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Advanced therapy medicinal products (ATMP) are medicinal products involving cells, gene therapy or tissue engineering. They are used in many different indications, such as immune diseases, Parkinson’s and Alzheimer’s Disease, regenerative medicine (e.g., cartilage defects, wound healing), and cancer immunotherapy. As they are of biological / biotechnology origin, they require special legislation when it comes to market authorization. Therefore, this product class, which covers various new therapeutic strategies, requires special knowledge and experience.
Outline of a clinical trial covering the testing of ATMP in a joint:
The goal of this phase III clinical study was to demonstrate the efficacy of an ATMP in the treatment of cartilage defects in a joint. The efficacy was evaluated by patients’ subjective assessments and physicians’ evaluations of the functional efficacy of the product. The cartilage regenerative effects as well as the safety profile were additionally assessed.
With our structured and supportive approach, our excellent communication skills with investigational sites aided by our acquired experience in trials with ATMP, SCOPE was able to maintain the initial proposed timelines despite many unforeseen challenges.
In addition to general project management and clinical monitoring activities, we provide the following operations in the trial: budget planning and budget management, regulatory affairs, data management, EDC programming, safety management, pharmacovigilance, medical monitoring activities, statistical programming and analysis.
Due to regulatory timelines, only 10 sites were able to recruit during the first six months. Additionally, one country was unable to participate due to regulatory changes during the approval process. SCOPE had to replace seven sites during the recruitment phase of the trial. SCOPE’s success in replacing these sites was due in large part to the strong cooperation with sites of our investigator-network in already active countries. Additionally, a new country with three sites was added. The basis for this decision was founded in strategic knowledge of regulatory requirements for the country and the submission process. Another hurdle successfully handled was the unforeseen protocol update after the initial submission / approval. The initial recruitment goal was successfully met in the first half of the trial recruitment period. This was obtained despite regulatory delays that resulted in only 50% of the sites being active for the first half of the trial.
Despite the many hurdles and difficulties during the study planning and submission, all timelines were kept and all subjects were successfully recruited within the projected timelines. The challenge of the long-term follow-up and close cooperation with the sites is part of our current service.
SCOPE has experience in conducting clinical studies dealing with ATMPs and can therefore provide the specific knowledge necessary for navigating such a study.
General challenges for studies dealing with ATMPs
Our experiences / recommendations
e.g. Arthritis-related Pain, Bladder Pain Syndrome, Cancer Pain, Chronic Pain, Headache / Migraine, Lower Back Pain, Neuropathic Pain (diabetic, postherpetic), Postoperative Pain Management, Rheumatic Pain, Acute Postoperative / Procedural Pain
e.g. ART (IVF, ICSI), Contraception, Endometriosis, Fibromyalgia Syndrome (FMS), Obstetrics, Oncology, Pain Management, PCOS, Polymyalgia Rheumatica, Postmenopausal Complaints, Preterm labor, Rheumatoid Arthritis, Vulvar and Vaginal Atrophy, Vulvovaginal Candidiasis
e.g. Asthma (mild, moderate, severe, paediatric), Asthma / COPD Overlap Syndrome (ACOS), Chronic Obstructive Pulmonary Disease (COPD), Community-Acquired Pneumonia (CAP), COVID-19, Respiratory Virus Infection (including paediatric)
e.g. Alzheimer’s Disease, Bipolar Disorder, Depression, Epilepsy (including paediatric), Mania, Multiple Sclerosis, Narcolepsy, Parkinson’s Disease, Sensorineural Disorder
e.g. Community-acquired pneumonia (CAP), COVID-19, GI and Intra-Abdominal Infections, Immune-Modulator to prevent SARS-CoV Complication, Respiratory Virus Infections (including paediatric)
e.g. Cell and Gene Therapies, Early-Phase Clinical Development of ATMPs, Immunotherapy, Personalized Therapies, Regenerative Medicine
e.g. acute myocardial infarction, hypertension, peripheral arterial disease (PAD), pulmonary arterial hypertension (PAH) and stable angina pectoris
e.g. allergy (allergic conjunctivitis/ rhinoconjunctivitis), dermatomyositis, primary immunodeficiency diseases (PIDs) and vaccination (hepatitis B, influenza, rabies, tuberculosis)
e.g. acute porphyria and type 1 diabetes
e.g. hematological malignancy, liver cancer, metastatic breast cancer, metastatic lung tumor, non-Hodgkin lymphoma, prostatic cancer, renal cancer, schwannoma resection, solid tumors, unresectable cholangiocarcinoma and urinary bladder cancer
e.g. caries prophylaxis
e.g. alopecia areata, hypertrophic scars and psoriasis
e.g. acute porphyria, hormone replacement therapies in women, type 1 diabetes and vitamin D deficiency
e.g. acute porphyria, cholecystectomy, colitis ulcerosa, constipation, fat absorption in HIV-infected patients, inflammatory bowel disease (IBD), intra-abdominal infections, irritable bowel syndrome (IBS), liver disease, cholecystectomy and unresectable cholangiocarcinoma
e.g. hepatitis B vaccination, hepatic impairment, liver cancer and liver disease
e.g. Alzheimer’s, asthma, colitis ulcerosa, osteoarthritis, Parkinson’s Disease, polymyalgia rheumatica (PMR), psoriasis and rheumatoid arthritis
e.g. allergic conjunctivitis, keratoconjunctivitis and retinopathy
e.g. acute porphyria, juvenile dermatomyositis, polymyalgia rheumatica (PMR), pulmonary arterial hypertension (PAH) and sudden sensorineural hearing loss (SSNHL)
e.g. cartilage defects, open Gustilo-Anderson grade IIIa/IIIb tibia shaft fractures and osteoarthritis
e.g. acute peripheral vertigo, acute sensorineural hearing loss (ASNHL), rhinoconjunctivitis and sudden sensorineural hearing loss (SSNHL)
e.g. bladder pain syndrome, prostatic cancer, renal cancer, renal dysfunction / failure
and urinary bladder cancer
e.g. hepatitis B, influenza, rabies and tuberculosis
