Clinical trials: in Pakistan
Glycogen Storage Disease Type II, Pompe Disease
Pompe Disease Registry Protocol
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both…
- Trial name
- NCT00231400
- Where
- 282 sites in United States, Argentina and 42 more
- Volunteers
- 2,000 people
- Sponsor
- Genzyme, a Sanofi Company
Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
- Trial name
- NCT07082725
- Testing
- Nizubaglustat
- Where
- 34 sites in United States, Argentina and 13 more
- Volunteers
- 72 people
- Sponsor
- Azafaros B.V.
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Wilson's Disease
Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease…
- Trial name
- TRADITiONAL (NCT07465718)
- Testing
- TETA 4HCl formulation, D-Penicillamine
- Where
- 10 sites in United States, China and 2 more
- Volunteers
- 38 people
- Sponsor
- Orphalan
Gaucher Disease, Cerebroside Lipidosis Syndrome
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status.
- Conditions
- Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00358943
- Where
- 317 sites in United States, Argentina and 50 more
- Volunteers
- 12,000 people
- Sponsor
- Genzyme, a Sanofi Company
Chronic Hepatitis D Infection
A Phase III Study to Evaluate the Efficacy and Safety of Libevitug in Participants With Chronic HDV Infection (D-clear Study)
This is an international, multicenter, randomized, controlled, open-label Phase III trial. It will evaluate the efficacy and safety of libevitug in participants with chronic HDV infection.
- Trial name
- NCT07499544
- Testing
- Libevitug 20 mg/kg, Libevitug 10 mg/kg, Delayed treatment with libevitug
- Where
- 7 sites in United States, China and 1 more
- Volunteers
- 160 people
- Sponsor
- Huahui Health
Gaucher Disease, Type 3
High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Type: Prospective, open-label, single center study Duration: 6 months with an optional 12-month extension phase Participants: 12 pediatric patients diagnosed with type III Gaucher disease (GD3) aged ≥3 to ≤18 years old treatment…
- Trial name
- NCT07285369
- Testing
- Ambroxol
- Where
- Pakistan
- Volunteers
- 12 people
- Sponsor
- Agyany Pharma LTD
Patent Ductus Arteriosus
Post Marketing Clinical Follow Up Study to Evaluate Efficacy and Safety of the Occlutech PDA Occluder in Patients With Patent Ductus Arteriosus Defects
This retrospective and prospective, multicenter, international post marketing follow up study evaluates the safety and efficacy of the Occlutech® PDA Occluder, delivered using the Occlutech Occlusions Pusher (OOP), in subjects…
- Trial name
- NCT05264753
- Testing
- Occlutech® PDA Occluder
- Where
- 15 sites in Canada, France and 7 more
- Volunteers
- 255 people
- Sponsor
- Occlutech International AB
Gaucher Disease Type 3
Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).
- Trial name
- NCT07715084
- Testing
- AZ-3102
- Where
- 7 sites in India, Pakistan and 1 more
- Volunteers
- 20 people
- Sponsor
- Azafaros B.V.
Mucopolysaccharidosis I (MPS I)
Mucopolysaccharidosis I (MPS I) Registry
The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I.
- Trial name
- NCT00144794
- Where
- 141 sites in United States, Argentina and 35 more
- Volunteers
- 1,406 people
- Sponsor
- Genzyme, a Sanofi Company
Growth, Tolerance
Human Milk Oligosaccharides (HMOs) - Post Marketing Study on Infants (NeHMO)
Human milk oligosaccharides (HMOs) represent the third largest solid component of breast milk. Technology advancements made it possible to supplement infant formulas with HMOs (2'FL, LNnT).
- Conditions
- Growth, Tolerance, Adverse Event
- Trial name
- NCT07302477
- Testing
- HMO Supplemented Infant Formula
- Where
- 4 sites in Pakistan
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Poliomyelitis, Polio
A Multi-center, Open-labelled, Randomized, Controlled, Extended Phase Ⅲ Clinical Trial of sIPV Vaccine
This study is designed to study the immunogenicity and safety of sIPV co-administered with other routine infant vaccines. According to the national immunization schedule of Bangladesh and Pakistan, sIPV was administered…
- Trial name
- NCT05850364
- Testing
- Sabin Strain Inactivated Poliovirus Vaccine
- Where
- 3 sites in Bangladesh, Pakistan
- Volunteers
- 1,442 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Rabies (Healthy Volunteers)
Immunogenicity and Safety of Rabies Vaccine (Serum-free Vero Cell) in a Simulated Post-exposure Prophylaxis Regimen
To demonstrate the immunogenicity of Sinovac rabies vaccine is non-inferior to the active-controlled rabies vaccine (Verorab®) after post-exposure prophylaxis (PEP) vaccination, and to confirm its satisfying safety profile in the…
- Trial name
- NCT07055893
- Testing
- Rabies vaccine, Verorab®
- Where
- Pakistan
- Volunteers
- 390 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Rabies
Immunogenicity and Safety of Rabies Vaccine (Serum-free Vero Cell) in a Pre-exposure Prophylaxis Regimen
To demonstrate the immunogenicity of Sinovac rabies vaccine is non-inferior to the active-controlled rabies vaccine (Verorab®) after pre-exposure prophylaxis (PrEP) vaccination, and to confirm its satisfying safety profile in the…
- Trial name
- NCT07055295
- Testing
- Rabies Vaccine (Serum-free Vero Cell), Freeze-dried, Verorab®
- Where
- Pakistan
- Volunteers
- 390 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Influenza, Human
Efficacy Study With QIVc in Pediatric Subjects
This phase 3 clinical study is a randomized, observer-blind, multicenter study of QIVc versus a non-influenza vaccine in subjects 6 months though 47 months of age.
- Trial name
- NCT03932682
- Testing
- QIVc, Comparator
- Where
- 74 sites in Bangladesh, Bulgaria and 13 more
- Volunteers
- 5,723 people, healthy volunteers welcome
- Sponsor
- Seqirus
Cough
Clinical Response of Syrup Hydryllin® in Pakistani Subjects With Cough in Routine Practice
A cough is one of the most common medical complaints accounting for as many as 30 million clinical visits per year. It is the single most prevalent symptom on presentation to a physician's office.
- Trial name
- HYD-EFFECT (NCT05129761)
- Testing
- Syrup Hydryllin
- Where
- Pakistan
- Volunteers
- 320 people
- Sponsor
- The Searle Company Limited Pakistan
Healthy Infants
Infant Feeding Practice and Gut Comfort Study
Multi-country, cross-sectional, observational study in diverse geographic locations all over the world.
- Trial name
- NCT03703583
- Testing
- There is no intervention in this observational study.
- Where
- 16 sites in Egypt, India and 4 more
- Volunteers
- 2,804 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Hypomelanotic Macules, Facial Angiofibroma
Biomarkers for Tuberous Sclerosis Complex (BioTuScCom)
International, multicenter, observational, longitudinal study to identify biomarker/s for Tuberous Sclerosis Complex and to explore the clinical robustness, specificity, and long´-term variability of these biomarker/s
- Conditions
- Hypomelanotic Macules, Facial Angiofibroma, Shagreen Patches, Ungual Fibromas
- Trial name
- TuScCom (NCT02654340)
- Where
- 8 sites in Albania, Egypt and 6 more
- Volunteers
- 20 people
- Sponsor
- CENTOGENE GmbH Rostock
Nephritis, Hereditary, Hematuria-Nephropathy-Deafness Syndrome
Biomarker for Alport Syndrome (BioAlport)
International, multicenter, observational, longitudinal monitoring study to identify biomarker/s for Alport syndrome and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s
- Trial name
- BioAlport (NCT02718027)
- Where
- 7 sites in Albania, Georgia and 5 more
- Volunteers
- 12 people
- Sponsor
- CENTOGENE GmbH Rostock
Inborn Errors of Metabolism
Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism (iPSC-IEM)
Human induced pluripotent stem cells (iPSCs), are reprogrammed from somatic cells that can self-renew indefinitely and produce different types of cells. They provide human model cell lines for orphan drug development.
- Trial name
- iPSC-IEM (NCT04097275)
- Testing
- Skin biopsy, Withdraw of blood
- Where
- Pakistan
- Volunteers
- 1,000 people
- Sponsor
- CENTOGENE GmbH Rostock