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22+ recruiting trials across all rare diseases. Filter by phase, location, and condition.

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Showing 20 of 22 recruiting trials for “peroxisomal-disease

NARecruitingNCT07478172

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

🏥 University of Missouri-Columbia📍 1 site📅 Started Mar 2026View details ↗
Phase 1, PHASE2RecruitingNCT07282847

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

🏥 AskBio Inc📍 10 sites📅 Started Feb 2026View details ↗
Enrolling by InvitationNCT07212764

Mobile App-Based Infection Monitoring in Familial Mediterranean Fever

👨‍⚕️ Bengisu Menentoğlu, Istanbul University📍 1 site📅 Started Nov 2025View details ↗
Phase 2RecruitingNCT07123155

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

🏥 Shionogi📍 28 sites📅 Started Oct 2025View details ↗
NAEnrolling by InvitationNCT07013045

Comparing Structured Neuromuscular Exercise and Exergaming Program in Adolescents With Familial Mediterranean Fever

🏥 Istanbul University - Cerrahpasa📍 1 site📅 Started Jun 2025View details ↗
NARecruitingNCT07129538

Outcomes of Inspiratory Muscle Training in FMF Adolescents

👨‍⚕️ Ali MA Ismail, lecturer, Cairo University📍 1 site📅 Started May 2025View details ↗
NARecruitingNCT07130318

Mediterranean Diet in Familial Mediterranean Fever: Is Fatty Liver Affected by Addition of Aerobic Exercise

👨‍⚕️ Ali MA Ismail, lecturer, Cairo University📍 1 site📅 Started May 2025View details ↗
NARecruitingNCT07130305

is There an Effect of Adding Body Vibration to Intake of Vitamin D on Some Outcomes of Familial Mediterranean Fever

👨‍⚕️ Ali MA Ismail, lecturer, Cairo University📍 1 site📅 Started May 2025View details ↗
Phase 4RecruitingNCT06666413

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

👨‍⚕️ Trial Transparency email recommended (Toll free for US & Canada)📍 1 site📅 Started May 2025View details ↗
RecruitingNCT06795152

Rare Glycogen Storage Diseases Natural History Study

👨‍⚕️ Priya Kishnani, M.D., Duke📍 1 site📅 Started Dec 2024View details ↗
RecruitingNCT06338891

Can Gluten/Wheat or Other Foods be Responsible for FMF Attacks

👨‍⚕️ Antonio Carroccio, MD, University of Palermo📍 1 site📅 Started May 2024View details ↗
Phase 1, PHASE2RecruitingNCT06391736

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

👨‍⚕️ Guang Yang, Chinese PLA General Hospital📍 1 site📅 Started Apr 2024View details ↗
RecruitingNCT06121011

A Global Prospective Observational Registry of Patients With Pompe Disease

🏥 Amicus Therapeutics📍 41 sites📅 Started Feb 2024View details ↗
Phase 3RecruitingNCT04808505

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

🏥 Amicus Therapeutics📍 14 sites📅 Started Jul 2023View details ↗
Phase 2RecruitingNCT05190991

Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever

👨‍⚕️ Mikhail Samsonov, R-Pharm📍 8 sites📅 Started Oct 2021View details ↗
RecruitingNCT04478409

Characterization of a Functional Test for Mediterranean Family Fever Screening - 2

🏥 Hospices Civils de Lyon📍 8 sites📅 Started Jul 2021View details ↗
RecruitingNCT03564561

Natural History of Pompe Disease

👨‍⚕️ Helge Amthor, MD, PhD, Hôpital Raymond Poincaré📍 1 site📅 Started Jun 2019View details ↗
RecruitingNCT07077473

Observing the Efficacy and Safety of Different Drugs Used in Real-world Familial Mediterranean Fever (FMF) Cases

🏥 Tongji Hospital📍 1 site📅 Started Jan 2018View details ↗
RecruitingNCT04639336

Cognitive and Neurological Pathologies in Pompe Disease

👨‍⚕️ Priya Kishnani, MD, Duke University📍 1 site📅 Started Aug 2016View details ↗
RecruitingNCT01665326

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

👨‍⚕️ Priya S Kishnani, MD, Duke University📍 1 site📅 Started Sep 2009View details ↗
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