Studies
Randomized, Placebo-Controlled, Double-Blind Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients with Hypothalamic Obesity, with an Open-Label Extension
A Phase III, Randomized, Double-blind, Placebo-controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Diamyd® to Preserve Endogenous Beta Cell Function in Adolescents and Adults with Recently Diagnosed Type 1 Diabetes, Carrying the Genet…
The objective of DIAGNODE-3 is to evaluate the efficacy and safety of three intranodal injections of 4 μg of Diamyd compared to placebo, along with oral Vitamin D supplementation, to preserve endogenous beta cell function and influence glycemic parameters in adolescent and adults recently diagnosed with T1D carrying the HLA DR3-DQ2 haplotype.
A Long-term, Open-label Extension Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS)
LUMOS 201-02
This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD). This study will also assess pharmacodynamics and efficacy response to therapy with LUM-201.
Low Dose Antithymocyte Globulin (ATG) to Delay or Prevent Progression to Stage 3 T1D
A multi-center, placebo-controlled, double blind, 2:1 randomized control clinical trial testing low-dose ATG vs. placebo in subjects with a 2 year 50% risk of progression to stage 3 T1D.
Pfizer Registry of Outcomes in Growth hormone RESearch (PROGRES): A multi-country, non-interventional, prospective cohort study among patients with human growth hormone (hGH) treatments under routine clinical care
A T cell phenotype signature driven dose finding study with siplizumab in type 1 diabetes mellitus
This is a multicenter, Phase Ib, open-label, siplizumab dose-finding study in individuals aged 8-45 years with a Type 1 diabetes mellitus (T1DM) diagnosis. within 18 months of V0. Participants will be randomized 1:1:1:1 to one of four possible siplizumab dosing arms. All dosing arms will receive weekly siplizumab doses for a total of 12 weeks. After the completion of treatment, participants will undergo follow-up visits at weeks 12, 24, 36 and 52 which include longitudinal MMTTs. If indicated, participants will enter into long-term safety monitoring for up to an additional 48 weeks. Blood samples for mechanistic analyses will be obtained during the treatment phase and thereafter. Adults aged 18- 45 will be enrolled initially at the study sites.
The primary objective is to identify a safe, metabolically favorable, dosing regimen for siplizumab in patients with type 1 diabetes that induces changes in T cell phenotypes observed with alefacept therapy in new-onset T1DM.
The secondary objectives are to:
1. Assess the safety profile of siplizumab in recently diagnosed T1DM.
2. Assess the effects of siplizumab on residual beta cell function in recently diagnosed T1DM participants.
Afrezza, inhaled insulin, in Pediatric Subjects with Type 1 or Type 2 diabetes
HYDROXYCHLOROQUINE FOR PREVENTION OFABNORMAL GLUCOSE TOLERANCE AND DIABETES ININDIVIDUALS AT-RISK FOR TYPE 1 DIABETES MELLITUS
The study is a 2-arm, double blinded, multicenter, 2:1 randomized, placebo controlled clinical trial. Subjects will receive hydroxychloroquine or placebo and close monitoring for progression of T1D.