SEK 464.60
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SEK · as of 2026-08-18 · marketstack
Clinical trials
catalyst calendar across sponsors →Studies where SOBI.ST is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
57 interventional · 22 observational · 34 with posted results
government filings 180d: none disclosed
| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country StudyNCT07746895Achieving urine protein-to-creatinine ratio (UPCR) of <1 g/gOpen-label · Treatment | Phase 4 | Recruiting | Drug: Pegcetacoplan | C3 Glomerulopathy (C3G), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) | 150 | 2031-12-31 | in 1,959 d | 2026-08-05 |
| A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)NCT05776472observationalChange in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months | — | Recruiting | Drug: Pegcetacoplan | Paroxysmal Nocturnal Hemoglobinuria | 200 | 2029-08-15 | in 1,091 d | 2026-07-01 |
| Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog AlfaNCT06940830Annualized joint bleeding rate (AjBR) over the prospective observation period.Open-label · Treatment | Phase 4 | Recruiting | Drug: Efanesoctocog alfa | Hemophilia A | 250 | Dec 2028 | ≤ 864 d | 2026-06-30 |
| Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog AlfaNCT06716814Number of injections to treat a bleeding episodeOpen-label · Treatment | Phase 3 | Recruiting | Drug: Efanesoctocog alfa | Haemophilia A (Moderate or Severe) | 104 | Mar 2027 | ≤ 223 d | 2026-06-26 |
| Pacritinib Effectiveness in Real-world SettingsNCT07774455observational≥50% spleen length reduction | — | Not yet recruiting | Other: Not applicable- observational study | Myelofibrosis (MF) | 60 | 2027-01-27 | in 160 d | 2026-08-19 |
| A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)NCT06782373Overall Clinical Response (OCR), defined as achieving Clinical Response or better at any time during the double-blind treatment period.Randomized · Triple-masked · Treatment | Phase 2 | Active, not recruiting | Drug: Pacritinib, Placebo | VEXAS, VEXAS Syndrome | 78 | 2026-12-30 | in 132 d | 2026-08-19 |
| A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa ProphylaxisNCT06752850Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovial hypertrophy domain score decrease.Open-label · Treatment | Phase 4 | Active, not recruiting | Drug: Efanesoctocog alfa | Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia, Classic Hemophilia, Synovial Hypertrophy | 37 | 2026-11-17 | in 89 d | 2026-04-30 |
| A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia MyelofibrosisNCT03165734Spleen volumeRandomized · Single-masked · Treatment | Phase 3 | Active, not recruiting | Drug: Pacritinib, Physician's Choice medications | Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis | 407 | 2026-10-05 | in 46 d | 2026-04-30 |
| A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint HealthNCT04293523observationalJoint health: Target joint development | — | Active, not recruiting | Drug: ELOCTA | Hemophilia A | 427 | Jul 2026 | — | 2026-03-19 |
| A Study of Anakinra in Japanese Patients With Still's Disease (SJIA and AOSD)NCT05814159An improvement of ≥ 30% from baseline in physician global assessment of disease activity (visual analogue scale [VAS]).Randomized · Quadruple-masked · Treatment | Phase 3 | Active, not recruiting | Drug: Anakinra, Placebo | Still's Disease, Juvenile Onset, Still's Disease, Adult-Onset | 15 | 2025-12-10 | — | 2025-03-05 |
| An Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Systemic Juvenile Idiopathic Arthritis (SJIA) and Adult-Onset Still's Disease (AOSD)NCT07706868ACR30 response at Week 4 with absence of fever attributable to the disease during the 7 days preceding Week 4 visit.Open-label · TreatmentStudy Protocol ↗ | Phase 4 | Withdrawnsponsor's stated reason: Business decision | Drug: Anakinra (Kineret®) | Systemic Juvenile Idiopathic Arthritis (sJIA), Adult Onset Still's Disease (AOSD) | 0 | 2028-09-30 | in 772 d | 2026-07-16 |
| A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMFNCT06666335Change from baseline in the number of FMF attacks per month.Open-label · Treatment | Phase 4 | Withdrawnsponsor's stated reason: Business decision. | Drug: Anakinra | Familial Mediterranean Fever (FMF ) | 0 | 2027-08-30 | in 375 d | 2026-06-29 |
| A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 PatientsNCT06227429observationalOccurrence of hepatic, renal or hematological adverse events (AEs) or death | — | Withdrawnsponsor's stated reason: No patients were prescribed to the study treatment due to the generic treatment being available. | Drug: Nitisinone | Hereditary Tyrosinemia, Type I | 0 | 2027-06-30 | in 314 d | 2025-12-23 |
| A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog AlfaNCT05817812Change from baseline in International Physical Activity Questionnaire (IPAQ) scoringOpen-label · Treatment | Phase 3 | Completed | Drug: Efanesoctocog alfa | Hemophilia A, Severe | 93 | 2026-06-02actual | — | 2026-07-02 |
| Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia ANCT06579144Half-life (t½) of efanesoctocog alfa, damactocog alfa pegol and turoctocog alfa pegol after a single i.v. injectionNon-randomized · Open-label · Other | Phase 1 | Completed | Drug: Efanesoctocog alfa | Hemophilia A | 24 | 2026-05-21actual | — | 2026-06-25 |
| Assessment of Macrophage Activation syndromE in STill's Disease in ItalyNCT06992505observationalOverall survival | — | Completed | — | Macrophage Activation Syndrome (MAS), Still Disease, Juvenile Onset, Still's Disease, Adult-Onset | 32 | 2025-11-27actual | — | 2025-12-02 |
| An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITPNCT04943042observationalCumulative number of weeks with a platelet count ≥30×109/L during Doptelet treatment. | — | Completed | Drug: Avatrombopag | Immune Thrombocytopenia, ITP | 199 | 2025-06-10actual | — | 2025-07-14 |
| A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese PatientsNCT05744063results2026-03-13Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse EventOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 4 | Completed | Drug: Emapalumab-Lzsg 5 MG/ML [Gamifant] | Primary Hemophagocytic Lymphohistiocytosis | 13 | 2025-02-21actual | — | 2026-03-13 |
| A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell TransplantationNCT05148299results2025-11-28Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Pegcetacoplan | Transplant-Associated Thrombotic Microangiopathy | 12 | 2024-12-08actual | — | 2025-11-28 |
| Assessment of Macrophage Activation syndromE in STill's DiseaseNCT06405152observationalOverall survival | — | Completed | Other: Chart review | Macrophage Activation Syndrome, Still's Disease, Adult-Onset, Stills Disease, Juvenile-Onset | 75 | 2024-09-26actual | — | 2024-10-21 |
| Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLENCT05001737results2026-02-19Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of EmapalumabOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Emapalumab | Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS | 33 | 2024-06-30actual | — | 2026-02-19 |
| A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)NCT05096403results2025-10-30Number of Patients Achieving a Response (R) at Week 24Randomized · Quadruple-masked · TreatmentStudy Protocol: Clinical Study Protocol v2.0 ↗ · Study Protocol: Clinical Study Protocol v4.0 ↗ · Study Protocol: Clinical Study Protocol v3.0 ↗ · Study Protocol: Clinical Study Protocol v5.0 ↗ · Study Protocol: Clinical Study Protocol v1.0 ↗ · Statistical Analysis Plan: Statistical Analysis Plan v2.0 ↗ · Statistical Analysis Plan: Statistical Analysis Plan v1.0 ↗ | Phase 3 | Completed | Drug: Pegcetacoplan, Placebo matching Pegcetacoplan | Cold Agglutinin Disease | 24 | 2024-05-27actual | — | 2025-10-30 |
| An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia BNCT03901755observationalAnnualised bleeding rate (ABR) | — | Completed | Drug: Alprolix | Hemophilia B | 151 | 2024-03-12actual | — | 2025-09-29 |
| An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog AlfaNCT05856266results2025-01-16Change From Baseline in Total HEAD-US Score up to Month 18 (End of Study - EOS)Open-label · OtherStudy Protocol and Statistical Analysis Plan ↗ | Phase 4 | Terminatedsponsor's stated reason: The Sponsor has decided to stop the phase 4 study Sobi.HAEM89-007 (JOIN-us) due to a realignment of its development activities. The clinical trial hasn't been terminated due to a change in the benefit-risk balance. | Procedure: Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) · Other: Haemophilia Joint Health Score (HJHS) | Hemophilia A, Hemophilia B | 10 | 2023-12-06actual | — | 2025-01-16 |
| A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy IINCT04596540results2025-12-03Proportion of Participants Who Achieved and Maintained Reduction in Serum Uric Acid (sUA) < 6 Milligrams Per Deciliter (mg/dL) for At Least 80% of The Time During Treatment Period 6 (Month 6)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: SEL-212 low-dose, SEL-212 high-dose · Other: Placebo | Chronic Gout | 153 | 2023-01-10actual | — | 2025-12-03 |
| rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia ANCT03951103observationalITI with rFVIIIFc: Main dose | — | Completed | Other: rFVIIIFc | Hemophilia A With Inhibitor | 44 | 2022-09-30actual | — | 2024-09-19 |
| A Clinical Study to Investigate Interferon Gamma (IFNɣ) Signature in Patients Post HSCT and in Patients With Impaired HSC Proliferation Pre-transplantNCT04494061observationalHSCT cohort: IFNγ signature pre-and post-transplant | — | Terminatedsponsor's stated reason: Decision related to Business Priorities, Assessment of Development Options | Procedure: blood collection | Graft Failure | 101 | 2022-08-31actual | — | 2022-10-21 |
| A Study of SEL-212 in Patients With Gout Refractory to Conventional TherapyNCT04513366results2025-09-11Proportion of Participants Who Achieved and Maintained Reduction in Serum Uric Acid (sUA) < 6 Milligrams Per Deciliter (mg/dL) for At Least 80% of The Time During Treatment Period 6 (Month 6)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: SEL-212 low-dose, SEL-212 high-dose · Other: Normal Saline | Chronic Gout | 112 | 2022-07-21actual | — | 2025-09-11 |
| Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST)NCT04731701observationalPsychometric validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST). | — | Completed | Diagnostic test: Hemophilia Joint Health Score (HJHS) | Hemophilia A, Hemophilia B | 180 | 2022-07-19actual | — | 2024-09-19 |
| Impact on French Physician's Haemophilia Treatment Management Decision Based on Systematic Joint ExaminationNCT04133883Changes in haemophilia management based on systematic joint examinations of ankles, knees and elbows with HJHS and HEAD-USOpen-label · Health services research | Not applicable | Completed | Procedure: Ultrasound, Haemophilia Joint Health Score | Hemophilia A | 88 | 2022-07-13actual | — | 2024-09-19 |
| Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT.NCT04731298results2023-12-28CXCL9 in SerumOpen-label · PreventionStudy Protocol and Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Sponsor Decision related to Business Priorities, Assessment of Development Options | Drug: Emapalumab | Graft Failure | 2 | 2022-04-21actual | — | 2023-12-28 |
| A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or BNCT03055611observationalAnnualised bleeding rate (ABR) | — | Completed | Drug: ELOCTA, ALPROLIX | Haemophilia A, Haemophilia B | 201 | 2022-04-19actual | — | 2024-10-02 |
| A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in FranceNCT03655340observationalAnnualised bleeding rate (ABR) (prophylactic treatment) | — | Completed | Drug: Alprolix | Haemophilia B | 91 | 2022-03-16actual | — | 2024-09-19 |
| Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor ProductsNCT02976753observationalAnnualised bleeding rate (ABR) | — | Completed | Drug: efmoroctocog alfa, Factor VIII | Hemophilia A | 361 | 2021-11-03actual | — | 2024-10-02 |
| Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic LymphohistiocytosisNCT03312751results2024-03-12Overall Response at Week 8 or End of Treatment (if Earlier)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Emapalumab | Primary Hemophagocytic Lymphohistiocytosis | 35 | 2021-08-18actual | — | 2024-03-12 |
| A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLHNCT03985423results2023-10-06Overall ResponseOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Terminatedsponsor's stated reason: Sponsor Decision | Drug: Emapalumab-Lzsg | Hemophagocytic Lymphohistiocytoses | 7 | 2021-06-29actual | — | 2023-10-06 |
| A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.NCT04765553results2023-11-24The Maximum Observed Concentration of EmapalumabRandomized · Single-masked · OtherStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1 | Completed | Drug: NI-0501, Saline | Rare Diseases | 8 | 2021-06-25actual | — | 2023-11-30 |
| A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal AntibodyNCT02069899results2022-06-28Number of Participants With Adverse Event (AE)Non-randomized · Open-label · OtherStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Completed | Drug: Emapalumab | Hemophagocytic Lymphohistiocytosis | 58 | 2021-05-18actual | — | 2022-06-28 |
| A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA PatientsNCT03811028results2022-02-25Safety as Measured by Adverse Events Frequencies (by Type and Severity)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: SOBI003 | Sanfilippo Syndrome Type A (MPS IIIA) | 6 | 2021-04-30actual | — | 2022-02-25 |
| Efficacy and Safety of Emapalumab and Anakinra in Reducing Hyperinflammation and Respiratory Distress in Patients With COVID-19 Infection.NCT04324021results2022-03-10Number of Participants With Treatment SuccessRandomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Terminatedsponsor's stated reason: Standard of care evolved during the timeframe of the study and had critical impact on recruitment. Early termination was not based on safety reasons but due to the reasons mentoined above. The ongoing patients were completed. | Biological: Emapalumab, Anakinra | SARS-CoV-2 | 16 | 2020-11-13actual | — | 2022-03-10 |
| A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)NCT03311854results2022-01-26Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Emapalumab | Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic, Arthritis, Juvenile, Adult Onset Still Disease | 14 | 2020-05-19actual | — | 2022-05-17 |
| Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and BNCT03276130observationalUse of medication for pain, depression and anxiety (yes/no?) | — | Completed | — | Haemophilia A, Haemophilia B | 2,300 | 2020-01-30actual | — | 2024-03-06 |
| Evaluation of IFNγ and Inflammatory Mediators in Patients With Malignancy-Associated Hemophagocytic LymphohistiocytosisNCT03259230results2024-09-19observationalMean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 10 (CXCL10)Study Protocol ↗ · Statistical Analysis Plan ↗ | — | Completed | Other: Blood Draws, Data Collection | Hemophagocytic Lymphohistiocytosis | 40 | 2020-01-29actual | — | 2024-09-19 |
| Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic LymphohistiocytosisNCT06047210results2024-09-19observationalMean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 10 (CXCL10)Study Protocol ↗ · Statistical Analysis Plan ↗ | — | Completed | Other: Blood Draws Data Collection | Hemophagocytic Lymphohistiocytoses | 14 | 2019-10-30actual | — | 2024-09-19 |
| A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA PatientsNCT03423186results2021-04-22Safety as Measured by Adverse Events Frequencies (by Type and Severity)Non-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: SOBI003 | Sanfilippo Syndrome Type A (MPS IIIA) | 6 | 2019-10-25actual | — | 2021-11-19 |
| Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical CareNCT02320084observationalOccurrence of Adverse events related to hepatic function | — | Completed | Drug: Nitisinone | Hereditary Tyrosinemia, Type I | 315 | 2019-09-30actual | — | 2024-09-19 |
| Kineret CAPS Post Authorisation StudyNCT02326376observationalRate of serious infections | — | Completed | Drug: anakinra (Kineret) | Cryopyrin-Associated Periodic Syndromes | 12 | 2019-09-12actual | — | 2020-01-18 |
| Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI TherapiesNCT03103542results2021-11-30ITI SuccessOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 4 | Completed | Biological: Recombinant coagulation factor (rFVIIIFc) | Hemophilia A | 16 | 2019-09-04actual | — | 2024-09-19 |
| Long-term Safety Study of Kineret® in Patients With Systemic Juvenile Idiopathic Arthritis (SJIA)NCT03932344observationalThe occurrence of non-serious adverse events (AEs) of at least moderate severity and serious AEs (SAEs), including macrophage activation syndrome (MAS) as an event of special interest (ESI). | — | Completed | Drug: Anakinra | Still Disease, Juvenile Onset | 306 | 2019-08-31actual | — | 2024-09-19 |
| A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Still's Disease (SJIA and AOSD)NCT03265132results2020-04-28Proportion of ACR30 Responders With Absence of Fever Attributable to the Disease During the 7 Days Preceding Week 2.Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Terminatedsponsor's stated reason: Meeting enrolment target (81 pat) will not be feasible within reasonable time. | Biological: anakinra · Drug: Placebo | Still's Disease, Adult-Onset, Still's Disease, Juvenile-Onset | 13 | 2019-02-13actual | — | 2021-06-30 |
| Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic LymphohistiocytosisNCT01818492results2020-09-04Overall Response Rate (ORR) Second LineOpen-label · TreatmentStudy Protocol: US protocol ↗ · Study Protocol: EU protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Completed | Biological: NI-0501 | Primary Haemophagocytic Lymphohistiocytosis | 45 | Jan 2019actual | — | 2023-02-21 |
| A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Acute Gouty ArthritisNCT03002974results2020-07-15Change in Patient-assessed Pain Intensity in the Index Joint From Baseline to 24-72 Hours for the First Gout Flare Treated in the Study as Measured by VASRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Anakinra 100 mg, Triamcinolone Acetonide 40 mg, Placebo to Anakinra 100 mg, Placebo to Triamcinolone Acetonide 40 mg | Acute Gouty Arthritis | 165 | Jun 2018actual | — | 2020-07-15 |
| A Study to Investigate the Potential Influence of Nitisinone on the Metabolism and the Transport of Other Drugs in Healthy VolunteersNCT03103568Area under the curve (AUC) infinity for CYP2C9 (tolbutamide) substrateNon-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: Nitisinone in Arm A, Tolbutamide, Metoprolol, Chlorzoxazone, Furosemide, Nitisinone in Arm B | Drug Drug Interaction | 36 | 2017-06-10actual | — | 2017-08-10 |
| Kineret in the Treatment of Rheumatoid ArthritisNCT02915094observationalDisease Activity Score 28 (DAS28) | — | Completed | Drug: Anakinra | Rheumatoid Arthritis | 30 | Jan 2016actual | — | 2017-08-11 |
| A Non-interventional Retrospective Study of the Current Treatment Practice in European Haemophilia CareNCT02049099observationalTotal dose (IU) issued of Factor VIII during a 12 month period | — | Completed | — | Haemophilia A, Haemophilia B | 1,658 | Sep 2015actual | — | 2016-03-14 |
| Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1NCT02323529Minimum serum concentration (Cmin) of nitisinoneOpen-label · Treatment | Phase 3 | Completed | Drug: Nitisinone | Hereditary Tyrosinemia, Type I | 18 | Sep 2015actual | — | 2015-11-11 |
| Safety and Tolerability of SOBI002 in Healthy Volunteers Following Single and Repeated AdministrationNCT02083666To assess the safety and tolerability of SOBI002 in healthy volunteers following single and repeated adminstrationRandomized · Triple-masked · Treatment | Phase 1 | Terminatedsponsor's stated reason: Transient adverse events observed | Drug: SOBI002, Placebo | Healthy | 24 | Nov 2014actual | — | 2015-07-28 |
| Comparison of rhBSSL With Placebo When Added to Infant Formula or Pasteurized Breast Milk During 4 Weeks of Treatment in Preterm InfantsNCT01413581Growth velocity in grams per kilogram per day during 4 weeks of treatment.Randomized · Triple-masked · Treatment | Phase 3 | Terminatedsponsor's stated reason: Primary endpoint showed no stat significant difference. FU \> M12 terminated. | Drug: rhBSSL (recombinant human bile-salt-stimulated lipase), Placebo | Prevention of Growth Restriction | 415 | Jul 2013actual | — | 2015-07-30 |
| Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules.NCT01857362results2014-10-27The Area Under the Serum Concentration vs. Time Profile During 72 Hours After Dose (AUC72h).Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Nitisinone 20 mg, Nitisinone 10 mg | Healthy | 12 | Jun 2013actual | — | 2021-10-12 |
| Taste and Palatability of Orfadin SuspensionNCT01734889results2014-03-04The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 YearsOpen-label · Treatment | Phase 1 | Completed | Drug: Nitisinone | Hereditary Tyrosinemia, Type I | 18 | Mar 2013actual | — | 2014-11-06 |
| First in Human Study of an Anti-IFN Gamma Monoclonal Antibody in Healthy VolunteersNCT01459562Safety and tolerability of escalating single IV doses of NI-0501.Randomized · Triple-masked · Basic science | Phase 1 | Completed | Drug: NI-0501, Placebo | Healthy Volunteers | 20 | Jan 2013actual | — | 2023-08-09 |
| Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailability of the SuspensionNCT01682538results2014-02-20The Area Under the Serum Concentration Curve (AUC) During 72 Hours After Dose (AUC72h) During Fasting Conditions.Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Nitisinone, capsule, Nitisinone, suspension | Healthy Volunteers | 12 | Oct 2012actual | — | 2014-03-17 |
| A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Subjects With Acute Leukemias Undergoing HSCTNCT00460421results2012-07-03Incidence of Dose Limiting Toxicities (DLTs)Open-label · Prevention | Phase 1 | Completed | Drug: Palifermin, Chemotherapy · Radiation: Total Body irradiation | Leukemia | 27 | May 2011actual | — | 2014-12-05 |
| Characterization of Cyanocobalamin in Healthy Voluteers After Intranasal and Intramuscual AdministrationNCT01306123Relative bioavailabilityRandomized · Open-label · Treatment | Phase 1 | Completed | Drug: Nascobal nasal spray (cyanocobalamin, USP), Vitamin B12-ratiopharm N, injection solution | Focus: Bioavailability | 16 | May 2011actual | — | 2014-11-03 |
| Study to Characterize the Effect of Heparin on Palifermin ActivityNCT01163097results2012-08-23Ratio to Baseline of Epithelial Cell Proliferation as Assessed by Ki67 Staining of Buccal Mucosal Tissue.Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Palifermin, Heparin | Oral Mucositis | 44 | Jan 2011actual | — | 2014-11-06 |
| Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Pasteurized Breast Milk for Preterm InfantsNCT00659243Coefficient of Fat Absorption measured in stoolRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: rhBSSL, Placebo | Replacement Therapy in Preterm Infants | 32 | Feb 2010actual | — | 2014-11-03 |
| Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic InsufficiencyNCT00743483results2014-11-18The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA)Open-label · Treatment | Phase 2 | Completed | Drug: rhBSSL | Cystic Fibrosis, Exocrine Pancreatic Insufficiency | 15 | Jun 2009actual | — | 2014-11-18 |
| Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Infant Formula for Preterm InfantsNCT00658905Coefficient of Fat Absorption in stoolRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: rhBSSL, Placebo | Replacement Therapy in Preterm Infants | 33 | May 2009actual | — | 2014-11-03 |
| A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple MyelomaNCT00434161results2015-03-10Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4)Randomized · Quadruple-masked · Supportive care | Phase 3 | Completed | Drug: Palifermin before only, Placebo, Palifermin before and after | Multiple Myeloma | 281 | Mar 2009actual | — | 2015-03-30 |
| Trial to Evaluate Palifermin in the Reduction of Acute Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Allogeneic Marrow/Peripheral Blood Progenitor Cell (PBPC) TransplantationNCT00189488results2014-09-15Number of Participants With Severe (Grade 3 and 4) Acute Graft Versus Host Disease (GVHD)Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: Palifermin, Placebo, Methotrexate · Other: Conditioning Regimen · Procedure: Allogeneic stem cell transplant | Graft Versus Host Disease, Hematologic Malignancies | 155 | Nov 2008actual | — | 2014-09-15 |
| A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Locally Advanced Head and Neck Cancer Receiving Postoperative RadiotherapyNCT00540332Incidence of treatment-emergent proteinuriaRandomized · Quadruple-masked · Supportive care | Phase 1/2 | Withdrawnsponsor's stated reason: The 20070201 study was terminated based on evaluation of Palifermin solid tumor data. | Drug: Placebo, palifermin | Head and Neck Cancer | 0 | Sep 2008actual | — | 2009-03-02 |
| Study to Evaluate Palifermin in the Reduction of Dysphagia in Patients With Locally Advanced Non-Small Cell Lung Cancer (NSCLC)NCT00094861results2014-03-06Number of Participants With Grade 2 or Higher DysphagiaRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: Palifermin, Placebo, Paclitaxel, Carboplatin · Radiation: Radiotherapy | Dysphagia, Non-Small Cell Lung Cancer, Lung Cancer | 100 | Dec 2007actual | — | 2017-03-14 |
| Efficacy and Tolerability of Novel A2A Agonist in Treatment of Diabetic Neuropathic PainNCT00452777Change in mean 24-hour pain intensity score from baseline using an 11-point Likert NRS assessed over the preceding 24 hours immediately upon awakening in the morningRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: BVT.115959, Placebo | Diabetic Neuropathic Pain | 193 | Dec 2007actual | — | 2014-11-03 |
| Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck CancerNCT00101582results2014-09-15Number of Participants With Severe (Grade 3 or 4) Oral MucositisRandomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Placebo, palifermin, cisplatin chemotherapy · Radiation: Radiotherapy | Mucositis, Solid Tumors, Stomatitis, Head and Neck Cancer, Squamous Cell Carcinoma | 188 | Sep 2007actual | — | 2016-09-27 |
| A Study of Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer Receiving Postoperative Radiotherapy and Concurrent ChemotherapyNCT00626639results2014-03-06Number of Participants With Adverse Events (AEs)Randomized · Double-masked · Supportive care | Phase 1/2 | Completed | Drug: Placebo, palifermin, Cisplatin · Radiation: Radiotherapy | Mucositis, Head and Neck Cancer | 5 | May 2007actual | — | 2017-06-21 |
| PROMPT - Palifermin in Reduction of Oral Mucositis in PBSC TransplantationNCT00352703The primary efficacy endpoints are the incidence (%) and duration of severe oral mucositis (WHO grades 3 or 4).Non-randomized · Open-label · Prevention | Phase 4 | Completed | Drug: Kepivance (Palifermin) | Non-Hodgkin's Lymphoma, Multiple Myeloma | 145 | Apr 2007actual | — | 2014-11-03 |
| Palifermin DDI (Drug Drug Interaction)NCT00361348To evaluate the effect of a continuous intravenous (IV) infusion of unfractionated heparin on the single-dose pharmacokinetics (PK) of palifermin in healthy subjects.Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Palifermin, Heparin | Healthy Volunteers | 45 | Jul 2006actual | — | 2014-11-06 |
| Palifermin for the Reduction of Oral Mucositis in Single-dose Evaluation (PROMISE)NCT00109031results2014-09-15Number of Participants With Severe Oral Mucositis (WHO Grade 3 and 4)Randomized · Double-masked · Supportive care | Phase 3 | Completed | Drug: palifermin, Cyclophosphamide, Etoposide, Placebo · Radiation: Total Body Irradiation | Cancer, Lymphoma, Leukemia | 47 | Feb 2006actual | — | 2014-09-15 |
| Pharmacokinetic Study of Palifermin in Subjects Receiving Radiation Therapy and Chemotherapy Followed by Blood Stem Cell SupportNCT00070616To characterize the PK profile of 3 daily intravenous (IV) doses of rHuKGF before total body irradiation (TBI) / high-dose chemotherapy conditioning treatment and after PBPC transplantationNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Palifermin 6 x 60 μg/kg/day, Palifermin 2 x 180 μg/kg/day, Chemotherapy · Radiation: radiotherapy | Leukemia, Lymphoma, Myeloma | 25 | Mar 2005actual | — | 2014-10-24 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19