Crick
HomeTrialsGenesDrugs23andMeGraphBlogContact
  • Home
  • Trials
  • Genes
  • Drugs
  • 23andMe
  • Graph
  • Blog
  • Contact
Crick

An open-source clinical intelligence platform. Explore clinical trials, gene networks, and molecular structures using public data sources.

Data Sources

  • ClinicalTrials.gov
  • OpenTargets
  • ClinVar
  • PubChem

Links

  • Contact
  • About
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Orphan Diseases”

66 trials

Filters

Phase
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Status
Recruiting
Active, Not Recruiting
Not Yet Recruiting
Completed
Enrolling by Invitation

Showing 20 of 66 results

Large-scale testing (Phase 3)Study completedNCT00049803
What this trial is testing

Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients

Who this might be right for
Narcolepsy
Orphan Medical 200
Not applicableLooking for participantsNCT02743845
What this trial is testing

Gene Discovery Core, The Manton Center

Who this might be right for
Undiagnosed ConditionsRare DisordersOrphan Diseases
Boston Children's Hospital 10,000
Testing effectiveness (Phase 2)Study completedNCT00781053
What this trial is testing

Open Label Extension (OLE) for the Patients Treated in the ISD002-P144-07 Study

Who this might be right for
Skin Fibrosis
ISDIN 38
Not applicableLooking for participantsNCT06615206
What this trial is testing

A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)

Who this might be right for
MECP2 Duplication Syndrome
HuidaGene Therapeutics Co., Ltd. 6
Not applicableStudy completedNCT06428474
What this trial is testing

Implementation of Oral Health Educational Program on Orphan Children

Who this might be right for
Dental CariesKnowledgePractice
Pharos University in Alexandria 80
Not applicableStudy completedNCT02307214
What this trial is testing

Tako-Tsubo Cardiomyopathy and Cardiac Syndrome X: New Insights Into the Pathophysiology

Who this might be right for
Syndrome XTakotsubo Cardiomyopathy
Ospedale San Giovanni Bellinzona 39
Large-scale testing (Phase 3)Study completedNCT01067222
What this trial is testing

Efficacy and Safety Study of BF2.649 in the Treatment of Excessive Daytime Sleepiness in Narcolepsy

Who this might be right for
NarcolepsyExcessive Daytime SleepinessCataplexy+1 more
Bioprojet 110
Large-scale testing (Phase 3)Looking for participantsNCT04220190
What this trial is testing

RAPA-501 Therapy for ALS

Who this might be right for
Amyotrophic Lateral Sclerosis
Rapa Therapeutics LLC 41
Not applicableStudy completedNCT05818566
What this trial is testing

Orphan Drugs for Inherited Metabolic Diseases

Who this might be right for
Inborn Errors of Metabolism
University of Lausanne 39
Large-scale testing (Phase 3)Study completedNCT01000961
What this trial is testing

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 43
Large-scale testing (Phase 3)Ended earlyNCT03495921
What this trial is testing

A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide

Who this might be right for
Ewing SarcomaEwing Family of TumorsEwing's Tumor Metastatic+10 more
Gradalis, Inc. 32
Testing effectiveness (Phase 2)Study completedNCT00872729
What this trial is testing

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 9
Testing effectiveness (Phase 2)Study completedNCT03679598
What this trial is testing

Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

Who this might be right for
Alpha-1 Antitrypsin Deficiency (AATD)Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I MutationsEmphysema or COPD
University of Alabama at Birmingham 63
Not applicableLooking for participantsNCT02852928
What this trial is testing

European Management Platform for Childhood Interstitial Lung Diseases - chILD-EU Register and Biobank

Who this might be right for
Lung Diseases, InterstitialPulmonary Alveolar ProteinosisPulmonary Eosinophilia+3 more
Matthias Griese 1,000
Large-scale testing (Phase 3)Study completedNCT02523638
What this trial is testing

Study to Assess the Self-administration of AOP2014 Using a Pen, Developed for the Treatment of Polycythemia Vera Patients

Who this might be right for
Polycythemia Vera
AOP Orphan Pharmaceuticals AG 30
Not applicableStudy completedNCT00213811
What this trial is testing

Bardet-Biedl Syndrome Study: Clinical and Genetic Epidemiology Study in Adults

Who this might be right for
Bardet-Biedl SyndromeOrphan Diseases
University Hospital, Strasbourg, France 40
Large-scale testing (Phase 3)Ended earlyNCT02697136
What this trial is testing

CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases

Who this might be right for
Familial Hypoalphalipoproteinemia
Cerenis Therapeutics, SA 30
Not applicableStudy completedNCT01002612
What this trial is testing

Reassessment of the Nutritional Status in Thai Orphans Living With HIV in a Family Style Community

Who this might be right for
HIV InfectionHIV Infections
Thai Red Cross AIDS Research Centre 73
Early research (Phase 1)Study completedNCT06480968
What this trial is testing

CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction

Who this might be right for
Fragile X Syndrome
Connecta Therapeutics, S.L. 76
Not applicableStudy completedNCT03563677
What this trial is testing

Dual Guidance Structure for Evaluation of Patients With Unclear Diagnosis in Centers for Rare Diseases

Who this might be right for
Rare DiseasesOrphan Diseases
Wuerzburg University Hospital 1,379
Load More Results