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Results for “Mucopolysaccharidosis II (MPS II)”

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Showing 20 of 0 results

Post-approval studies (Phase 4)Study completedNCT00607386
What this trial is testing

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

Who this might be right for
Hunter SyndromeMucopolysaccharidosis IIMPS II
Shire 28
Testing effectiveness (Phase 2)Study completedNCT01155778
What this trial is testing

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 12
Not applicableActive Not RecruitingNCT04597385
What this trial is testing

Long-term Follow-Up for RGX-121

Who this might be right for
Mucopolysaccharidosis II
REGENXBIO Inc. 27
Early research (Phase 1)Study completedNCT04539340
What this trial is testing

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 20
Not applicableWithdrawnNCT04591834
What this trial is testing

Mucopolysaccharidosis Type II Observational

Who this might be right for
Mucopolysaccharidosis II
REGENXBIO Inc.
Large-scale testing (Phase 3)Enrolling By InvitationNCT06075537
What this trial is testing

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 99
Not applicableStudy completedNCT05155488
What this trial is testing

Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Who this might be right for
Mucopolysaccharidosis (MPS)
Takeda 250
Early research (Phase 1)Active Not RecruitingNCT05422482
What this trial is testing

Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

Who this might be right for
Mucopolysaccharidosis IIHunter Syndrome
GC Biopharma Corp 12
Testing effectiveness (Phase 2)Study completedNCT02060526
What this trial is testing

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Who this might be right for
Sanfilippo Syndrome
Shire 21
Testing effectiveness (Phase 2)Active Not RecruitingNCT05665166
What this trial is testing

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis II
University of Manchester 5
Not applicableStudy completedNCT01602601
What this trial is testing

Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723

Who this might be right for
Mucopolysaccharidosis II
GlaxoSmithKline 10
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
Not applicableEnded earlyNCT04628871
What this trial is testing

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

Who this might be right for
Hemophilia BMucopolysaccharidosis IMucopolysaccharidosis II
Sangamo Therapeutics 13
Not applicableStudy completedNCT03292887
What this trial is testing

Hunter Outcome Survey (HOS)

Who this might be right for
Hunter Syndrome
Shire 1,443
Testing effectiveness (Phase 2)Study completedNCT00004454
What this trial is testing

Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis II
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 2
Large-scale testing (Phase 3)Active Not RecruitingNCT04573023
What this trial is testing

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 86
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07818759
What this trial is testing

Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

Who this might be right for
Sanfilippo Syndrome (MPS III)
NeuroGT 9
Post-approval studies (Phase 4)Study completedNCT02455622
What this trial is testing

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Who this might be right for
Hunter Syndrome
Shire 21
Not applicableEnded earlyNCT02095015
What this trial is testing

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 159
Not applicableStudy completedNCT00882921
What this trial is testing

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Who this might be right for
Hunter Syndrome
Shire 26
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