None listed
Conditions
Brief summary
APL-2 is an experimental drug being developed by Apellis Pharmaceuticals Inc for the potential use as a treatment for people with a broad range of blood disorders (including paroxysmal nocturnal hemoglobinuria (PNH)) and certain types of auto-immune diseases). PNH is caused by a small change to the individual’s genes, which results in red blood cells being broken down prematurely. People with PNH typically feel tired and often see some blood in their urine. The condition is unfortunately progressive with sufferers needing increasing medical care, with an average life expectancy of only 10 years after diagnosis. The currently available treatments are insufficient to deal with this complex disease with most patients not fully responding to the treatments. APL-2 works in a different way to the drugs that are currently approved, and has been shown in animal studies to prevent the breakdown of red blood cells and keep them healthy. It is hoped that APL-2 will help improve the quality of life and reduce the severity of the condition for PNH sufferers. In this study, multiple ascending doses of APL 2 will be assessed in healthy volunteers. The assessments of the safety, tolerability, pharmacokinetics, and pharmacodynamics following administration of single doses of APL-2 will guide decisions to further develop the drug.
Interventions
Subjects will be randomly assigned to treatment with either daily subcutaneous injections of APL-2 or daily subcutaneous injections of placebo, for 28 consecutive days. This study will be conducted in 4 sequential cohorts. The first cohort will receive 30 mg of APL-2 (4 subjects) or placebo (1 subject).
The second cohort will receive 90 mg of APL-2 (4 subjects) or placebo (1 subject).
The third cohort will receive 180 mg of APL-2 (4 subjects) or placebo (1 subject).
The fourth cohort will rec
Subjects will be randomly assigned to treatment with either daily subcutaneous injections of APL-2 or daily subcutaneous injections of placebo, for 28 consecutive days. This study will be conducted in 4 sequential cohorts. The first cohort will receive 30 mg of APL-2 (4 subjects) or placebo (1 subject). The second cohort will receive 90 mg of APL-2 (4 subjects) or placebo (1 subject). The third cohort will receive 180 mg of APL-2 (4 subjects) or placebo (1 subject). The fourth cohort will receive 270 mg of APL-2 (4 subjects) or placebo (1 subject). Injections will be administered by a study nurse or physician. A second study nurse or physician will observe and document the injection procedure. An independent monitor will review the documentation and verify that each injection was correctly administered.
Sponsors
Clinical Network Services (CNS) Pty Ltd
Study design
Allocation
Randomised controlled trial
Intervention model
Other
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
Healthy adult male or female subject; body mass index (BMI) greater than or equal to 18.5 and less than or equal to 32.0 (kg/m2); weight greater than or equal to 60.0 kg and less than or equal to 80.0 kg
Exclusion criteria
* Subject is mentally or legally incapacitated or has significant emotional problems; or has a history of: clinically significant medical or psychiatric condition or disease, any illness that might confound the results of the study or pose a risk to the subject by their participation in the study, alcoholism or drug abuse, and/or hypersensitivity or idiosyncratic reaction to compounds related to APL-2 or particular antibiotics. * Subject has a history of chronic infections (eg COPD) or any active infection * Female subjects who are pregnant or lactating. * Use of any prescription and non-prescription medications, herbal remedies, or vitamin supplements within the last 14 days, or use of some particular drugs such as St. John’s Wort within the last 28 days, up until the end of the study (paracetamol may be permitted) * Blood donation or significant blood loss within the past 56 days or plasma donation within the last 7 days. * Participation in another clinical trial within the past 28 days * Significant surgery within the past 90 days * Presence of any scars or tattoos on the abdomen which may obscure the injection site * Subjects who have participated in the Single Ascending Dose study with APL-2
Outcome results
None listed