Phase 1 Mutations Clinical Trials
19 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–19 of 19 trials
Recruiting
Phase 1Phase 2
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Regeneron Pharmaceuticals30 enrolled16 locationsNCT05788536
Recruiting
Phase 1Phase 2
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Precision BioSciences, Inc.18 enrolled1 locationNCT07429240
Recruiting
Phase 1
ORIC-114 in Combination With Subcutaneous Amivantamab in Patients With EGFR Exon20 Insertion Mutant NSCLC
Solid TumorsNSCLCEGFR Exon 20 Insertion Mutations+1 more
ORIC Pharmaceuticals76 enrolled4 locationsNCT06816992
Recruiting
Phase 1Phase 2
A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Shanghai Euhearing Therapeutics Co., Ltd30 enrolled1 locationNCT07288580
Recruiting
Phase 1
Revumenib in Combination With 7+3 + Midostaurin in AML
Acute Myeloid LeukemiaAMLLeukemia+2 more
Richard Stone, MD22 enrolled2 locationsNCT06313437
Recruiting
Phase 1
BY002 IIT Study in R/R Acute Leukemia
Acute LeukemiaKMT2A Rearrangements or NPM1 Mutations Acute Leukemia
The First Affiliated Hospital of Soochow University18 enrolled1 locationNCT07270770
Recruiting
Phase 1Phase 2
A Study Evaluating FMC-376 in Participants With KRAS G12C Mutated Solid Tumors
Colorectal, CancerPancreatic CancerMetastatic Solid Tumor+5 more
Frontier Medicines Corporation403 enrolled26 locationsNCT06244771
Not Yet Recruiting
Phase 1
A Study of the Safety, Pharmacokinetics and Exploratory Efficacy of RC220 in Combination with Osimertinib in Adult Patients with Non- Small Cell Lung Cancer.
Non-Small Cell Lung Cancer (NSCLC) with activating Epidermal Growth Factor Receptor (EGFR) mutations
Racura Oncology Ltd.40 enrolled1 locationACTRN12626000325303
Recruiting
Phase 1
A Study of HRS-2329 in Participants With Advanced Solid Tumors Harboring RAS Mutations or Amplifications
Advanced Solid Tumors Harboring RAS Mutations or Amplifications
Jiangsu HengRui Medicine Co., Ltd.120 enrolled1 locationNCT07189949
Recruiting
Phase 1Phase 2
A Multicenter Phase 1b/2 Study of Adagrasib, Cetuximab, and Cemiplimab for Metastatic Colorectal Cancer Harboring KRAS G12C Mutations
Metastatic Colorectal CancerKRAS G12C Mutations
M.D. Anderson Cancer Center31 enrolled2 locationsNCT06412198
Not Yet Recruiting
Phase 1
A study to assess the safety of increasing single doses of AP003 in healthy adults.
Liver-associated inborn errors of metabolismArg-TGA premature termination codon mutationsLiver stop codon disease+1 more
Alltrna, Incorporated30 enrolled1 locationACTRN12626000194369
Recruiting
Phase 1
Dose Escalation and Expansion Study to Evaluate the Safety, PK, PD and Efficacy of ZE46-0134 in Adults With FLT3 Mutated or Spliceosome Mutated Relapsed or Refractory Acute Myeloid Leukemia
AML With Gene Mutations
Lomond Therapeutics Holdings, Inc.150 enrolled23 locationsNCT06366789
Recruiting
Phase 1Phase 2
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
Retinal Dystrophy Due to Biallelic ABCA4 MutationsStargardt Disease 1
VeonGen Therapeutics GmbH15 enrolled1 locationNCT07002398
Recruiting
Phase 1Phase 2
A Study of GFH375 in Patients With Advanced Solid Tumors With KRAS G12D Mutations
Advanced Solid TumorsKRAS G12D Mutations
Genfleet Therapeutics (Shanghai) Inc.407 enrolled20 locationsNCT06500676
Recruiting
Phase 1
A Phase I Study of HRS-7172 in Participants With Advanced Solid Tumors Harboring RAS Mutations or Amplifications
Advanced Solid Tumors Harboring RAS Mutations or Amplifications
Jiangsu HengRui Medicine Co., Ltd.120 enrolled1 locationNCT07142980
Recruiting
Phase 1
Phase I Study of HMPL-306 for the Treatment of Gliomas With IDH1 and/or IDH2 Mutations
Gliomas Harboring IDH1 and/or IDH2 Mutations
Hutchmed52 enrolled1 locationNCT07025018
Recruiting
Phase 1Phase 2
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Leber Congenital AmaurosisInherited Retinal Diseases Caused by RPE65 Mutations
HuidaGene Therapeutics Co., Ltd.20 enrolled3 locationsNCT05906953
Recruiting
Phase 1Phase 2
GH55 for Advanced Cancers With MAPK Mutations: A Study on Safety and Early Results
Advanced Solid Tumors With MAPK Signal Pathway Mutations
Suzhou Genhouse Bio Co., Ltd.110 enrolled1 locationNCT06310382
Recruiting
Phase 1Phase 2
Phase 1b/2 Study to Evaluate ABT-101 in Solid Tumor and NSCLC Patients
Advanced Solid TumorNon-small Cell Lung CancerHER2 Mutations
Anbogen Therapeutics, Inc.61 enrolled5 locationsNCT05532696