Genetic Diseases, X-Linked clinical trials
The registry lists 40 studies for Genetic Diseases, X-Linked across more than 10 countries. 8 are recruiting or about to start. 1 of them are mainly about Genetic Diseases, X-Linked (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09. Counting its 15 narrower conditions too, such as Muscular Dystrophy, Duchenne: 1,673 studies, 336 open.
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Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
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Who runs them
AO GENERIUM (3) · Connecta Therapeutics, S.L. (3) · Spark Therapeutics, Inc. (3) · PTC Therapeutics (3) · National Institute of Allergy and Infectious Diseases (NIAID) (2) · Taysha Gene Therapies, Inc. (2) · Leipzig University Medical Center (2) · Capricor Inc. (2)
What is being tested here
Drugs under test in Genetic Diseases, X-Linked studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 3, phase 3 first
- Sep 2027To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome · Phase 3 · Biomed Industries, Inc.
- May 2027Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome · Connecta Therapeutics, S.L.
- Jul 2027Rett Syndrome Registry · International Rett Syndrome Foundation
Who these studies take
Of the 7 Genetic Diseases, X-Linked studies recruiting now, 1 (14%) set no upper age limit and 5 (71%) stop at 75 or younger. 4 (57%) take children under 18. 2 (29%) are for women only and 2 (29%) for men only; 1 (14%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (29%) | 2 (29%) |
| 18 | 4 (57%) | 4 (57%) |
| 30 | 3 (43%) | 3 (43%) |
| 40 | 3 (43%) | 3 (43%) |
| 50 | 3 (43%) | 2 (29%) |
| 65 | 3 (43%) | 2 (29%) |
| 75 | 2 (29%) | 1 (14%) |
| 85 | 2 (29%) | 1 (14%) |
Across all 40 studies, recruiting or not: 57% stop at 75 or younger and 55% at 65; 68% take children; 18% are for women only and 53% for men only; 15% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 40 of 40. search them.
- Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and GranulocytesRecruitingNCT000014051 country
- Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)RecruitingNCT068056953 countries
- Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 SkippingAvailableNCT072507371 country
- Quality of Life in Women With X-linked AdrenoleukodystrophyRecruitingNCT046757491 country
- Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X SyndromeRecruitingNCT07654114Phase 21 country
- Rett Syndrome RegistryRecruitingNCT054323491 country
- Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)RecruitingNCT07608432Phase 31 country
- To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett SyndromeRecruitingNCT06840496Phase 32 countries
- An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC DeficiencyActive, not recruitingNCT06255782Phase 34 countries
- A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type IIActive, not recruitingNCT05208281Phase 2/31 country
- A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular DystrophyActive, not recruitingNCT05126758Phase 31 country
- Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett SyndromeActive, not recruitingNCT07480564Phase 31 country
- A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)Active, not recruitingNCT05606614Phase 32 countries
- A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett SyndromeActive, not recruitingNCT05898620Phase 33 countries
- Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyCompletedNCT03179631Phase 318 countries
- A Trial of Metformin in Individuals With Fragile X Syndrome (Met)CompletedNCT03862950Phase 21 country
- Mackenzie's Mission: The Australian Reproductive Carrier Screening ProjectCompletedNCT04157595Not applicable1 country
- A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular DystrophyCompletedNCT03406780Phase 21 country
- Neural Biomarkers in Fragile X SyndromeCompletedNCT069570541 country
- To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett SyndromeCompletedNCT06849973Phase 2/32 countries
- CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food InteractionCompletedNCT06480968Phase 11 country
- A Trial of Metformin in Individuals With Fragile X SyndromeCompletedNCT03479476Phase 2/32 countries
- A Gene Transfer Study for Hemophilia ACompletedNCT03003533Phase 1/25 countries
- Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMDTerminatedNCT04632940Phase 311 countries
- A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersCompletedNCT06475404Phase 11 country
- SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)CompletedNCT04687007Not applicable1 country
- Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII InhibitorsCompletedNCT03734588Phase 1/21 country
- Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A ParticipantsCompletedNCT038763014 countries
- A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With InhibitorsCompletedNCT03417102Phase 317 countries
- A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without InhibitorsCompletedNCT03417245Phase 319 countries
- Kidney Information Network for Disease Research and EducationTerminatedNCT033216041 country
- A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersCompletedNCT04539340Phase 11 country
- The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable MutationApproved for marketingNCT04337112
- An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyTerminatedNCT02090959Phase 319 countries
- Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT01826487Phase 318 countries
- Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X SyndromeCompletedNCT02642653Phase 41 country
- Genetic Studies of X-linked Lymphoproliferative DiseaseCompletedNCT003594111 country
- Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia BWithdrawnNCT02048111Phase 32 countries
- Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2Status unknownNCT02484560Phase 11 country
- DHA and X-Linked Retinitis PigmentosaCompletedNCT00100230Phase 21 country