
A Study to Evaluate the Safety, Tolerability, and Efficacy of BIIB017 (Peginterferon Beta-1a) in...
Multiple SclerosisRelapsing-RemittingThis study will evaluate the safety, tolerability, and descriptive efficacy of BIIB017 in pediatric participants with relapsing-remitting multiple sclerosis (RRMS) and to assess the pharmacokinetics (PK) of BIIB017 in pediatric participants with RRMS in Part 1. In Part 2, the study will evaluate the long-term safety of BIIB017 and further describe safety and the long-term multiple sclerosis (MS) outcomes after BIIB017 treatment in participants who completed the study treatment at Week 96 in Part 1 of the study.

Methylprednisolone During the Switch Between Natalizumab and Fingolimod
Multiple SclerosisProgressive multifocal leukoencephalopathy (PML) is the most feared complication when natalizumab (NTZ) is used in the treatment of relapsing multiple sclerosis (MS). The risk of PML increases after 18 months of treatment. When switching from NTZ to another disease modifying treatment (DMT) in these MS patients with an active disease, there is a high risk of inflammatory reactivation. Nonetheless, a washout period of several weeks is necessary before initiating a new DMT. The primary purpose of this protocol is to investigate the impact of high dose of oral methylprednisolone, given once a month during the washout period between NTZ and Fingolimod (FTY).

Belinostat Therapy With Zidovudine for Adult T-Cell Leukemia-Lymphoma
Adult T-cell Leukemia-LymphomaATLLThe investigators propose to use Belinostat in combination with AZT as consolidation therapy for the treatment of ATLL.

Adderall XR and Cognitive Impairment in MS
Multiple SclerosisThis 12 week randomized placebo-controlled study will compare the effects of 10 mg and 20 mg of a mixed amphetamine salt, extended release medication (trade name Adderall XR) to placebo on objective measures of processing speed and memory, as well as on self-reported measures of cognition and quality of life. To be enrolled in the study, MS subjects must demonstrate impaired processing speed on the Symbol Digit Modalities Test (SDMT).

Treatment of PCNSL With R-IDARAM and Intrathecal Immunochemotherapy
Primary Central Nervous System LymphomaThis study suggests that R-IDARAM combined with intrathecal immunochemotherapy may be high effective in primary central nervous system lymphoma (PCNSL) patients.

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
Severe Combined Immunodeficiency Due to RAG1 DeficiencyThis study is a prospective, non-randomized, open-label, two-centre phase I/II intervention study designed to treat children up to 24 months of age with RAG1-deficient SCID with an indication for allogeneic hematopoietic stem cell transplantation but lacking an HLA-matched donor. The study involves infusion of autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (hereafter called RAG1 LV CD34+ cells) in five patients with RAG1-deficient SCID.

HIV Treatment Adherence Dose Determination Trial
HIV InfectionsThe proposed research will conduct the first dose-determination trial to find the optimal number of behavioral counseling sessions (dose) needed to achieve and sustain optimal HIV treatment adherence. The results of this study will determine how much intervention is needed for whom and at what cost to guide health policy and implementation of behavioral interventions designed to improve durable viral suppression.

Study of Efficacy and Safety of LNP023 in Primary IgA Nephropathy Patients
IgA NephropathyThe study is designed as a multicenter, randomized, double-blind, placebo controlled study to demonstrate the superiority of iptacopan (LNP023) at a dose of 200 mg b.i.d. compared to placebo on top of maximally tolerated ACEi or ARB on reduction of proteinuria and slowing renal disease progression in primary IgA Nephropathy patients.

Allogeneic Transplantation of Expanded Pancreatic Islet Cells
Brittle Type 1 DiabetesThis study will evaluate the efficacy and safety of allogeneic pancreatic islet cells transplantation in patients with "brittle" type 1 diabetes.

Digital Phenotyping and Lifestyle Intervention in Patients With Myasthenia Gravis
Myasthenia GravisMyasthenia Gravis (MG) is a chronic autoimmune neurological disorder where an antibody attack of muscle receptors causes fatigable skeletal muscle weakness. In addition to fatigue, several MG patients experience general fatigue. Small supervised studies during 12 weeks of physical exercise interventions have indicated safety and beneficial neuromuscular outcomes in MG patients. Longer and unsupervised studies are required to obtain guidelines for physical activity in MG patients. Further, the development of smart rings enables remote digital supervision of physical activity, sleep, and biological parameters such as heart frequency, number of steps, and temperature. These parameters could add to the lack of biomarkers in MG. The project design is a randomized controlled trial with a lifestyle intervention to improve fatigue in the autoimmune neuromuscular disease Myasthenia Gravis (MG). The intervention includes digital group counseling regarding physical activity, sleep, general health, and digital follow-up with a "smart ring" (OURA).