letters • Clinical diagnosis must be consistent with retinal degeneration

  47. Exclusion Criteria: • Glaucoma (defined as independent optic atrophy causing vision loss) • Classic syndromic RP • Participant has optic nerve atrophy beyond modest pallor, primary cone-rod dystrophy, unilateral bulls-eye maculopathy, cystoid maculopathy as judged by OCT, or other retinal dystrophy • Lens opacities

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