Phase I/II – First Administration

453 trials 190 recruiting

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A double-blind, randomised, placebo-controlled, first-in-human (FIH) Phase I/IIa, multi-centre trial to assess safety, tolerability, and imm… Pending

2024-515627-10-00 generalised myasthenia gravis
A PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCUL… Pending

2024-519004-27-00 Duchenne Muscular Dystrophy
A Phase I/IIa study to assess the safety and biological activity of TdT-3, an autologous TCR T-cell therapy targeting TdT, in HLA-A*02:01+ p… Pending

2024-519320-24-00 Acute lymphoblastic leukemia (ALL) or lymphoblastic lym
CAN-GT Pending

2024-519412-13-00 Canavan Disease
A trial in patients with ABCA4 mutation-associated retinal dystrophy. Pending

2024-520425-37-00 Biallelic ABCA4 Mutation-Associated Retinal Dystrophy.
A study to evaluate the safety and tolerability of TTX-381 in participants with ocular manifestations of CLN2 disease Pending

2025-521175-31-00 Ocular manifestations of Neuronal Ceroid Lipofuscinosis
A Study of REGN7041 for Active Noninfectious Uveitis in Adult Participants Pending

2025-521713-57-00 Noninfectious Uveitis
An Open-label, Multicenter Trial to Assess Safety and Efficacy of a Subretinal Administration of AAVB-039 Pending

2025-522207-15-01 Stargardt Disease (STGD1)
A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults with Hypophosphatasia in … Pending

2025-522378-36-00 Hypophosphatasia
Study of ASP2957 in Male Patients with X-linked Myotubular Myopathy Who Need Ventilators Pending

2025-523213-29-00 X-linked myotubular myopathy
A clinical trial to learn more about the effects of DJI136 in people with extensive-stage small cell lung cancer Pending

2025-523276-23-00 Extensive stage small cell lung cancer
A Phase 1/2, Open‑Label, Multi‑Center, Dose Escalation, Dose Expansion, and Single Repeat Dose Study of TSRA‑196 in Adults With the PiZZ Gen… Pending

2025-523497-16-00 Lung and/or Liver Disease Associated with Alpha-1 Antit
Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome Pending

2025-523835-20-00 Dravet Syndrome
Study QEL-005-CLN-01. Phase I/II study of QEL-005 in patients with diffuse cutaneous systemic sclerosis (dcSSc) and in patients with difficu… Pending

2025-523971-46-00 Diffuse cutaneous systemic sclerosis (dcSSc) Difficult
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants with Late-Onset Pompe Disease. Pending

2025-524082-25-00 Late-Onset Pompe Disease
A study of SGX-001 in patients with chronic granulomatous disease caused by p47phox deficiency (p47-CGD) Pending

2025-524423-50-00 Chronic granulomatous disease (CGD) caused by p47phox d
An Open Label, Single Arm, Phase I/II Clinical Study of Autologous CD4+ T-Cells Edited Ex-Vivo at the CD40LG Locus by CRISPR/Cas9 and IDLV-b… Pending

2025-524635-39-00 X-linked immunodeficiency with hyper-IgM type 1 (HIGM1)
A Phase 1/2 Study of BMS-986525 with and without Nivolumab in Relapsed/Refractory Small Cell Lung Cancer Recruiting

2025-524090-16-00 Relapsed/Refractory Small Cell Lung Cancer 2026-06-24
A trial to learn how safe AZD9750 is and how well it works in people with metastatic prostate cancer when given with or without other antica… Recruiting

2024-516976-14-00 Metastatic Prostate Cancer 2026-06-12
A Phase 1/2 Study of the Safety and Tolerability of ICM VTx-002 in participants with ALS Recruiting

2025-522697-37-00 Amyotrophic Lateral Sclerosis 2026-06-05
Study of GVV858 as a single agent or in combination with endocrine therapy in patients with HR+/HER2- breast cancer and other advanced solid… Recruiting

2025-521911-38-00 Advanced HR+/HER- breast cancer 2026-05-26
A clinical study to Evaluate the Safety, Tolerability and Preliminary Efficacy of SB-007 in Subjects with Stargardt Disease. Recruiting

2024-519535-42-00 Stargardt Disease 2026-05-26
A Study of the Safety, Tolerability, PK, PD, and Efficacy of CRMA-1001 in Chronic Hepatitis B Recruiting

2025-523619-12-00 Chronic Hepatitis B 2026-05-22
A Study to Investigate the Safety and Effects of LTX-002 Administered in the Spinal Canal of Participants with Amyotrophic Lateral Sclerosis Recruiting

2025-522039-33-00 Amyotrophic Lateral Sclerosis 2026-04-09